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New Trends and Most Promising Therapeutic Strategies for Epilepsy Treatment

Antonella riva.

1 Pediatric Neurology and Muscular Diseases Unit, IRCCS Istituto Giannina Gaslini, Genoa, Italy

2 Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genoa, Genoa, Italy

Alice Golda

Ganna balagura.

3 Department of Functional Genomics, Center for Neurogenomics and Cognitive Research, Vrije Universiteit, Amsterdam, Netherlands

Elisabetta Amadori

Maria stella vari, gianluca piccolo, michele iacomino.

4 Unit of Medical Genetics, IRCCS Istituto Giannina Gaslini, Genoa, Italy

Simona Lattanzi

5 Department of Experimental and Clinical Medicine, Neurological Clinic, Marche Polytechnic University, Ancona, Italy

Vincenzo Salpietro

Carlo minetti, pasquale striano.

Background: Despite the wide availability of novel anti-seizure medications (ASMs), 30% of patients with epilepsy retain persistent seizures with a significant burden in comorbidity and an increased risk of premature death. This review aims to discuss the therapeutic strategies, both pharmacological and non-, which are currently in the pipeline.

Methods: PubMed, Scopus, and EMBASE databases were screened for experimental and clinical studies, meta-analysis, and structured reviews published between January 2018 and September 2021. The terms “epilepsy,” “treatment” or “therapy,” and “novel” were used to filter the results.

Conclusions: The common feature linking all the novel therapeutic approaches is the spasmodic rush toward precision medicine, aiming at holistically evaluating patients, and treating them accordingly as a whole. Toward this goal, different forms of intervention may be embraced, starting from the choice of the most suitable drug according to the type of epilepsy of an individual or expected adverse effects, to the outstanding field of gene therapy. Moreover, innovative insights come from in-vitro and in-vivo studies on the role of inflammation and stem cells in the brain. Further studies on both efficacy and safety are needed, with the challenge to mature evidence into reliable assets, ameliorating the symptoms of patients, and answering the challenges of this disease.


Epilepsy is the enduring predisposition of the brain to generate seizures, a condition that carries neurobiological, cognitive, psychological, and social consequences ( 1 ). Over 50 million people worldwide are affected by epilepsy and its causes remain partially elusive, leaving physicians, and patients an unclear insight into the etiology of the disease and the best treatment approach ( 2 ). Over than 30% of individuals do not respond to common anti-seizure medications (ASMs) and are addressed to as “drug-resistant,” a term which the International League Against Epilepsy (ILAE) applies to those patients who do not respond to the combination of two appropriately chosen and administered ASMs ( 3 , 4 ). Hence, a great deal of responsibility laid upon the research and development of innovative pharmacological and non-pharmacological approaches given a targeted approach, aiming at improving the symptoms of patients and their quality of life (QoL), together with that of the caregivers.

As several investigations are currently in progress, this review aimed to discuss the novel therapeutic insights, with the hope they may establish as turning points in the treatment of patients in the next few years.

A search on PubMed, Scopus, and EMBASE databases using the terms “epilepsy,” “treatment” or “therapy,” and “novel” was conducted. The search covered the period between January 2018 and September 2021. Existing literature was reviewed, including both experimental and clinical studies, meta-analysis, and topic reviews summarizing the most up-to-date researches. Only studies published in English were reviewed.

Precision Medicine

Precision medicine (PM) is an outstanding approach tended to use the genetics, environment, and lifestyle of individuals to help determine the best “way” to prevent or treat disease ( 5 ). It embeds a holistic evaluation, assessing not only the effect of an own condition but also that of treatment ( 6 ). Precision medicine is endorsed in epilepsy management for many decades, as in the clinical practice ASMs are selected after a careful and pointful evaluation of seizure types of patients, their epilepsy syndrome, comorbidities, concomitant drugs, and expected vulnerability to specific adverse events (AEs) ( 7 ). Discoveries and progress in genetics have provided the strongest basis for PM: as more and more genes are being identified as disease-causing, hope has grown on possible targeted approaches ( 6 ). An “ideal” therapy would be able to both relieve symptoms and reverse the functional alterations caused by specific genetic mutations. This firstly implies identifying putative disease-causing genes and, secondly, the specific functional alterations caused by the pathogenic variants. Lastly, it should have been demonstrated that therapeutic intervention may modify the effect caused by the mutation.

The ketogenic diet (KD) used to treat glucose transporter 1 (GLUT1) deficiency syndrome is probably the best example of PM applied to epilepsy. In GLUT1 patients the uptake of glucose into the brain is impaired because of the SLC2A1 mutation, hence, the KD provides neurons with an alternative source of energy, compensating for the consequences of the metabolic defect ( 8 ). Another clear application of a PM-based approach is the avoidance of those drugs which may cause worsening of seizures by exasperating the underlying molecular defect, i.e., sodium channel blockers must be avoided in patients with Dravet syndrome (DS) carrying loss-of-function mutations in the sodium voltage-gated channel alpha subunit 1 ( SCN1A ). Another one is memantine for the treatment of GRIN-related disorders due to gain-of-function mutations in the NMDA receptor ( 8 – 11 ) or quinidine and retigabine for epilepsies caused by potassium channels genes mutations ( KCNT1 and KCNQ2 ) ( 6 , 12 ). In epileptic encephalopathies (EE), it would be also of interest to investigate the effect of a PM treatment on cognitive function, to that targeting a specific gene mutation and abolishing related epileptic activity may result in improved cognitive functions ( 10 ).

Precision medicine may prove complex, as the same mutation may cause quite different clinical phenotypes; moreover, additional genetic variants may contribute to modifying a phenotype. Again, wide-genome variations or even the epigenome may influence the resulting expression of pathogenic variants ( 5 ).

Nowadays, evidence indicates PM may be applied to individuals with both rare and common forms of epilepsy, and, consequently, drug development is increasingly being influenced by PM approaches. Although extensive research focuses on genome-guided therapies, important opportunities also derive from immunosuppressive therapies and neuroinflammation-targeting treatments ( 2 , 13 ). The identification of cellular and molecular biomarkers would possibly allow clinicians to have early prediction markers of a disease and its progression. Additionally, it could lead to the development of unique models to cost-effectively screen treatments and also decrease the costs of clinical trials through better patient selection ( 14 ).

Novel Mechanisms of Anti-Seizure Medications

Many medications are currently under study in clinical practice, ranging from those with a mechanism similar to that of well-known ASMs, like the GABA-A receptor agonists, to those with novel mechanisms such as the stimulation of melatonin receptors. Moreover, some drugs are yet known medications, previously used for other indications; while a large group remains orphan of a well-comprised mechanism of action ( 6 ). It is in this perspective, that the wider term ASMs should be addressed, aiming at referring to the large heterogeneity of action mechanisms nowadays available to counteract seizures.


In 2018, the Food and Drug Administration (FDA) approved the first-in-class drug derived from the cannabis plant. Although the precise mechanism by which the cannabidiol (CBD) exerts its anti-seizure effects is still poorly known, it seems not to act through interaction with known cannabinoid receptors ( 15 ), but holds an affinity for multiple targets, resulting in the reduction of neuronal excitability which is relevant for the pathophysiology of the disease ( 16 , 17 ).

Cannabidiol is approved for the treatment of seizures in children with DS or Lennox-Gastaut syndrome (LGS) aged 2 years or older, based on three pivotal phases 3 trials ( 12 ). In 2019 CBD gained approval in Europe in conjunction with clobazam (CLB), based on clinical trial data showing that the combination of both CBD and CLB resulted in greater efficacy outcomes ( 16 ).

The first clinical trial ( 17 ) included 120 patients with DS aged between 2 and 18 years. The median frequency of convulsive seizures decreased from 12.4 to 5.9 per month, as compared with a decrease from 14.9 to 14.1 per month with the placebo. Furthermore, 43% of patients in the active arm and 27% in the placebo group showed at least a 50% reduction in the convulsive-seizure frequency. Overall, 62% of patients under CBD did gain at least one category at the seven-category Caregiver Global Impression of Change scale, as compared to 34% in the placebo group. Five percent of patients under CBD became seizure-free, while none in the placebo group did.

Another randomized, double-blind, placebo-controlled, trial ( 18 ) included 171 LGS patients aged between 2 and 55 years and measured the reduction in drop-seizures. The median percentage reduction was 43.9% in the CBD group and 21.8% in the placebo group. In 2018, Devinsky et al. ( 19 ) compared a lower 10 mg/kg/day dose of CBD with the full 20 mg/kg/day in LGS patients. A median 41.9% reduction in drop-seizure frequency was observed in the 20-mg CBD group, while the median reduction was 37.2% in the 10-mg group and 17.2% in the placebo group. Although this study demonstrated patients may gain benefit in seizure reduction by increasing the dose to 20 mg/kg/day, it also displayed an increased risk in AEs. It is generally recommended to begin at 5 mg/kg divided into two intakes a day, then increase to 10 mg/kg/day. If the 10 mg/kg/day dose is well-tolerated and the anti-seizure effect continues, dosing can be increased to the maximum of 20 mg/kg/day ( 15 ).

Cannabidiol also proved to effectively reduce seizure frequency at long-term follow-up ( 20 ), retaining a consistent reduction (between 42.9 and 44.3%) in seizure frequency at 48 weeks of follow-up. Moreover, 5 out of 104 patients (4.8%) were convulsive seizure-free at 12 weeks of treatment, with more than 40% having a reduction of convulsive seizure frequency ≥50% at each programmed visit of follow-up ( 18 ). In terms of median percentage reduction in convulsive seizures, rates of responders, reduction in total seizures, and CGIC-assed improvements, CBD proved greater in the subset of patients concomitantly treated with CLB. Moreover, the combination CBD+CLB showed a benefit in the number of convulsive seizure-free days ( 16 ). However, a drug-to-drug interaction increasing levels of active metabolites of both compounds must be assessed and hence CLB dose reduction is recommended if patients experience somnolence or sedation ( 15 , 16 ).

In conclusion, RCTs settle CBD as a well-tolerated drug, with patients primarily experiencing somnolence, diarrhea, and decreased appetite. The elevation of liver transaminases may be observed mostly in patients on concomitant valproate, and the dose reduction of valproate or CBD is often decisive. The efficacy of CBD on both convulsive and drop seizures is established, with retained efficacy at long-term follow-up. New RCTs in other syndromic or isolated epilepsies populations may widen the field of use of CBD in the next few years.


Fenfluramine (FFA), formerly used at 10 times higher dosage (up to 120 mg/day) as a weight-loss drug, exerts its anti-seizure effect both through the release of serotonin which stimulates multiple 5-HT receptor subtypes, and by acting as a positive modulator of sigma-1 receptors ( 16 , 21 – 23 ). Fenfluramine has been approved by the FDA in June 2020 and is currently under evaluation by the European Medicines Agency (EMA). The drug proved significantly effective in reducing seizures in phase-3 trials on DS patients: the 0.8 mg/kg/day treated group did experience a mean 64% reduction in seizures as compared to 34% in the 0.2 mg/kg/day group. Notably, a >75% reduction in seizures occurred in 45% of patients under 0.8 mg/kg/day, in 20.5% of those on 0.2 mg/kg/day compared to 2.5% in the placebo group ( 23 ). Fenfluramine has then continued to provide a clinically meaningful reduction in convulsive seizure frequency over a median of 445 days of treatment. The median percent reduction in monthly convulsive seizures frequency was 83.3%. Overall, 62% of patients showed a 50% reduction in convulsive seizure frequency ( 16 ).

Together with the anti-seizure effect, FFA has also relatively few drug-drug interactions, primarily a moderate effect on stiripentol (STP), which requires the downward adjustment of FFA dosing to.5 mg/kg/day. No additional interaction with other drugs such as valproate, CLB, and CBD are known ( 15 ). The most common AEs reported under FFA treatment include decreases in appetite, weight loss, diarrhea, fatigue, lethargy, and pyrexia ( 16 ). The main AEs leading to FFA withdrawal as a weight-loss agent were the occurrence of valvular heart disease (VHD) and pulmonary arterial hypertension (PAH), for which 6-month-echocardiographic monitoring is required together with an ECG. However, at the anti-seizure dosages, no VHD or PAH was observed after a median duration treatment of 256 days. No ECG alterations indicative of atrioventricular conduction or cardiac depolarization alterations were seen, and no mitral or aortic valve regurgitation greater than “trace” was observed in any of the 232 patients with DS who participated in the open-label extension (OLE) study ( 21 , 24 , 25 ).

Cenobamate ( Xcopri or YKP3089 ) is a new ASM that has recently gained approval by the FDA for the treatment of focal-onset seizures in adults. The EMA is currently reviewing the drug for approval as an adjunctive treatment in focal-onset epilepsies. Cenobamate is a tetrazole-derived carbamate compound with a dual mechanism of action; the drug can both enhance the inactivated state of voltage-gated sodium channels, and act as a positive allosteric modulator of the GABA-A receptors, binding at a non-benzodiazepine site ( 26 ).

A multicenter, randomized study of patients with uncontrolled focal seizures ( 27 ) showed that the adjunctive cenobamate, with dosage groups of 100, 200, and 400 mg/day led to a consistent reduction in focal-seizures frequency after 18-weeks of treatment, with the greatest reduction observed in the 200 and 400 mg/day doses groups. A similar dose-effect relationship was seen when evaluating the responder rates (≥50% in seizure reduction). Post-hoc analysis proved seizure frequencies decreased early during cenobamate titration; while, during the 12-week maintenance phase, significantly more patients under the active 200 or 400 mg/day harms achieved seizure freedom as compared to that receiving placebo. Cenobamate is overall well-tolerated, showing mild to moderate severity AEs on the CNS system, such as somnolence, dizziness, and disturbances in gait and coordination, with a linear incidence-dose correlation and disappearance at maintenance. Four cases of hypersensitivity adverse reactions occurred during two RCTs, including one serious AEs of Drug Rash with Eosinophilia and Systemic Symptoms (DRESS) ( 26 , 27 ). In this case, the rapid titration of 100 mg/week from 200 to 400 mg dose might have contributed to the higher rates of AEs in the 400 mg group; a lower starting dose and a slower titration rate have been shown to reduce the occurrence of hypersensitivity reactions, possibly through the development of immune tolerance ( 27 ). As cenobamate inhibits the P450 family cytochrome CYP2C19 * 18, dosing adjustment is needed when adding cenobamate to ASM regimens containing phenytoin or phenobarbital ( 28 ); moreover, a dose reduction of CLB should be considered, counteract the increase in plasma levels of desmethylclobazam, its active metabolite. Cenobamate has also been shown to decrease by 25% the plasma exposure to carbamazepine, through the induction of the CYP3A4. Cenobamate could shorten the QT-interval on the ECG in a dose-dependent manner. Hence, cenobamate is contraindicated in patients with familial short QT syndrome, and caution is required in co-administration with other drugs known to reduce the QT interval since a synergistic effect may occur ( 26 , 27 ). In a short time, data will help to assess cenobamate active time-window on seizures control and real-life data will help to acknowledge whether freedom rates will be borne out in clinical practice. The mechanisms of action and the potential additive or synergistic interactions of cenobamate with concomitant ASMs also warrant further investigation ( 26 ).

Novel Non-Pharmacological Treatments

Neurostimulation comprises different techniques, already implemented in the clinical practice, direct to deliver electrical or magnetic currents to the brain in a non-invasive or invasive way and hence modulating neuronal activity to achieve seizure suppression.

Vagal Nerve Stimulation

Vagal nerve stimulation (VNS) is approved both in Europe and in the United States as an adjunctive treatment in patients with refractory epilepsies, and it is routinely available in many epilepsy centers, with more than 100,000 patients treated worldwide ( 6 ). Vagal stimulation may then turn off seizures originating in regions susceptible to heightened excitability, such as the limbic system, thalamus, and thalamocortical projections. Moreover, an additional mechanism of action derives from the activation of the locus coeruleus and the raphe nuclei, and the regulation of the downstream release of norepinephrine and serotonin, both having antiepileptic effects ( 29 ).

Two large RCTs showed VNS efficacy in reducing seizures, achieving a 50% reduction in 31% of patients, and over 50% seizures reduction in 23.4% of the studied population. On the other hand, seizure freedom at long-term follow-up was observed in <10% of patients. Side effects are usually mild and include hoarseness, throat paraesthesia or pain, coughing, and dyspnea. This tends to improve over time or through the adjustment of setting parameters ( 6 ).

In conclusion, evidence suggests VNS is well-tolerated in both children and adults with drug-resistant partial epilepsies ( 30 – 32 ); moreover, the newest VNS models can detect ictal tachycardia and automatically deliver additional stimulation to abort seizures or reduce their severity ( 6 ).

Transcutaneous VNS

Developed as a non-invasive alternative to VNS, the transcutaneous VNS (tVNS) acts on the auricular branch of the vagus nerve (ABVN), targeting thick-myelinated afferent fibers in the cymba conchae, and hence activating the ipsilateral nucleus of the solitary tract (NTS) and locus coeruleus. This activation pathway overlaps with the classical central vagal projections, leading to a brain activation pattern similar to that produced by invasive VNS ( 33 ). The device consists of a programmable stimulation apparatus and an ear electrode ( 34 ). Stimulation setup is adjusted by applying decreasing and increasing intensity ramps and achieving a level just above the individual detection threshold, but clearly below that of pain. Patients usually apply tVNS for 1 h/three times per day ( 33 ) and adherence is usually high (up to 88%) ( 35 ). Trials converge in demonstrating up to 55% reduction in seizure frequency, with mild or moderate side effects mainly including local skin irritation, headache, fatigue, and nausea ( 6 , 35 ).

Deep Brain Stimulation

Deep brain stimulation (DBS) is a minimally invasive neurosurgical technique, which through implanted electrodes can deliver electrical stimuli to deep brain structures. Patients with refractory focal epilepsies and not eligible for surgery are usually good candidates ( 29 ). Both stimulation of the ictal onset zone and the anterior thalamus have gained approval by the FDA as effective stimulation sites , providing a significant and sustained reduction in seizures together with the improvement of the QoL. Nowadays, both DBS and responsive neurostimulation (RNS) are available, being the latter a system able to monitor electrical changes in cortical activity and give small pulses or bursts of stimulation to the brain to interrupt a seizure ( 36 ). The interim results of a prospective, open-label, and long-term study did show that the median 60% or greater reduction in seizure frequency is retained over years of follow-up. Moreover, the majority of patients took advantage of treatment with the RNS® System, and 23% experienced at least one 6-month period of seizure freedom ( 37 ). The most relevant reported side effects were depressive mood and memory impairment, besides the local side effect of implantation. Nonetheless, it should be stated that RNS is a feasible option in most epilepsy centers in the US, but its use remains limited in other parts of the world. In these cases, DBS could be an option with targets and stimulation parameters selection are largely driven by the experience of the referred center ( 38 , 39 ).

Trigeminal Nerve Stimulation

Trigeminal nerve stimulation (TNS) is a novel neuromodulation therapy, designed to deliver high frequencies stimulation in a non-invasive way, hence modulating mood and relieving symptoms in drug-resistant epilepsies. The study of DeGeorgio et al. ( 40 ) found that the responder rate (at least 50% reduction in seizures) was 30.2% in the active group, while it was 21.1% in the control group. Moreover, the responder rate did further increase over the 18-week treatment period in the actively treated group. TNS was overall well-tolerated and, when occurring, treatment-related AEs were mild including anxiety (4%), headache (4%), and skin irritation (14%). However, long-term follow-up studies showed inconclusive results ( 6 ), meaning further studies and patient monitoring will be needed in the next years.

Transcranial Direct Current Stimulation

The transcranial direct current stimulation (tDCS) displays the use of two skull electrodes (anode and cathode) to induce widespread changes of cortical excitability through weak and constant electrical currents. Cortical excitability may increase following anodal stimulation, while it generally decreases after cathodal stimulation. Based on this principle, hyperpolarization using cathodal tDCS has been proposed to suppress epileptiform discharges. Major six clinical studies are promising with 4 (67%) showing an effective decrease in epileptic seizures and 5 (83%) exhibiting a reduction of epileptiform activity. However, some results may be misleading due both to the small and heterogeneous nature of the studied populations and to the different setting parameters applied. Hence, nowadays the major achievement is the demonstration that tDCS may be effective and safe in humans; however, further studies will be needed to define setting stimulation protocols and understand the long-term tDCS effectiveness ( 41 ).

Transcranial Magnetic Stimulation

The nerve cells of a brain to a maximum depth of 2 cm can be stimulated using transcranial magnetic stimulation (TMS). To this, low-frequency and repetitive magnetic stimulations have been shown to induce long-lasting reductions in cortical excitability and, hence, have been proposed as a treatment for drug-resistant epilepsies ( 4 ). Probably, it is the repeated nature of magnetic pulses which allows modulating the neuronal activity, wherein high frequencies (>5 Hz) would have an overall excitatory effect, while low-frequencies (0.5 Hz) would exert an inhibitory effect on neurons ( 29 ).

Despite the optimal stimulation parameters still needing to be clearly defined, they are likely of crucial importance because treatment intensity depends both on the number of pulses and the number of sessions applied over the treatment period. Superior results are achieved in patients with neocortical epilepsy, whit a calculated effect size of 0.71 and 58–80%. This makes sense taking into account the rapid decay of the strength of the magnetic field with distance hence no adequate secondary currents can be elicited in the deep cortex. However, evidence suggests the effects of repetitive TMS may not be restricted to the only site of stimulation but may spread from focal areas to wider areas of the brain.

In conclusion, results should be reproduced in larger cohorts with double-blinded randomized trials, but are promising if compared to the effects currently achieved with invasive neurostimulation techniques for the treatment of epilepsy ( 42 ).

Neuroinflammation and Immunomodulation

Nowadays, the neuroinflammatory pathways are known to contribute to both the development and progression of epilepsy and could be targeted for disease-modifying therapies in epilepsies of wide-range etiologies. Studies on patients with surgically resected epileptic foci have demonstrated inflammatory pathways may be involved, hence the neuroinflammation is not merely a consequence of seizures or brain neuropathology but may induce seizures and brain anatomical damage itself ( 2 ).

Finally, any inflammatory response within the brain will be associated with the blood-brain barrier (BBB) dysfunction. Evidence indicates that BBB opening and the subsequent exposure of brain tissue to serum proteins induces upregulation of proinflammatory cytokines and complement system components: this suggests positive feedback between increased brain permeability, local immune/inflammatory response, and neuronal hypersynchronicity ( 43 ).

It should also be considered that overall neuroinflammation is a negative disease modifier in epilepsy, however, some inflammatory processes may be involved in tissue repair and brain plasticity after injury hence interference with these beneficial mechanisms should be avoided: anti-inflammatory intervention in the wrong patient and at the wrong time could be ineffective or even harmful. Yet, it is for this reason that evidence remains set at the preclinical level with few reports of use in the clinical practice. The discovery of non-invasive biomarkers of pathological neuroinflammation would enable physicians to identify patients who could benefit from the treatments, also providing a potential marker of therapeutic response.

IL-1R1-TLR4 Signaling

The Interleukin IL-1R1-TLR4 signaling pathway originates the neuroinflammatory cascade in epilepsy through increased levels of either the endogenous agonists or their receptors, or even a combination of both ( 2 ). These findings prompted the clinical use of anakinra, the recombinant, and modified form of the human IL-1Ra protein. Case report studies of Anakinra in patients with intractable seizures did result in a significant reduction of seizure activity and improvement of cognitive skills ( 44 ). Moreover, IL-1R1 and TLR4 signaling have been targeted by specific, non-viral, small interfering RNAs (siRNAs) to knock down the inflammasomes or caspase 1 in rats with kindling-induced SE ( 45 ).


Prostanoids are a family of lipid mediators generated from the cell membrane arachidonic acid by cyclooxygenase enzymes 1 and 2 (COX1 and COX2). Prostanoids bind to specific G protein-coupled receptors (GPCR), hence regulating both innate and adaptive immunity ( 46 ).

Monoacyl Glycerol Lipase

The monoacyl glycerol lipase (MAGL) is a lipase constitutively expressed by neurons and a key enabler of 2-arachidonoylglycerol (2-AG) hydrolysis. 2-Arachidonoylglycerol is an endocannabinoid, which likewise prostaglandins are involved in seizures genesis. Hence, the upstream inhibition of the MAGL has the potential to be an effective target in epilepsy therapy ( 2 ). In 2018 Terrone et al. ( 47 ) did demonstrate CPD-4645 (a selective and irreversible MAGL inhibitor) was effective in terminating diazepam-resistant status epilepticus (SE) in mice. Moreover, clinically relevant outcomes such as reduced cognitive deterioration were ensured by CPD-4645 action: reducing post-SE brain inflammation to prevent neural cell damage. Lastly, the authors noted that SE was more promptly stopped in those mice concomitantly receiving the KD, hence suggesting brain inflammation is the common, final, target. Striking inflammation through different inflammatory pathways may enhance neuroprotection and seizure control.

COX2 Inhibitors and Prostaglandin Receptor Antagonists

Targeting the inducible enzyme COX2 to that of blocking the prostanoid cascade has been tested to interfere with acute seizures or SE. The importance of timing was demonstrated by early anti-inflammatory interventions showing worsening seizures as compared to late-onset interventions ( 2 , 48 ). Prostaglandin F 2α (PGF), which is anti-ictogenic, is indeed predominant in the first hour after SE onset, then the ratio between PGF and the pathogenic prostaglandin E 2 (PGE) normalizes in association with an increase in COX2 synthesis ( 2 ). Hence, punctual COX2-related treatments have been considered to prevent epileptogenesis and reduce the frequency of seizures in epileptic patients. COX2 inhibition could either be selective ( coxibs = selective COX2 inhibitors) or non-selective ( aspirin ). In two in-animal studies testing celecoxib and parecoxib over evoked SE, treatment with celecoxib or parecoxib did show to consistently reduce the number and severity of seizures, together with the improvement of spatial memory deficits ( 2 ).

Non-selective blockade of COX2 has been also tested in experimental models of epilepsy, and ASA administration over the chronic, latent, epileptic phase could consistently suppress recurrent spontaneous seizures and inhibit the seizure-induced neuronal loss, preventing aberrant neurogenesis in the hippocampus. Thus, ASA is being actively investigated and has the potential to prevent the epileptogenic processes, including SE occurrence, and may avoid pathological alterations in CNS areas ( 2 , 49 ). Potential cardiotoxicity is the main limit, bordering COX2 inhibition in clinical practice.

Shifting attention downstream to prostaglandin receptors, highly potent PGE receptor (EP2R) antagonists administered from a 4 h-starting point after the onset of pilocarpine-induced SE, proved to mitigate deleterious consequences such as delayed mortality, functional deficits, alterations of the BBB permeability, and hippocampal neurodegeneration ( 50 ). The delayed timepoint of administration further brings evidence that EP2R blockade may allow obtaining neuroprotection later in SE stages, mainly reducing long-term sequelae ( 2 ).

Inflammatory Response Lipid Mediators

Specialized pro-resolving lipid mediators that activate GPCRs have a major role in controlling inflammatory responses in peripheral organs. G protein-coupled receptors activation leads both to reduced expression of pro-inflammatory molecules and increased synthesis of anti-inflammatory mediators which can modulate immune cell trafficking and restore the integrity of the BBB. Neuroinflammation was reduced after the intracerebroventricular injection of the omega-3 (n-3) docosapentaenoic acid-derived protectin D1 (PD1 n−3DPA ) in mouse models of epilepsy. Interestingly, recognition of memory deficits after SE also gained improvements ( 2 , 51 ). Since PD1 n−3DPA derives from n-3 polyunsaturated fatty acids (PUFAs), in humans, it may be possible to non-invasively increase PD1 n−3DPA levels through the dietary intake of n-3 PUFAs, which are found in flaxseed, walnuts, marine fish, and mammals ( 52 ). Another way may then be the developing stable analogs of pro-resolving lipids ( 51 ).

Oxidative Stress

Activation of the Toll-like receptors (TLRs) can lead to reactive oxygen species (ROS) production, hence promoting and sustaining inflammatory pathways. The detrimental effects of ROS are usually counteracted through the activation of the nuclear factor E2-related factor 2 (Nrf2). Activated Nrf2 translocates to the nucleus where it heterodimerizes with the small Maf proteins (sMaf) and binds to the antioxidant response element (ARE 5′-TGACXXXGC-3′) battery activating transcription of genes that are involved in antioxidant and cytoprotective tasks ( 53 ).

Transient administration of N -acetyl-cysteine (NAC), a glutathione precursor, did prove to activate Nrf2 in mouse models of SE, thus inhibiting high mobility group box 1 (HMGB1) cytoplasmic translocation in the hippocampal neural and glial cells and preventing the linkage between oxidative stress and neuroinflammation for which the redox-sensitive protein HMGB1 is central ( 2 ). Also, high doses (4–6 g/day) of NAC were used in Unverricht-Lundborg disease (ULD), progressive myoclonus epilepsy (PME), showing overall improvement of myoclonus, ataxia, and generalized tonic–clonic and absence seizures. Neuroprotection and improvements in spatial learning abilities were also observed with retained beneficial effects during treatment ( 54 , 55 ).

Adeno-associated viral (AAV) vectors gene delivery may provide long-term, persistent, induction of Nrf2 expression in a variety of cell types in the brain, with minimal toxicity. The injection of AAV coding for human Nrf2 in the hippocampus of mice with spontaneously recurrent seizures resulted in a reduction in the number and duration of generalized seizures, which interestingly was performed in the already established epileptic phase, highlighting the direct potential of such interventions in the treatment of epilepsy ( 56 ).

Inhibition of P-Glycoproteins

One of the major neurobiological mechanisms proposed to cause drug resistance in epilepsies lays in the removal of ASMs from the epileptogenic tissue through the expression of multidrug efflux pumps such as the P-glycoproteins (P-gps). P-glycoproteins are the final encoded product of the human multi-drug resistance-1 ( MDR-1 ) gene, and play a role in treatment response possibly inducing MDR ( 57 , 58 ). The increased activity of P-gps reduces clinically effective concentrations of ASMs despite adequate serum concentrations, reversing the anti-seizure effects on epileptogenic areas in the parenchyma of the brain ( 3 ).

Following the general rule that the higher the lipophilicity of a drug, the faster the entrance into the brain ( 59 ), available ASMs are very lipophilic, but more than one-third of the patients do not respond to treatment. The possible reason may be ASMs serve as P-gps substrates; secondly, the P-gps levels are higher ( 3 ). Different clinical studies had shown poor prognoses associated with MDR1 gene products, which gave rise to extensive experimental research on the P-gps ( 3 ). The adjunctive use of a P-gps inhibitor might counteract drug resistance and efficiently decrease seizure frequency. In addition to verapamil, other first-generation P-gps inhibitors include nifedipine, quinidine, amiodarone, nicardipine, quinine, tamoxifen, and cyclosporin A. It is primarily due to the lack of selectivity and the pharmacokinetic interactions that trials using such agents failed to rule out P-gps inhibition efficacy in other fields such that of oncology ( 60 , 61 ). First-generation MDR inhibitors required high concentrations to reverse MDR and thus were associated with unacceptable toxicity. In recent years, second and third-generation compounds have been developed which are more selective, highly potent, and non-toxic. Notwithstanding second-generation agents have better tolerability, they still have unpredictable pharmacokinetic interactions (i.e., valspodar is a substrate for cytochrome P450, altering plasma availability of co-administered drugs) and may inhibit other transport proteins. Third-generation inhibitors have more advantages such as high specificity for P-gp, lack of non-specific cytotoxicity, relatively long duration of action with reversibility, and good oral bioavailability. However, despite their selectivity and potency, also this last generation of MDR modulators is far from being perfect and further studies will be needed to outline their effectiveness and safely overcome drug resistance ( 3 , 60 ). As pertains to clinical research, Iannetti et al. ( 62 ) first demonstrated the action of verapamil in a case of prolonged refractory SE and then, subsequently on small series of other types of drug-resistant epilepsies ( 63 , 64 ).

A novel, yet preclinical, approach for reversing multidrug resistance in epilepsy may derive from the modulation of P-gp by herbal constituents. Nowadays, several herbal formulations and drugs which act by modulating P-gps are available and can be explored as alternative treatment strategies. For example, curcumin (the natural dietary constituent of turmeric) orally administered to pentylenetetrazole-kindled epileptic mice models is known to prevent seizures and related memory impairments ( 65 ). The mechanism of action may lie on that curcumin and can reverse multidrug resistance. Hence, curcumin synthetic analogs, which hold more favorable pharmacodynamic properties, have been developed (i.e., GO-Y035); or curcumin has been encapsulated in nanoparticles (NPs) enhancing its solubility and sustaining release inside the brain ( 66 ).

Again, piperine (an alkaloid present in black pepper) and capsaicin (the active component of chili peppers) are known to increase curcumin and other P-gps substrates bioavailability and can be therefore used as basic molecules for the development of non-toxic P-gps inhibitors ( 67 , 68 ).

In conclusion, the identification of an optimal P-gps inhibitor that is potent, effective, and well-tolerated, is desirable to reverse MDR in epileptic patients and will be the challenge of the upcoming years.

Gene Therapies

Currently lying at the preclinical evidence, gene-based therapy modulates gene expression by introducing exogenous nucleic acids into target cells. The delivery of these large and negatively charged macromolecules is typically mediated by carriers (called vectors) ( 69 ). In treating epilepsy, the main hitch is the BBB, which prevents genetic vectors from entering the brain from the bloodstream. Consequently, an invasive approach may be needed ( 29 ). Moreover, several considerations need to be taken into account when translating gene therapy into clinical practice, namely the choice of the viral vector, promoter, and transgene ( 6 ).

Viral Vectors

Viral gene therapy may employ three classes of viral vectors, namely, adenovirus (AD), adeno-associated virus (AAV), and lentivirus. All these three viral vectors have successfully demonstrated to attain high levels of transgene delivery in in-vivo disease models and clinical trials. However, the risks of immunogenic responses and transgene mis-insertions, together with problems in large-scale production are still a deal to face ( 70 ).

Adeno-associated viruses belong to the Parvoviridae family and proved to retain favorable biology, leading their recombinant forms (rAAVs) to become the main platform for current in-vivo gene therapies ( 29 ). A limited clinical trial on patients with late-infantile neuronal ceroid lipofuscinosis (LINCL) did prove neurosurgical gene therapy to be practical and safe, supporting the potentialities of this kind of approach ( 71 ). However, in the view of removing invasiveness, interest was moved to engineered capsid which can confer the ability to cross the BBB and transduce astrocytes and neurons, allowing direct intravenous injection. This was achieved through a process of directed selection in a mouse strain, and further work would be needed to develop a similar variant for use in humans ( 6 , 72 ).

Retroviruses such as lentivirus share with AAVs the ability to infect neurons and lead to a stable expression of transgenes. Lentiviral vectors (lentivectors) are RNA viruses and the transgenes can integrate into the host genome through the reverse transcriptase gene. However, possible insertional mutagenesis may be reduced by using integration-deficient lentivectors, which simultaneously ensure stable transduction ( 73 ). Lastly, lentivectors can package larger genes or regulatory elements as compared to AAVs ( 6 ).

Different viral vectors intrinsically tend to infect different neuronal and glial subtypes, but the high specificity of the target is far from their properties. Hence, several efforts have been made that to identify specific neuron-type targeting promoters: the calcium/calmodulin-dependent protein kinase II (CamKII) promoter is suitable to manipulate excitatory neurons in the forebrain; on the other hand, targeting inhibitory interneurons may be difficult as promoters for specific GABAergic neurons are poorly defined ( 6 ). Finally, the optimal promoter should provide the expression of a level of transgene which is sufficient to moderately alter cell properties but avoids cytotoxicity ( 6 , 74 ).

As for the transgene, gene therapies have been commonly built on the basis that the excitation–inhibition balance is altered in epilepsy. Hence, on a general principle, gene therapy may work through modulating the expression of neuropeptides, and regulation of the neuropeptide Y (NPY) did already show promise, acting both on pro-excitatory Y1 and pro-inhibiting Y2 receptors in the hippocampus ( 6 , 75 ). Another way may be that of regulating potassium channels; overexpression of the potassium channel Kv1.1 proved effective in preventing epileptogenesis in a mouse model of focal epilepsy, the physiological basis may lie on the modulation of both neuronal excitability and neurotransmitter release ( 76 , 77 ). Lastly, chemogenetics refers to the possibility to use gene transfer to express receptors that are insensitive to endogenous neurotransmitters but highly sensitive to exogenous drugs, in a receptor-to-drug therapeutic approach. This promising approach will also allow adjusting the activating drugs to find the optimum dosage with low interference with normal brain function but efficiently suppressing seizures ( 6 ). Further refinements of chemogenetics have jet got underway, which may use receptors detecting out-of-range extracellular elevations of the concentration of glutamate and, therefore, inhibiting neurons, preventing drug administration. Although attractive, this strategy will need further work to assess the risk of immunogenicity ( 6 ).

Non-viral Strategies

Some of the issues of viral vector-based gene therapy may be overcome by non-viral gene strategies, which provide advantages with regards to the safety profile, localized gene expression, and cost-effective manufacturing. Non-viral gene delivery systems are engineered complexes or NPs composed of the required nucleic acid (pDNA or RNAs) and other materials, such as cationic lipids, peptides, polysaccharides, and so on ( 70 ). These vectors have low production costs, can be topically administered, can carry large therapeutic genes, use expression vectors (such as plasmids) that are non-integrating, and do not elicit detectable immune response also after repeated administrations ( 29 , 70 ). Cationic lipid-based vectors are currently the most widely used non-viral gene carriers. Limitations may include low efficacy due to the poor stability and rapid clearance, or the possible generation of inflammatory or anti-inflammatory responses. Hence, cationic polymers, such as poly(L-lysine) (PLL) or modified variants (PEGylated PLL), constitute alternative non-viral DNA vectors that are attractive for their immense chemical diversity and their potential for functionalization ( 69 ).

Antisense Oligonucleotides Therapies

Oligonucleotides are unmodified or chemically modified single-stranded DNA sequences (of up to 25 nucleotides) that hybridize to specific complementary mRNAs. Once bound to targeted mRNAs, oligonucleotides can either promote RNA degradation or prevent the translational machinery through an occupancy-only mechanism, referred to as steric blockage . Anyhow, the process leading to protein formation is inhibited. Synthesizing antisense oligonucleotides (ASOs) must deal with making a structure that must be suitable for a stable and selective oligonucleotide/mRNA complex. Moreover, oligonucleotides are rapidly degraded by endo- and exonucleases and the mononucleotides products may be cytotoxic ( 29 , 78 ). Hence, the use of ASOs in clinical practice requires overcoming problems related to the design, bioavailability, and targeted delivery ( 78 ). To date, few in-human studies have been conducted that primarily addressed invariably progressive and fatal diseases such as PMEs ( 79 , 80 ). The authors proved the feasibility of the ASOs-based approach by specifically customizing oligonucleotides over the genetic defect of patients. This opens the way to N-of-1 trials, which will hopefully be the road of the next few years not only in oncology but also in epileptic patients ( 81 ).

Stem Cell Therapy

Recurrent seizures are associated with the loss of inhibitory GABAergic interneurons. Herby, the replacement of lost interneurons through grafting of GABAergic precursors might improve the inhibitory synaptic and reduce the occurrence of spontaneous seizures ( 6 ).

Currently, in a pioneering way, progenitors from the medial ganglionic eminence (MGE) derived either from fetal brains or, to avoid the need for immune suppression, from human induced pluripotent stem cells (hiPSCs) proved the most suitable for treating epilepsy, particularly with temporal lobe onset features. Medial ganglionic eminence cells show pervasive migration, differentiate into distinct subclasses of GABAergic interneurons, and efficiently get incorporated into the hippocampal circuitry improving inhibitory synaptic neurotransmission ( 82 , 83 ). An important point is that MGE progenitors from fetal brains hoist ethical issues, and it is also a challenge to obtain the adequate amount of cells required for clinical application ( 82 ). Consequently, the MGE progenitors derived from hiPSCs appear the most suitable donor cell type, as they do not raise ethical problems and are also compatible with patient-specific cell therapy in non-genetic epileptic conditions. However, it will be important to understand whether the suppression of spontaneous recurrent seizures is transient or enduring after the GABAergic progenitor cells grafting ( 82 ); moreover, it will be important to assess the safety profile of these hiPSCs, hence they may either exhibit genomic instability or cause undesired differentiation raising concerns for in human application ( 6 ). In conclusion, the results are exciting, but some points need to be addressed in the next years, before starting a true in human application.


A variety of drugs are being investigated for the treatment of epilepsy, many of whom target previously neglected pathophysiological pathways but demonstrate a favorable efficacy profile, together with low to mild grade AEs ( 15 ). Traditional ASMs, given alone or in a fair combination, are invariably the initial therapeutic approach; afterward, if drug resistance occurs, more than one underlying pathophysiological mechanism may likely contribute ( 14 ). Currently, uncontrolled epilepsy is often disabling, with patients experiencing increased comorbidity, psychological, and social dysfunction, combined with an increased risk of premature death. In younger patients, cognitive and neurodevelopmental impairments are severe consequences of recurrent spontaneous seizures, impacting the QoL and future independence ( 44 ). Accordingly, gaining a reduction of either the severity or frequency of seizures might have benefits ( 44 ) and hitherward new therapeutical strategies are in the pipeline.

Cannabidiol, FFA, and cenobamate have been shown to efficiently control seizures and are generally well-tolerated; particularly, an increase in the number of seizure-free days was observed with positive outcomes on the QoL of patients ( 16 ). Comparison of treatments such as VNS, DBS, and TNS are needed to decide which modality is the most effective; moreover, data collection on promising non-invasive neurostimulation modalities will allow getting a precise estimate of their therapeutic efficacy and long-term safety ( 30 ) ( Tables 1 , ​ ,2 2 ).

Advanced RCTs on new drugs for epilepsy treatment.

AEs, adverse events; ASMs, antiseizure medications; BDI, beck depression inventory; CaGI, caregiver global impression; CBD, cannabidiol; CSF, convulsive seizure frequency; d, day; drug-R, drug-resistant; DS, Dravet syndrome; LGS, Lennox-Gastaut syndrome; LINCL, late infantile neuronal ceroid lipofuscinoses; M, mean; n°, number; na, not assessed; OLE, open label extension; PAH, pulmonary arterial hypertension; PGIC, patient global impression of change; Pts, patients; RCT, randomized clinical trial; Ref, reference; SD, standard deviation; SF, seizure frequency; SUDEP, sudden unexpected death in epilepsy; STP, stiripentol; sz, seizures; VHD, valvular heart disease; w, weeks; y, years .

Advanced RCTs on new non-pharmacological treatments for epilepsy.

AEs, adverse events; ASMs, antiseizure medications; bpm, beats per minute; CBSDA, cardiac-based seizure detection algorithm; DRE, drug-resistant epilepsy; eTNS, external trigeminal nerve stimulation; FP, false positive; FU, follow-up; h, hours; iTC, ictal tachycardia; M, mean; n°, number; na, not assessed; Pts, patients; RCT, randomized clinical trial; Ref, reference; RNS, responsive neurostimulation; RR, retention rate; SF, seizure frequency; sGTC, secondarily generalized tonic-clonic; sz, seizures; VNS, vagal nerve stimulation; y, years .

Evidence on the role of neuroinflammation in epilepsy suggests that drugs that modulate specific inflammatory pathways could also be used to control seizures and improve neurological comorbidities, such as cognitive deficits and depression. Notably, many anti-inflammatory drugs are already available and could be repurposed in patients with epilepsy. Another mechanism likely involved in drug-resistant epilepsies is the undue expression of multidrug efflux transporters such as P-gps ( 52 ); however, the use of P-gps inhibitors in the clinical practice did prove disadvantageous for inseparable systemic toxicity ( 3 ). This arises the need to directly modulate not the transport but the expression of the P-gps ( 3 ). Finally, epilepsy represents a field suitable for the development of personalized approaches, requiring integration of clinical measures with both genomics and other -omics modalities ( 14 ).

Today epilepsy carries restrictions in the everyday life of the affected people, together with social burdens, and eventually high-level burdens for caregivers in EE. Hitherward, the continuous pursuit of the best treatment approach that nowadays, with the widening understanding of the pathophysiological basis of the epilepsies, is inevitably moving toward a “ precision ” approach. Gene hunting and new genes discovery proved essential in this way, but further support derives from functional in-vitro and in-vivo studies, i.e., in epileptic channelopathies it is crucial to understand whether the phenotype is caused by the loss- or gain-of-function mutations in the encoded protein through patch-clamp studies ( Figure 1 ). Likewise, if a novel gene is identified it is fundamental to understand through which mechanism it may cause the disease, consequently identifying the best treatment to reverse the functional defect. However, given a PM-based approach, this may not yet be enough, and a holistic evaluation of the patient involves the clinician to deeply know an own expected vulnerability to drugs through pharmacogenomics ; thus, avoiding potential AEs.

An external file that holds a picture, illustration, etc.
Object name is fneur-12-753753-g0001.jpg

Example for precision medicine in epileptic channelopathies. Toward N-of-1 trials. Created with BioRender.com . ASOs, antisense oligonucleotides; GoF, gain of function; LoF, loss of function.

Targeting the biological mechanism responsible for epilepsy could lead either to repurpose as ASMs and adjust dosages of drugs yet used in other fields of medicine (i.e., FFA, COX2 inhibitors, or inhibitors of P-gps) or even to develop outstanding treatments such as gene therapy. Great advances have been achieved in gene-based therapies, ranging from the development of new delivery material to the improved potency and stability of delivered nucleic acids. However, this field is still actually limited by the little understanding of exogenous-endogenous DNAs interaction and the invasive nature of some neurosurgical approaches. Moreover, targeted approaches (i.e., gene therapy, but also innovative drugs) currently carry high economic costs, which are covered by pharmaceutical industries during clinical trials but are hardly affordable for patients. In the new few years, the standardization of drug development, together with a larger use, and faster approval by regulatory agencies will probably make these treatments cheaper for patients.

The inflammatory pathways are common over epilepsies of different etiology and may therefore be reliable targets for treatment. However, targeting such complex and cross-interacting pathways of the human system may prove difficult, potentially altering basic life signals and causing a plethora of AEs further impacting the QoL of patients. Hence, also from this site, the next few years will be important to expand our knowledge and act consciously or even early, having fully comprised the red flags (biomarkers) of altered pathways through -omics studies.

Overall, research has changed our approach to epileptic patients, but PM is not always straightforward, and the pathophysiology of diseases may be more complex than what we can model , as different concomitant genetic variants, epigenetics, or the environment may modulate phenotypes in unintelligible and irreproducible ways. Moreover, nowadays patients are still often belatedly diagnosed raising the need to better define the way clinicians address phenotyping , which if incomplete could lead primarily toward the application of NGS epilepsy panels and then to whole-exome or genome sequencing, but invariably delaying diagnosis. Hence, also newer and standardized means of phenotyping will be needed, and wide opportunities in this are opened by the human phenotype ontology (HPO), a standardized vocabulary to describe phenotypic abnormalities. The hope will remain that of early diagnosis, early and non-invasive treatment to heal symptoms, improving the QoL of patients, and, in encephalopathies, improving the learning curve of patients.

Author Contributions

AR: conceptualization, writing-original draft, writing-review, and editing lead. AG: writing-original draft. GB: writing-review and editing support. EA, MV, GP, MI, SL, VS, and CM: writing-review and editing support. PS: conceptualization, funding acquisition, supervision, writing-review, and editing. All authors contributed to the article and approved the submitted version.

This work was developed within the framework of the DINOGMI Department of Excellence of MIUR 2018-2022 (legge 232 del 2016).

Conflict of Interest

AR has received honoraria from Kolfarma s.r.l and Proveca Pharma Ltd. PS has served on a scientific advisory board for the Italian Agency of the Drug (AIFA); has received honoraria from GW Pharma, Kolfarma s.r.l., Proveca Pharma Ltd., and Eisai Inc., and has received research support from the Italian Ministry of Health and Fondazione San Paolo. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Publisher's Note

All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article, or claim that may be made by its manufacturer, is not guaranteed or endorsed by the publisher.


The authors thank the Italian Ministry of Health Ricerca Corrente 2021.

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New tech gives hope for a million people with epilepsy

Jon Hamilton 2010

Jon Hamilton

Aaron Scott

Gabriel Spitzer, photographed for NPR, 6 June 2022, in Washington DC. Photo by Farrah Skeiky for NPR.

Gabriel Spitzer

new epilepsy research

The ROSA machine allows surgeons to more precisely target parts of the brain responsible for epileptic seizures. UC San Diego Health hide caption

The ROSA machine allows surgeons to more precisely target parts of the brain responsible for epileptic seizures.

Listen to Short Wave on Spotify , Apple Podcasts and Google Podcasts .

About three million people in the United States have epilepsy, including about a million who can't rely on medication to control their seizures.

For years, those patients had very limited options. Surgery can be effective, but also risky, and many patients were not considered to be candidates for surgery.

But now, in 2023, advancements in diagnosing and treating epilepsy are showing great promise for many patients, even those who had been told there was nothing that could be done.

One of those patients visited Dr. Jerry Shih at the Epilepsy Center at UC San Diego Neurological Institute, after getting a bleak prognosis a few years earlier.

"When I saw him, I said, 'You know what, we're in a unique situation now where we have some of the newer technologies that were not available in 2010." he says. "We knocked out that very active seizure focus. And he has subsequently been seizure free."

Using precise lasers, microelectronic arrays and robot surgeons, doctors and researchers have begun to think differently about epilepsy and its treatment.

"If you think about the brain like a musical instrument, the electrophysiology of the brain is the music." says Dr. Alexander Khalessi , a neurosurgeon at UCSD. "And so for so long, we were only looking at a picture of the violin, but now we're able to listen to the music a little bit better. And so that's going to help us understand the symphony that makes us us ."

Today on Short Wave, host Aaron Scott talks with NPR science correspondent Jon Hamilton about these advances in treating epilepsy. He explains why folks should ask their doctors about surgery — even if it wasn't an option for them a few years ago.

If you have a science question or idea for a show, we want to hear it. send us an email at [email protected] .

This episode was produced by Thomas Lu, edited by Gabriel Spitzer and fact checked by Anil Oza. The audio engineer for this episode was Hannah Gluvna.

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Epilepsy articles from across Nature Portfolio

Epilepsy refers to a group of neurological disorders of varying aetiology, characterized by recurrent brain dysfunction that result from sudden excessive and disordered neuronal discharge. These episodes can manifest as epileptic seizures, but they can also occur with subtle or no behavioural signs.

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Identification of four biotypes in temporal lobe epilepsy via machine learning on brain images

Brain imaging-based disease progression modelling is a promising technique for disease stratification. Here the authors characterize distinct ‘trajectories’ of brain atrophy in temporal lobe epilepsy and identify four subtypes with distinct neuroanatomical signatures.

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Epilepsy is a chronic, heterogeneous disease with an urgent need for novel therapies. Here, the authors show a systematic comparison of the global molecular signature of refractory epilepsies elucidating the key mechanisms of the disease pathology.

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Metabolic changes in status epilepticus.

Status epilepticus is associated with changes in metabolic pathways, a new study has shown.

Neurosteroids alleviate seizures in rats

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Post-zygotic brain mosaicism as a result of partial reversion of pre-zygotic aneuploidy

Brain somatic mosaicism is linked to several neurological disorders and is thought to arise post-zygotically. A study suggests that pre-zygotic aneuploidy followed by post-zygotic partial reversion leads to a recurrent form of brain mosaicism-related epilepsy.

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Seizure-associated changes in the Golgi apparatus

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Cardiovascular risk factors for epilepsy and dementia

A new study using the UK Biobank database has shown that people with epilepsy are at an increased risk of developing dementia. The results demonstrate that this risk is multiplied in individuals who also have high cardiovascular risk, highlighting the importance of addressing modifiable cardiovascular risk factors.

  • Michele Romoli
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New blood biomarker of refractory epilepsy

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New Epilepsy Research Findings: Long-Term Outcomes with Laser Ablation Surgery

Dr. Brett Youngerman and researchers from the Columbia Comprehensive Epilepsy Center have led the largest multicenter series to date of magnetic resonance-guided laser interstitial thermal therapy (MRgLITT) recently published in the Journal of Neurology, Neurosurgery, and Psychiatry .

On the impacts of this study, Dr. Youngerman stated, "In the largest multi-center study to date, we found that MRI-guided laser ablation is a viable minimally invasive surgical treatment with lasting seizure control for drug resistant temporal lobe epilepsy. Congratulations and thanks to all of the authors for their important contributions."

What is Interstitial Thermal Therapy AKA Laser Ablation?

For people with clearly localized seizure sites (foci) that may be difficult or risky to access with traditional surgery, Columbia Neurosurgery offer a minimally invasive techniques called laser interstitial thermal therapy (LITT- also known as laser ablation). Using computer-guided navigation, both of these techniques attempts to eliminate the seizure focus without making a large opening in the skull. Aided by MRI, our surgeons guide a laser through a three-millimeter incision and into the focus of a seizure to destroy the brain tissue causing seizures. Most people are able to go home the next day.

Details of the Study

MRgLITT is a minimally invasive alternative to surgical resection for drug-resistant mesial temporal lobe epilepsy (mTLE). The durability of seizure freedom after MRgLITT and outcomes after subsequent resection were previously largely unknown. The study found that among 268 consecutively treated patients at 11 centers, nearly half (49.3%) of patients had durable seizure freedom at last follow-up (median 47, range 12–95 months) and an additional 16.7% had rare disabling seizures. Preoperative focal to bilateral tonic-clonic seizures were independently associated with seizure recurrence. Among patients with seizure recurrence after MRgLITT, 14/21 (66.7%) became seizure-free after subsequent anterior temporal lobectomy (ATL) and 5/10 (50%) after repeat MRgLITT. The authors concluded that MRgLITT is a viable minimally invasive, first-line surgical treatment with durable outcomes for patients with drug-resistant mTLE evaluated at a comprehensive epilepsy center. Although seizure freedom rates were lower than reported with ATL, this series represents the early experience of each center and a heterogeneous cohort. ATL remains a safe and effective treatment for well-selected patients who fail MRgLITT. The study was co-authored by Matei Banu, MD, Farhan Khan, MD, and Guy McKhann , MD in the Department of Neurological Surgery and Catherine Schevon , MD, PhD in the Department of Neurology.

Please use the link to access the full paper via PubMed.

Related Information

Meet our team, brett evan youngerman, md, guy m. mckhann ii, md, epilepsy and seizures, temporal lobe epilepsy, laser ablation.

A New Hope for Patients With Epilepsy

March 24, 2021

Electroencephalogram, EEG, which can be used to detect seizures associated with epilepsy

Epilepsy, also known as a seizure disorder, is a condition that can seriously disrupt a patient’s everyday life. About 3.4 million people of all ages in the United States have this neurological disorder. But unlike with other brain-related conditions, about two dozen medications can successfully treat many cases of epilepsy . Although there is no cure, these anti-seizure drugs turn the disease into a chronic, but well-managed condition for many to the point where it barely interferes with life.  

But about one-third of patients aren’t so lucky. They experience no relief from anti-seizure drugs and are looking for additional treatment options.  

Now they may have found one in a new generation of neurostimulation devices used for epilepsy. In 2018, the Food and Drug Administration (FDA) approved a deep brain stimulation (DBS) device, manufactured by Medtronic, that sends electrical pulses through the brain to reduce the frequency of seizures. (It works by stimulating an important relay station deep in the brain called the thalamus.) And in June 2020, the FDA approved the Percept PC , also from Medtronic. Facilitating more customized therapy, this modified version allows doctors to treat epilepsy and record electrical activity from deep in the brain. (The Percept PC is also approved for other conditions, such as Parkinson’s disease and essential tremor.)  

Last year, the Yale Comprehensive Epilepsy Center became the first epilepsy center in the U.S. to implant the device in a patient with epilepsy. As of March 2021, eight epilepsy patients have been implanted with the Percept PC device at Yale.

What is epilepsy?

At the most basic level, everything we think, feel, and do is controlled by brain cells communicating with one another. They send messages through neurotransmitters like dopamine or via electrical pulses that travel through axons—long nerve fibers that connect the cells, or neurons, to each other.  

Normally, these electrical signals move between cells in a steady, consistent pattern. When a person has a seizure, however, the pattern is disrupted so that large groups of neurons send messages at the same time. This produces a flood of electrical activity that temporarily prevents areas of the brain responsible for language, memory, emotion, and consciousness from functioning.  

“Epilepsy is a sort of electrical activity that builds up over time before it is released and causes abnormality throughout the brain,” says Yale Medicine neurologist Imran Quraishi, MD, PhD .  

There are many different types of seizures, with a variety of causes. Focal (partial) epilepsy refers to seizures that start in one part of the brain. In some seizures, the electrical activity stays in that part of the brain and can cause a variety of symptoms including an inability to speak, “spacing out,” and memory lapses. In other seizures, the activity either spreads to or starts on both sides of the brain and can cause a person to pass out, faint, or even stop breathing.  

There are many treatment options for patients with focal epilepsy, including medications, surgical resection, laser ablation, dietary therapy, and neurostimulators. In some cases, however, neurostimulation is the only option that is effective, says Dr. Quraishi.

How deep brain stimulation can help with epilepsy

The 2018-approved DBS device uses electrical pulses to regulate the brain’s electrical activity. It’s similar to a pacemaker, which sends electrical signals to keep the heart beating normally.  

The device has two components: a neurostimulator, which is surgically implanted in a patient’s chest, and electrodes that are inserted into the brain. The neurostimulator releases electrical pulses through thin wires connected to electrodes that transfer the electricity to an area of the brain called the thalamus.  

“The thalamus connects a lot of areas of the brain,” says Lawrence Hirsch, MD , co-director of the Yale Comprehensive Epilepsy Center. “It’s a central networking place for electric signals in the brain. You can think of it like a big airport where flights pass through to connect to other places.” With DBS, electricity inside neurons in the path of an electrical pulse gets turned off. “This helps to make the brain less excitable or less likely to cause seizures,” he adds.

The Percept PC approved last June is Medtronic’s same DBS device, but with one significant difference—it records a patient’s brain signals.        

Our goal is to have an app connected to the device that tells patients they have, for example, a 90% chance of having a seizure that day. Yale Medicine neurologist Imran Quraishi, MD, PhD

Using information collected by the Percept PC, Dr. Hirsch explains, neurologists can more precisely adjust the device’s programming. “We may start the pulses at 30 seconds on and five minutes off during a 24-hour cycle and make changes from there,” he says.  

So far, the device is only approved for adults with focal epilepsy.

Research shows how the deep brain stimulation device improves symptoms

The FDA approved Medtronic’s 2018 DBS device for epilepsy based on data gathered from the SANTE (Stimulation of the Anterior Nucleus of the Thalamus in Epilepsy) clinical trial. This randomized double-blind study enrolled 110 adults who were experiencing an average of six seizures per month. In the first several months of the trial, half the patients had their device turned on and half did not; neither the patients nor the assessing physicians knew whether the device was on or off. Those with the device turned on had fewer seizures than those with the device off. The benefit continued to increase over time, says Dr. Hirsch.  

The study followed patients for seven years. In the first year after the device was implanted, 43% of participants with it had experienced half as many seizures as they’d been having when the study began. After seven years, researchers found that 74% of patients experienced the same reduction in seizure frequency—seizures were cut in half or better, says Dr. Hirsch—according to Medtronic. And in that same seven-year period, patients experienced a median of 75% fewer seizures from their baseline.

More data for more accurate predictions

Neurologists have been collecting patient data from the new Percept PC for several months, but it’s too soon to know if the new device will help patients more than the previous model.   

“We’re hoping that by relying on the patient’s seizure patterns from the Percept, we’ll be able to make adjustments more quickly and won’t have to wait years to get the maximum benefit from the device,” Dr. Hirsch says.   

You can think of it [the thalamus] like a big airport where flights pass through to connect to other places. Lawrence Hirsch, MD, co-director of the Yale Comprehensive Epilepsy Center

For his part, Dr. Quraishi hopes that when reams of data have been collected from enough people, neurologists may have better insight into how to more accurately predict a patient’s risk of a seizure. This could lead to a significant improvement in quality of life for people with treatment-resistant epilepsy, since being able to forecast one’s daily risk of a seizure would make everyday activities feel less risky.  

To give a sense of how this would work, Dr. Quraishi compares the device to common apps we all use to plan our activities. “Right now, before you go outside you check a weather app,” Dr. Quraishi says. “Our goal is to have an app connected to the device that tells patients they have, for example, a 90% chance of having a seizure that day.” If that’s the case, patients would know they might want to stay home and get extra rest, he adds.  

Or even better, says Dr. Hirsch, they may be able to take extra medications based on the prediction algorithm to prevent that seizure from even happening.

[ Read about what happens when the diagnosis isn't epilepsy, but "psychogenic non-epileptic seizure” or PNES. ]

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New treatment could transform the mental health of children with epilepsy

by University College London


A new psychological treatment for children with epilepsy, developed by a UCL-led team of scientists, has been shown to reduce mental health difficulties compared to standard care, a new study finds.

Mental health problems such as worries, low mood and behavior problems are more common in children and young people with brain conditions such as epilepsy, than in the general population—with up to 60% of those with epilepsy having associated mental health disorders and many having more than one mental health condition.

These conditions can have a big impact on patients' quality of life and overall health.

Currently, mental health problems in children and young people with epilepsy are often not identified because centers that treat epilepsy are usually separated from those that treat mental health difficulties. When mental health difficulties are identified, standard treatment for children who also have epilepsy is usually carried out by specialists, such as child and adolescent mental health services (CAMHS) or hospital-based pediatric psychology services. The treatment given usually involves treating each mental health condition (i.e., anxiety, depression, behavioral issues) individually.

The new treatment, named the Mental Health Intervention for Children with Epilepsy (MICE), is based on the treatments that the National Institute for Health and Care Excellence (NICE) recommends for the treatment of common mental health difficulties, like cognitive behavioral therapy for anxiety and depression. However, it uses a modular approach that enables multiple mental health conditions to be treated at once, instead of having different treatments for different mental health difficulties.

It was also modified specifically for children and young people with epilepsy, for example, including sessions that explain the relationship between epilepsy and mental health.

Additionally, the treatment can be delivered over the phone or via video call so that people do not have to travel to the hospital and miss time from school or work. And rather than being outsourced to services such as CAMHS, it is integrated into epilepsy services—meaning that it could be delivered by non-mental health specialists.

Lead author Dr. Sophie Bennett, who carried out the research while working at UCL Great Ormond Street Institute of Child Health, said, "This treatment breakthrough means that we have a new way to help children and young people with epilepsy who also have mental health difficulties .

"The treatment can be delivered from within epilepsy services to join up care. It doesn't need to be delivered by specialist mental health clinicians like psychologists. Integrating the care can help children with epilepsy and their families more effectively and efficiently. We were particularly pleased that benefits were sustained when treatment ended."

The new treatment, outlined in The Lancet , was created together with young people and their families and the professionals who care for them, including doctors, nurses and psychologists.

Patients were given an initial assessment followed by weekly calls with the clinician—although face-to-face therapy was available if preferred. The sessions were delivered to either the young person directly, or via their caregiver, based on their individual circumstances.

Researchers trialed the treatment with 334 children and young people aged three to 18. Of these, 166 received the new MICE treatment and 168 received the usual treatment for mental health problems in children with epilepsy.

They assessed adolescents' mental health and overall well-being from a parent-reported Strengths and Difficulties Questionnaire (SDQ)—covering areas such as emotional problems, conduct, hyperactivity and peer problems.

The results showed that the children who had the MICE treatment had fewer mental difficulties than those who had the usual treatment, and the change is equivalent to a decrease of 40% in the likelihood of having a psychiatric disorder.

Co-Chief Investigator, Professor Roz Shafran (UCL Great Ormond Street Institute of Child Health and GOSH), said, "These groundbreaking findings not only promise brighter futures for children with epilepsy, but also pave the way for a revolutionary shift in mental health care practices. The collaborative efforts of scientists, patients, and health care professionals have brought forth a new era of treatment of mental health challenges associated with epilepsy, offering a beacon of hope for families in the face of mental health challenges associated with epilepsy."

Co-Chief Investigator, Professor Helen Cross (UCL Great Ormond Street Institute of Child Health and GOSH), added, "This study shows real progress for clinicians considering the high rate of mental health problems in children with epilepsy, as we demonstrate the benefit of a therapy that can be implemented within existing epilepsy services."

Co-author, Professor Isobel Heyman (UCL Great Ormond Street Institute of Child Health and Clinical Co-Lead for mental health at Cambridge Children's Hospital), noted, "These promising results show that staff working in pediatric settings can be trained to deliver effective mental health treatment to children with a physical health condition ( epilepsy ). It clearly demonstrates that children's health care needs can be met in a holistic way to treat the 'whole child," in the same place at the same time."

The work was conducted in collaboration with experts at Great Ormond Street Children's Hospital (GOSH), King's College London and UCLA.

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Epilepsy Research News: August 2021

This month, we share a recent study in which researchers have discovered the potential underlying mechanism of a rare genetic mutation that can cause epilepsy. We also highlight the development of a new ‘tool’ to control neuronal activity using an already-existing technique called optogenetics that harnesses the power of light to understand seizures.

In other news, we highlight a study in which researchers have discovered a previously unknown repair process in the brain that they hope can be harnessed and enhanced to treat seizure-related brain injuries.

Lastly, we report on the first clinical trial for a new dietary treatment for children and adults with severe forms of epilepsy. This study evaluated the use of K.Vita® (also known as Betashot), an oral liquid dietary supplement developed to reduce the adverse side effects caused by the ketogenic diet.

Summaries of these articles and more can be found below.

Understanding Rare Genetic Epilepsy : Researchers have discovered the potential underlying mechanism of a rare genetic mutation referred to as S1459G that can cause epilepsy. Using an artificial neuronal preparation, the team studied protein structures, called receptors, that are attached to cell surfaces and found that this particular mutation causes receptors in brain cells to behave differently, resulting in an imbalance in brain cell communication that could lead to disorders including epilepsy. The researchers are hopeful that understanding the way this mutation might lead to epilepsy will provide a springboard for developing personalized or precision medicines to target this mutation. Learn more

New Tool to Understand Seizures : Scientists have discovered a new ‘tool’ to control neuronal activity using light and genetic engineering which is based on an already-existing technique known as optogenetics. This tool is a protein called Opn7b that is turned off when blue or green light is shone on it. When Opn7b is deactivated by light, the researchers showed that it triggers seizures in animals. This is unlike typical optogenetic proteins that are turned on by light, a process that the researchers note may be more time-consuming and less reliable. The researchers hope it will be possible to use this optogenetic tool to better understand the underlying mechanisms and timeline of the development of epileptic seizures. Learn more

Brain Repair after Seizures:  Researchers have discovered a previously unknown repair process in the brain that they hope can be harnessed and enhanced to treat seizure-related brain injuries. They used an advanced imaging technique known as two-photon microscopy to examine what happened in the brains of laboratory mice after severe seizures. The researchers saw that immune cells called microglia were not just removing damaged tissue after seizures but actually appeared to be healing damaged neurons. Though the researchers note that more work must be done to understand the role of microglia in seizures, they also state that this research may help provide a pathway for developing approaches to enhance this process and treat epilepsy. Learn more

Dietary Treatment for Epilepsy : The first clinical trial of a new dietary treatment, based on the ketogenic diet, for children and adults with severe forms of epilepsy has been completed. For the study, clinicians evaluated the use of K.Vita® (also known as Betashot), an oral liquid dietary supplement, developed to reduce the adverse side effects caused by the ketogenic diet. Though this was a trial that was primarily designed to test side effects of the K.Vita® treatment, researchers also found that there was a 50% reduction in seizures. The researchers state that the study provides early evidence of the effectiveness of this treatment for severe drug-resistant epilepsies. Learn more

Breath Test to Improve Epilepsy Treatment Success : Researchers have developed a new method to measure drug concentrations in the breath of epilepsy patients. They hope that this new approach will enable doctors to react more precisely when treating the disease. The advantage is that this test does not require a blood sample that would need to be sent to the laboratory; the results are available immediately. The goal is to use the results to determine whether the active substances in the drug treatment are present at the right concentrations in the body and whether they have the desired effect on the disease. Learn more

Citizens United for Research in Epilepsy does not recommend or endorse any specific tests, therapies, physicians, products, procedures, or opinions reported in the news section of this website. News is presented for informational purposes only. The purpose of epilepsy research news is to increase awareness of epilepsy research. It is not intended to be a substitute for professional medical advice, diagnosis or treatment. Always seek the advice of your physician or other qualified health care provider with any questions you may have regarding a medical condition or treatment and before undertaking a new health care regimen, and never disregard professional medical advice or delay in seeking it because of something you have read at CureEpilepsy.org

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Death rate higher than expected for patients with functional, nonepileptic seizures.

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Curing the Epilepsies: The Promise of Research

Request free mailed brochure

For individuals living with a treatment-resistant form of the seizure disorders known as “the epilepsies,” each day can bring debilitating seizures as well as challenges from co-occurring conditions that dramatically impair quality of life. This brochure, for people with the disorders, their caregivers, and advocates, outlines the state of progress made in the past decade toward achieving the Epilepsy Research Benchmarks, a set of widely recognized objectives aimed at prioritizing the efforts of scientists and health care providers to find cures for the epilepsies. Much of this work has been funded by grants from the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH).


At least 2.3 million adults and nearly 500,000 children in the U.S. currently live with some form of epilepsy, a disorder in which clusters of nerve cells, or neurons, in the brain sometimes signal abnormally, causing seizures. Each year, another 150,000 people are diagnosed with epilepsy. The disorders affect both males and females and can develop at any age. In the U.S. alone, the annual costs associated with the epilepsies are estimated to be $15.5 billion in direct medical expenses and lost or reduced earnings and productivity.

The disturbances of neuronal activity that occur during seizures may cause strange sensations, emotions, and behaviors. They also sometimes cause convulsions, abnormal movements, and loss of consciousness. In some people, seizures happen only occasionally. Other people may experience hundreds of seizures a day. There are many different forms of epilepsy, and symptoms vary greatly from one person to another. Recent adoption of the term “the epilepsies” underscores the diversity of types and causes.

About three-quarters of the individuals diagnosed with the epilepsies can control their seizures with medicine or surgery. However, about 25 to 30 percent will continue to experience seizures even with the best available treatment. Doctors call this treatment-resistant epilepsy. In some cases, people experience a type of seizure called status epilepticus, defined as seizures that last for more than five minutes or seizures that recur without recovery of consciousness. Prolonged status epilepticus can damage the brain and may be life-threatening.

Section 1: About Epilepsies

Seizures can be classified as focal or generalized. Focal seizures begin in one area of the brain and may or may not spread to other areas. Generalized seizures are the result of abnormal neuronal (nerve cell) activity on both sides of the brain from the beginning of the seizure. 

About 60 percent of people with epilepsy have focal seizures. Some focal seizures cause unusual sensations, feelings, or movements, but do not cause loss of consciousness. Other focal seizures cause a change in or loss of consciousness and may produce a dreamlike experience or strange, repetitive behavior. Focal seizures are often named for the area of the brain in which they originate. For example, temporal lobe epilepsy, or TLE, begins in the temporal lobe located on either side of the brain. TLE is the most common type of epilepsy to feature focal seizures and can sometimes be difficult to treat with available medications. 

Generalized seizures impair consciousness and distort electrical activity of the whole or a large portion of the brain, leading to falls or abnormal movements. There are several different types of generalized seizures. In absence seizures, which usually begin in childhood or adolescence, an individual may appear to be staring into space or may have jerking or twitching muscles. Tonic seizures cause stiffening of muscles. Clonic seizures cause repeated jerking movements of muscles on both sides of the body. Myoclonic seizures cause jerks or twitches of the upper body, arms, or legs. Atonic seizures cause a loss of normal muscle tone, which may lead to falls or sudden drops of the head. Tonic-clonic seizures cause a combination of symptoms, including stiffening of the body and repeated jerks of the arms or legs as well as loss of consciousness.

Epilepsies with Childhood Onset 

Compared to adults, infants and children have a relatively high risk of developing the epilepsies. Many epilepsy syndromes, such as infantile spasms, Lennox-Gastaut syndrome, and Rasmussen’s encephalitis, begin in childhood. Infantile spasms usually begin before the age of six months and may cause a baby to bend forward and stiffen. Children with Lennox-Gastaut syndrome have severe epilepsy with several different types of seizures, including atonic seizures, which cause sudden falls called drop attacks. Rasmussen’s encephalitis is a rare, chronic inflammatory neurological disease that usually affects only one hemisphere of the brain. It causes frequent and severe seizures and a loss of motor skills, and can lead to severe intellectual disability. Hypothalamic hamartomas can cause another rare form of epilepsy that presents during childhood and is associated with malformations in the hypothalamus at the base of the brain. People with hypothalamic harmatomas have seizures that can resemble laughing (gelastic) or crying (dacrystic). Such seizures frequently go unrecognized or are difficult to diagnose. 

Some childhood epilepsy syndromes, such as childhood absence epilepsy, may go into remission or stop entirely as a child matures, although this is not true in all cases. Other syndromes, such as juvenile myoclonic epilepsy and Lennox-Gastaut syndrome, are usually present for the rest of the person’s life. 

Children with delayed brain development and neurological disorders are more likely to have seizures. Seizures are more common, for example, among children with autism spectrum disorder, cerebral palsy, tuberous sclerosis complex (TSC), or Rett, Aicardi, or Down syndromes. In one study, one-third of children with autism spectrum disorder had treatmentresistant epilepsy.

For about half of all people with an epilepsy, a cause for the disorder is not identified. In other cases, the epilepsies are clearly linked to genetic factors, infection, head trauma, stroke, brain tumors, or other identifiable problems. 

The epilepsies have many possible causes. Almost anything that disturbs the normal pattern of brain circuit activity—from abnormal brain development to traumatic brain injury (TBI) or illness—can lead to seizures and epilepsy. For example, seizures may develop because of an abnormality in brain wiring that occurs during brain development, an imbalance of neuronsignaling chemicals called neurotransmitters, or a combination of these factors. Researchers believe that some people with epilepsies have abnormally high levels of responsiveness to excitatory neurotransmitters, chemicals that increase the activity of nerve cells. Other people may have an abnormally low level of responsiveness to neurotransmitters that inhibit nerve cell activity. Either situation can result in too much neuronal activity and cause epilepsy. In some cases, inflammation and neuronal damage after a head injury, stroke, or other trauma may lead to epilepsy. In addition, the brain’s attempts to repair itself after such injury may inadvertently generate abnormal nerve connections that lead to seizures. Supportive cells (known as glial cells) in the brain may play a role in certain types of epilepsy. 

Sidebar: The Role of the National Institutes of Health

The U.S. Federal government supports research to better understand the epilepsies and to reduce their burden through improved treatments and prevention. Much of this research support comes from the National Institutes of Health (NIH). The National Institute of Neurological Disorders and Stroke (NINDS) is the lead NIH institute for research on the epilepsies. Several other NIH Institutes also fund epilepsy-related research. Representatives from NIH institutes, the Centers for Disease Control and Prevention (CDC), the Department of Defense, the Department of Veterans Affairs, and the U.S. Food and Drug Administration (FDA) work together as part of the Interagency Collaborative to Advance Research in Epilepsy (ICARE), which was formed to facilitate communication and opportunities for coordination among institutes and agencies sponsoring research related to the epilepsies.

Section 2: Research Progress on the Epilepsies

In 2000, NINDS and epilepsy research and advocacy organizations co-sponsored a White House initiated conference, “Curing Epilepsy: Focus on the Future.” The conference has been viewed as a turning point for research on the epilepsies by shifting the focus from treating seizures to identifying cures, defined as “no seizures, no side effects, and the prevention of epilepsy in those at risk.” The first Epilepsy Research Benchmarks grew out of the momentum created by this conference, as a way to communicate and address important research priorities and as a framework for periodically “benchmarking” progress. A second conference in 2007, “Curing Epilepsy: Translating Discoveries into Therapies,” reassessed the state of research on the epilepsies and revised the Epilepsy Research Benchmarks, adding emphasis to the conditions that co-occur with the epilepsies and sudden unexpected death in epilepsy (SUDEP). A third conference in 2013, “Curing the Epilepsies: Pathways Forward,” provided an update on the state of research and will result in another revision of the Benchmarks. 

Today, more than a decade since they were first developed, the Benchmarks are increasingly embraced by the entire epilepsy community, including NIH , researchers, and professional and advocacy organizations. While the ultimate goal of curing the epilepsies has not yet been achieved, researchers have made substantial progress. Research on the epilepsies has yielded exciting advances across all areas of the Benchmarks. 

What New Discoveries Have Been Made About the Causes of the Epilepsies?

To understand how to prevent, treat, and cure the epilepsies, researchers first must learn how they develop. Where, how, when, and why do neurons begin to display the abnormal firing patterns that cause epileptic seizures? This process, known as epileptogenesis, is the key to understanding the epilepsies. Researchers are learning more about the fundamental processes—known as mechanisms—that lead to epileptogenesis. The discovery of each new mechanism involved in epileptogenesis has the potential to yield new targets that can be affected by medications or other therapies to block that mechanism. Among this growing list of candidate mechanisms, two stand out as being closest to yielding potential targets for drug therapy: 1) the mTOR (mammalian target of rapamycin) signal transduction pathway; and 2) activation of the cytokine protein interleukin-1ß (IL-1ß).

  • The mTOR pathway is a master regulator that is involved in several genetic and acquired forms of epilepsy. An inhibitor of the mTOR pathway is being studied for the prevention of seizures related to tuberous sclerosis complex (TSC), a rare genetic disease that causes the growth of noncancerous tumors in the brain and in other organs such as the kidney, heart, eyes, lungs, and skin.
  • In various types of epilepsy, inflammatory processes may play a key role. Cytokines are signaling molecules that, among other functions, help regulate the body’s inflammatory responses. Researchers are exploring why IL-1ß appears to be activated in different types of epilepsy. An inhibitor of IL-1ß synthesis is being tested in people with treatment-resistant epilepsy.

Other Areas of Epileptogenesis Research Include:

  • Proteins in the cell membrane are crucial for generating the electrical impulses that neurons use to communicate with one another. For this reason, researchers are studying the membrane structure and the channels that allow molecules like sodium, calcium, and potassium to move across them to generate electrical impulses. A disruption in any of these processes can cause changes that may lead to epilepsy.
  • Studies have suggested how a breakdown of the blood-brain barrier may lead to seizures. When proteins from the blood cross this important barrier between the circulatory system and fluid surrounding the brain, they trigger a reaction that leads to hyperactivity of neurons in the area of the brain surrounding the breakdown.
  • Glial cells are non-neuronal cells that play a critical supportive role in the brain. For example, astrocytes are a type of glial cell that acts as a “housekeeper” by removing excessive levels of glutamate, a major neurotransmitter that mediates excitatory signals in the central nervous system. When astrocytes are impaired, levels of glutamate rise excessively in the spaces between brain cells, which may then contribute to the onset of seizures. In animal studies, the introduction of ceftriaxone, an antibiotic that supports the housekeeping role of astrocytes, has been shown to reduce seizure frequency.
  • The body’s immune system may contribute to the development of certain forms of epilepsy. In aggressive forms of the disorders, antibodies may impair the function of brain receptors, leading to abnormal neuronal activity. Testing for many of these antibodies is already available, and findings from early-stage clinical trials suggest that strategies aimed at adjusting the body’s immune system may provide a means of treating these otherwise untreatable forms of epilepsy.

Researchers are studying the membrane structure and the channels that allow molecules like sodium, calcium, and potassium to move across them to generate electrical impulses. A disruption in any of these processes can cause changes that may lead to epilepsy.

Genetic Mutations

Recent studies have yielded substantial progress in the identification of genetic mutations involved in the epilepsies. Several types of epilepsy have been linked to defective genes for ion channels, the “gates” that control the flow of ions in and out of cells and that regulate neuronal activity.

Mutations in single genes have been found among family members affected by certain epilepsy syndromes. For example, some infants with Dravet syndrome, a type of epilepsy associated with seizures that begin before the age of one year, carry a mutation in the SCN1A gene that is believed to cause seizures by affecting sodium channels in the brain. Surprisingly, the SCN1A mutations and other epilepsy mutations are often de novo mutations, meaning that they are not present in the parents. Building on that genetic discovery, researchers have created models of Dravet syndrome in the fruit fly, zebrafish, and mouse that are now being used to test potential therapies for controlling seizures. In addition, researchers have successfully taken connective tissue cells from individuals with Dravet syndrome, reprogrammed them to create induced pluripotent stem cells (cells that can become any type of cell in the body), and differentiated them into neurons that also can be used to test potential drugs and to study the mechanisms that lead to Dravet syndrome.

Continued progress in the identification of genetic causes of the epilepsies could guide the care and medical management of individuals. In the case of heritable mutations, this will help affected families understand their risks. 

A major driver of success on the genetics front is the advent of next-generation sequencing—highthroughput methods of genetic sequencing that have revolutionized the search for the genetic underpinnings of diseases and disorders. Next-generation sequencing has significantly cut the time and costs required to identify genes involved with the epilepsies, as well as other diseases. 

Major collaborative efforts have enabled researchers to efficiently investigate the effects of many risk factors, including genetic ones, among large populations of people affected by the epilepsies.

Researchers have taken skin cells from people with Dravet syndrome, turned them into stem cells, and then turned those stem cells into brain cells. Electrical activity among the brain cells can be measured, creating a testing ground for studying the disorder.

Identify Biomarkers of Seizure Onset and Epileptogenesis

Researchers see a potential opportunity to prevent the epilepsies before the onset of recurrent spontaneous seizures. Surrogate measures of epileptogenic processes, or biomarkers, could aid in the development of interventions that would prevent epilepsy in at-risk individuals. Other types of biomarkers could help researchers and health care providers better identify and monitor seizure-onset zones or predict seizure occurrence, which could enable more targeted treatments. The identification of reliable biomarkers for the epilepsies is one of the more critical areas in need of research advances.

A number of changes in the brain shown on imaging and electroencephalography (EEG) are known to be associated with epilepsy-related processes. The challenge is that people without epilepsy also can have similar brain changes and there is little evidence to show clearly which of these changes is predictive of someone who will develop a form of epilepsy. 

Newer technologies are allowing researchers to map epileptic networks and track seizure generation with increasing resolution. Implantable “microelectrodes” are revealing complex brain activity during seizures. Using microelectrodes, researchers are able to better characterize high-frequency oscillations (HFOs). Abnormal HFOs have been linked to seizureonset zones and may serve as a biomarker of epileptogenesis; this could help identify people at risk for developing epilepsy after an initial insult to the brain, such as a stroke or TBI. 

Investigators also have improved devices for measuring electrical activity in the brain. New electrode arrays are flexible enough to mold to the brain’s complex surface, providing unprecedented access for recording and stimulating brain activity. While these arrays have not yet been used in humans, they are a promising advance toward expanded options for epilepsy diagnosis and treatment. 

Epilepsy researchers have increasingly explored how connections between different brain regions—structurally and functionally—may explain how seizures start in the first place. Much of this research grows out of observations that seizures are not merely the result of focal areas of hyperactivity, but arise from the complex interactions of the network. A better understanding of how this network operates may explain, for example, why some people do not improve even after focal areas of hyperactivity, which appeared to be the source of seizure, are surgically removed.

Diffusion tensor imaging, a type of magnetic resonance imaging (MRI) that shows microstructural detail of tissues based on the diffusion of water molecules, has shown abnormal structural connectivity during focal and generalized seizures. Advances in MRI have shown that functional connectivity patterns in people with epilepsy differ from those of normal controls. Interestingly, patterns of abnormal functioning occur both during seizures and during the “resting-state” period between seizures.

Newer technologies are allowing researchers to map epileptic networks and track seizure generation with increasing resolution. Implantable “microelectrodes” are revealing complex brainactivity during seizures. Using microelectrodes, researchers are able to better characterize high-frequency oscillations.

Develop New Animal Models for Studying Epileptogenesis and for Testing Treatments

The diversity of epilepsy syndromes and their causes precludes investigators from using any single animal model system for learning about the epilepsies and for testing potential therapies. Multiple syndrome-specific models are therefore needed to advance research on the epilepsies. 

Several substantial advances in the development of animal models have occurred over the last few years, including new models of Dravet syndrome, infantile spasms, cortical dysplasia, and viral encephalitis, as well as for stroke, TBI, and other conditions that can lead to acquired forms of epilepsy.

The zebrafish has emerged as a promising model for screening new drug compounds for antiseizure activity. Fish that are bred to express mutations known to be associated with particular types of epilepsy can be quickly and cost-effectively produced for research. The drosophila, or fruit fly, is another model developed to investigate the cellular mechanisms of the epilepsies.

Paramedics stopped status epilepticus seizures earlier thanks to drug delivery with an autoinjector. Similar to the EpiPen used by people with serious allergic reactions, the autoinjector may someday be on hand for people with epilepsy and their families.

Develop New Treatment Strategies and Optimize Existing Treatments

There have been several key advances in diagnostics, therapeutics, and technologies that are either approved or in various stages of approval in the U.S. and Europe. New chemical entities have been developed for treatment-resistant epilepsy. For example, ezogabine (also called retigabine) was approved by the U.S. Food and Drug Administration (FDA) in 2011 for the prevention of focal seizures by a novel mechanism of action. Several other potential drugs and chemical compounds (brivaracetam, perampanel, YKP3089, VX-765) are in development and also are aimed at  preventing seizures by novel mechanisms.

In addition, several agents have been approved for specific seizure types or syndromes: rufinamide (Lennox-Gastaut syndrome), stiripentol (Dravet syndrome), adrenocorticotropic hormone also known as ACTH (infantile spasms), and vigabatrin (infantile spasms). mTOR inhibitors (such as everolimus) are being tested for the treatment of seizures and other manifestations of tuberous sclerosis complex (TSC). The drug everolimus has been approved by the FDA for preventing the growth of tumors in individuals with TSC. 

Progress has been made in determining the best single-agent therapy for childhood absence epilepsy (CAE), the most common pediatric epilepsy syndrome, occurring in 10 to 17 percent of all children with epilepsy. People with CAE tend to have several seizures each day. In the interest of finding a drug regimen that would limit an individual’s exposure to drug-related side effects, researchers compared ethosuximide, lamotrigine, and valproic acid to treat CAE. Ethosuximide was found to be the best single-agent therapy because of its optimal balance between effectiveness and relatively few side effects.

NINDS-funded researchers have made significant strides in improving the management of individuals with status epilepticus seizures. These prolonged seizures can be particularly challenging to treat given the difficulty of establishing an intravenous line (IV) when a person is having convulsions. The results of the randomized controlled trial, known as Rapid Anticonvulsant Medication Prior to Arrival Trial (RAMPART), showed that seizures stopped significantly earlier in people treated with midazolam delivered by an autoinjector compared to individuals treated with lorazepam by IV. The autoinjector is similar to the EpiPen drug delivery system used to treat serious drug reactions. Faster resolution of seizures also translated into fewer people requiring hospitalization. 

Ongoing basic research efforts continue to identify targets for therapy development. For example, studies have focused on the role of gamma-aminobutyric acid (GABA), a key neurotransmitter that inhibits activity in the central nervous system. Other studies are investigating ways of blocking the activity of the excitatory neurotransmitter glutamate.

Given that the epilepsies involve so many different underlying mechanisms, the development of a single therapy is unlikely. Instead, management approaches will need to be tailored for specific syndromes.

Sidebar: Anticonvulsant Screening Program

In 1975, NINDS established the Anticonvulsant Screening Program (ASP) to promote the development and evaluation of new antiseizure drugs. At the time, few incentives existed for the pharmaceutical industry to support epilepsy research on the development of therapeutic agents. Since its launch, the ASP has been instrumental in bringing new antiseizure medications to the marketplace by giving researchers a common platform for submitting potential therapeutic agents to standardized testing in animal models. The resources provided to researchers through the ASP can save years in development time. 

ASP’s priorities have evolved to focus on the development of therapies aimed at preventing epileptogenesis, modifying the course of disease progression, finding therapies for the cases that do not respond to currently available treatments, and identifying specific epilepsy subtypes and their unmet needs. The ASP maintains a database of more than 30,000 submitted compounds, and plans are underway to improve the usefulness of the data for researchers pursuing novel compounds. NINDS continues to look for new ways to improve the ASP. New assays and procedures are being developed and implemented to significantly expand the sensitivity of the traditional screening approach to identify novel pharmacotherapies targeting the major unmet medical needs in epilepsy.

Surgery remains an effective option for individuals with treatment-resistant epilepsies. The most common type of surgery involves the removal of a seizure focus, the small area of the brain where seizures originate. In some extremely severe cases, surgeons perform a procedure called multiple subpial transection, which involves making cuts designed to prevent seizures from spreading into other parts of the brain while leaving the person’s normal abilities intact. Doctors also may use surgical procedures called corpus callosotomy (severing of the nerve fibers that connect the two sides of the brain) or hemispherectomy (removal of half of the brain). Researchers continue to refine surgical techniques to make them less invasive and to prevent cognitive and other neurological deficits that can result from surgery.

  • New imaging technologies are key advances for localizing the effects of surgery and minimizing adverse events. Many epilepsy centers have begun to use functional magnetic resonance imaging (fMRI) to “map” language and memory zones prior to surgery. NIH-funded researchers are aiming to verify whether fMRI actually improves surgical outcomes and to standardize best practices for its use.
  • Researchers also are looking for ways to combine imaging modalities to more accurately map language zones. In one study, for example, diffusion tensor imaging (DTI) is being used along with fMRI and magnetoencephalography (MEG), another brain mapping technique based on magnetic fields, to evaluate preoperative language processing and preserve key language zones during surgery for temporal lobe epilepsy.
  • Evidence suggests that high-frequency oscillations (HFOs) measured in the neocortex and temporal lobe may be biomarkers of epileptic networks, and can therefore help in surgical mapping and predicting outcomes after epilepsy surgery. Retrospective studies show that the removal of zones generating HFOs is associated with improved results following surgery.
  • Minimally invasive MRI-guided laser surgery is being studied for the treatment of epilepsies associated with tumors, such as hypothalamic hamartomas and tuberous sclerosis complex. The technique involves drilling a very small hole in the skull through which a thermal laser is inserted to ablate an epileptogenic zone under MRI-guidance.

New imaging technologies are key advances for localizing the effects of surgery and minimizing adverse events. Many epilepsy centers have begun to use functional magnetic resonance imaging to “map” language and memory zones prior to surgery.

Brain Stimulation

Electrical stimulation of the brain remains a therapeutic strategy of interest. The types of stimulation include: deep brain, intracranial cortical, peripheral nerve, vagal nerve, and trigeminal nerve. So far, deep brain stimulation has involved either the thalamus or the hippocampus, and only thalamic stimulation has been tested in a large clinical trial. 

A clinical trial of deep brain stimulation in the anterior thalamic nucleus showed significant seizure reduction over the long term, and the majority of participants saw benefit. Thalamic stimulation has been cleared for use in Europe, but not in the U.S. 

A report on trigeminal nerve stimulation showed efficacy rates similar to those for vagal nerve stimulation, with about half of the people responding (a responder is defined as having greater than a 50 percent reduction in seizure frequency). Freedom from seizures, although reported, remains rare for both methods. 

NINDS-supported investigators are developing methods to predict seizures by analyzing brain activity patterns that precede their onset. A promising application of this research is the development of implantable devices that can detect a forthcoming seizure. Once detected, the implanted device administers an intervention, such as electrical stimulation or a fast-acting drug to prevent the seizure from occurring. The first generation of seizure control devices in clinical trials uses such seizure prediction technology. The NeuroPace RNS system is among these devices, known as responsive stimulation or closed-loop devices. 

Optogenetics is an emerging experimental technique that may eventually lead to future generations of closed-loop devices. It involves the genetic delivery of light-sensitive proteins to specific populations of brain cells. The lightsensitive proteins can be inhibited or stimulated by exposure to light delivered by fiber optics. Although optogenetic methods are not currently used in humans, such an approach could allow highly targeted regulation of network excitability, providing a means for intervening at or before the onset of a seizure. 

A high-fat, very low carbohydrate ketogenic diet is an age-old treatment for medicationresistant epilepsies and there has been a renewed interest in recent years in how it works. The diet effectively reduces seizures for some people, especially children with certain forms of epilepsy. Studies have shown that more than 50 percent of people who try the ketogenic diet have a greater than 50 percent improvement in seizure control and 10 percent experience seizure freedom. However, for some people, the regimen is difficult to maintain. 

Researchers are trying to learn exactly how the ketogenic diet prevents seizures. They hope to find ways to mimic its seizure-blocking effects without the dietary restrictions. Studies have advanced the understanding of the connection between  energy metabolism and neuronal excitability, and in the process and may contribute to a better understanding of how the ketogenic diet promotes seizure control. 

In addition, researchers are looking at modified versions of and alternatives to the ketogenic diet. For example, studies show promising results for a modified Atkins diet and for a low-glycemicindex diet, both of which are less restrictive and easier to follow than the ketogenic diet. However, well-controlled randomized controlled trials have yet to assess the approaches, and many questions remain about the optimal circumstances of their use. 

Researchers are trying to learn exactly how the ketogenic diet prevents seizures. They hope to find ways to mimic its seizure-blocking effects without the dietary restrictions. Studies have advanced the understanding of the connection between energy metabolism and neuronal excitability..

Gene and Cell Therapies

The discovery of genetic mutations that are linked to specific epilepsy syndromes suggests the possibility of using gene-directed therapies to counter the effects of these mutations. Gene therapies remain the subject of many studies in animal models of epilepsy, and the number of potential approaches continues to expand. A common approach in gene therapy research, called transfection, uses modified components of viruses to introduce new genes into brain cells, which then act as “factories” to produce potentially therapeutic proteins.

Several proteins have been targeted for transfection. Animal studies have shown that it is possible to introduce a new protein into a cell, and in some cases, there has been an associated reduction in the frequency, duration, and severity of seizures.

Cell therapy differs from gene therapy in that instead of introducing genetic material, it involves the transplantation of whole cells into a brain. In animal studies, for example, NINDS-funded researchers have successfully controlled seizures in mice by grafting special types of neurons that produce the inhibitory neurotransmitter GABA into the hippocampus of their brains.

Gene and cell therapies remain attractive and promising strategies for treating, and potentially curing, some forms of epilepsy. However, their advancement as a viable treatment option in people will require new technologies and methods that can target specific neurons in the brain. These approaches need to be able to create more long-lasting changes.

Viruses are introduced into brain cells, which then act as “factories” to produce potentially therapeutic proteins.

Preventing the Development of the Epilepsies

Until recently, therapy development for the epilepsies focused largely on treating seizures in people already affected by the disorders. Now, in addition to efforts to develop new and improved antiseizure treatments, researchers are striving to prevent the epilepsies among people at risk. 

Measures that reduce the risk of head injury and trauma—such as improvements in automobile safety and the use of seat belts and bicycle helmets—can prevent epilepsies related to TBI. Good prenatal care, including treatment of high blood pressure and infections during pregnancy, can prevent brain damage in developing babies that may lead to epilepsy and other neurological problems later in life. Treating cardiovascular disease, high blood pressure, infections, and other disorders that affect the brain during adulthood and aging also may prevent some types of epilepsy. 

However, while such measures can prevent brain damage from occurring in the first place, there are currently no interventions known to specifically reduce the risk of seizure onset once damage to the brain has occurred. None of the available antiseizure medications have been shown to modify the development of the epilepsies in people. Researchers are working to change this.

  • Recent animal studies have helped clarify the mechanisms of hypoxic-ischemic encephalopathy (HIE) seizures (caused by a lack of oxygen in the brain), and clinical studies involving newborns have begun to assess potential treatment strategies. These include drugs both alone and in combination with each other or in combination with a strategy that involves deliberately cooling babies with HIE for the prevention of epilepsy.
  • Adenosine is an inhibitory neuromodulator that is believed to promote sleep and suppress arousal. Studies in animal models have shown that increasing adenosine levels in the brain can inhibit the development of spontaneous recurrent seizures after an initial injury.

Section 3: Reducing the Risk of Conditions that Co-occur with the Epilepsies

  psychiatric, neurodevelopmental,and sleep disorders.

Co-occurring psychiatric conditions are relatively common in individuals with epilepsy. In adults, depression and anxiety disorders are the two most frequent psychiatric diagnoses. Attention Deficit Hyperactivity Disorder and anxiety frequently affect children with epilepsy.

Therapies commonly used to treat depression in the general population have been shown in randomized controlled trials to be effective in treating depression in people with epilepsy. In those trials, depression medications did not appear to be associated with an increased risk of seizures. However, larger trials with longer followup would be required to provide reliable estimates of seizure exacerbation risk.

Basic research investigations currently are exploring the possibility that the development of depression, anxiety, and seizures may involve similar causes. In addition, studies of antiseizure drugs have focused on determining whether there may be an increased risk of suicide associated with specific medications. 

People with neurodevelopmental disabilities, such as autism spectrum disorder, attention deficit disorder, and learning disabilities are known to be at higher risk for epilepsy. Further investigation is needed to better understand these associations and if there is a shared mechanism between these neurodevelopmental disabilities and the epilepsies. Sleep disorders are common among people  with the epilepsies. By one estimate, fully 70 percent of people with epilepsy had some form of disordered breathing during sleep. In another study, researchers found that certain types of seizures were associated with sleeping, while others were more common during times of wakefulness—suggesting that more research is needed on how these patterns might inform medication adjustment.

Sudden Unexpected Death in Epilepsy (SUDEP)

Some people with epilepsy are at risk of SUDEP, which for years was largely unrecognized. Estimates of SUDEP risk vary, but some studies suggest that each year approximately one case of SUDEP occurs for every 1,000 people with the epilepsies. For some, this risk can be higher, depending on several factors. People with more difficult-to-control seizures tend to have a higher incidence of SUDEP. 

One study suggested that use of more than two antiseizure drugs at one time is a risk factor for SUDEP. However, it is not clear whether the use of multiple drugs causes SUDEP, or whether people who use multiple antiseizure drugs have a greater risk of death because their epilepsy is more severe or more difficult to control. People with tonic-clonic seizures, uncontrolled seizures, or  epilepsy combined with other neurological disorders also have an elevated risk for SUDEP.

Findings from an analysis of four studies showed that the highest risk of SUDEP can be seen in men younger than 60 years of age with at least a 15-year history of epilepsy from unexplained causes, who had frequent generalized tonicclonic seizures, and who were taking multiple antiseizure drugs. Although SUDEP is considered rare in children, some evidence suggests that children with certain types of epilepsies, such as Dravet syndrome, may have an elevated risk for SUDEP. 

Seizures are known to alter breathing and cardiac activity. Research suggests that drug therapies that address respiratory arrest and implantation of cardiac devices may reduce the risk of SUDEP in some individuals.

Early studies have described certain EEG patterns that may help identify people at elevated risk for SUDEP. In addition, several devices in the early stages of development aim to provide a warning when a seizure has the potential to put someone at risk for SUDEP. 

NINDS , nonprofit lay and professional organizations, and the CDC are providing significant funding toward studies aimed at better understanding SUDEP risk factors and mechanisms, which may yield strategies for screening and prevention. Plans are underway for an Epilepsy Center without Walls initiative devoted to multi-disciplinary research on SUDEP and increased surveillance and epidemiology studies.

Progress in Managing Specific Populations

Pregnancy and the epilepsies.

Understanding how to treat epilepsy in pregnant women and the impact of antiseizure medications on an unborn child are of paramount importance and have been the focus of several studies. The American Academy of Neurology and the American Epilepsy Society conducted evidence-based systematic reviews of pregnancy-related studies among women with epilepsy.

Emerging data from the NINDS-funded Maternal Outcomes and Neurodevelopmental Effects of Antiepileptic Drugs study, as well as multiple hospital- and population-based registries, are helping to better characterize the risk of birth defects associated with antiseizure medications. In general, higher doses of these  medications are associated with an increased risk of major congenital malformations. Findings from the registries and other studies include: 

  • Valproate is consistently associated with an increased risk of major congenital malformations, and studies suggest a specific increased risk of neural tube defects, such as spina bifida. Prenatal exposure to valproate has been shown to be associated with symptoms of autism in humans and animals. Valproate exposure in utero also has been shown to adversely affect a child’s cognitive function, particularly verbal abilities.
  • Carbamazepine may increase the risk of neural tube defects, but this is not a consistent finding. Verbal cognitive skills also have been shown to be impaired among children who were exposed to carbamazepine during gestation.
  • Topiramate increases the risk of oral clefts (birth defects in which the tissues of the lip or mouth do not form correctly during fetal development) as demonstrated in multiple studies. The FDA has classified it as a category D drug in pregnancy, meaning that evidence shows that the drug involves risk to a developing fetus, but the potential benefits from the drug may warrant its use in  pregnant women despite potential risks.
  • Levetiracetam appears to have a lower risk of major congenital malformations than other antiseizure drugs.
  • Motor, adaptive, and emotional behavioral functioning were impaired in children of mothers who had taken phenytoin, lamotrigine, valproate, or carbamazepine during pregnancy, with dose-response effects seen with valproate and carbamazepine. Breastfeeding did not affect cognitive health of the studied children.

Infants and Children

Febrile seizures occur in infants and young children and involve convulsions brought on by high fever. The vast majority of febrile seizures are brief and harmless. In rare cases, however, some children—including those with cerebral palsy, delayed development, or other neurological abnormalities—have an increased risk of developing epilepsy. 

Results from an ongoing NINDS-funded study suggested that MRI and EEG may help determine which children with febrile seizures are subsequently at increased risk of developing epilepsy. 

Older Adults

Epidemiological studies demonstrate that the elderly are at a substantially higher risk for the development of the epilepsies. In addition to stroke (hemorrhagic and ischemic), seizures in the elderly may be associated with brain tumors, TBI, and Alzheimer’s disease.

NIH-funded researchers have found that blood concentrations of antiseizure medications fluctuate markedly among many residents of nursing homes even when there is no change in dosage and no change in other medications the resident may be taking. Prospective studies will continue to follow older adults in nursing homes to help determine optimal levels of antiseizure drugs and to identify  factors that may contribute to such fluctuations in drug levels.

Diagnosing, Treating, and Preventing Non-epileptic Seizures

An estimated five to 20 percent of people diagnosed with epilepsy actually have nonepileptic seizures (NES) which outwardly resemble epileptic seizures but are not associated with seizure-like electrical discharges in the brain. A history of traumatic events is among the known risk factors for psychogenic nonepileptic seizures, which are largely thought to be psychological in origin. 

A NINDS-funded pilot trial showed a reduction in NES frequency when individuals with psychogenic non-epileptic seizures were treated with sertraline compared with a placebo. Two other studies showed a reduction in seizures and fewer comorbid symptoms following treatment with cognitive behavioral therapy.

Section 4: Furthering Research on the Epilepsies

There are several ways in which individuals with epilepsy and their families can help move research forward. Resources include: 

  • People with epilepsy can help researchers test new medications, surgical techniques, and other treatments by enrolling in clinical studies. Information about finding and participating in clinical studies can be found at the NIH Clinical Trials and You website ( www.nih.gov/health/clinicaltrials ). Additional studies can be found at www.clinicaltrials.gov and through many pharmaceutical and biotech companies, universities, and other organizations. A person who wishes to participate in a clinical trial must ask his or her physician to work with the doctor in charge of the trial and to forward all necessary medical records.
  • To learn more about why clinical trial research is important, visit the Human Epilepsy Research Opportunities (HERO) website at www.epilepsyhero.org. 
  • Pregnant women with epilepsy who are taking antiseizure drugs can help researchers learn how these drugs affect unborn children by participating in the Antiepileptic Drug Pregnancy Registry. This registry is maintained by the Genetics and Teratology Unit of Massachusetts General Hospital. For more information, call 1-888-233-2334 or visit the website at http://www.massgeneral.org/research/researchlab.aspx?id=1493&display=overview .
  • People with epilepsies can help further research by making arrangements to donate tissue either at the time of surgery for epilepsy or at the time of death. Researchers utilize the tissue to study epilepsy and other disorders so they can better understand what causes seizures. Some brain banks accept tissue from individuals with epilepsy. Each brain bank may have different protocols for registering a potential donor. Individuals are strongly encouraged to contact a brain bank directly to learn what needs to be done ahead of the time of tissue donation. These banks include:

NICHD Brain and Tissue Bank for Developmental Disorders University of Maryland School of Pediatrics 655 West Baltimore Street, 13-013 BRB Baltimore, MD 21201-1559 800-847-1539 http://www.btbank.org    University of Miami Brain Endowment Bank University of Miami Department of Neurology 1951 NW 7th Avenue, Suite 240 Miami, FL 33136 305-243-6219 or 800-862-7246 www.brainbank.med.miami.edu  

National Disease Research Interchange/National Human Tissue Resource Center 8 Penn Center, 15th Floor 1628 JFK Boulevard Philadelphia, PA 19103 215-557-7361 or 800-222-6374 www.ndriresource.org  

The Human Brain and Spinal Fluid Resource Center 11301 Wilshire Boulevard (127A) Building 212, Room 16 Los Angeles, CA 90073 310-268-3536 www.brainbank.ucla.edu  

The pace of research on the epilepsies has accelerated considerably over the past few decades. Progress has been made in understanding how and why the epilepsies develop and how they might be prevented. Investigators have identified a variety of potential new treatments, and they may soon be able to use knowledge about genetic variations and other individual differences to tailor treatment for each person. With time and continued work, the missing pieces of the puzzle will be filled in to form a complete picture of how to treat and prevent all types of epilepsy

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New approaches in epilepsy research.

new epilepsy research

At UCB, we have been leaders in the research and development of treatments for epileptic seizures for more than 20 years. In 2022, more than 2.6 million people living with epilepsy benefited from our anti-seizure medications, and our commitment to providing new solutions for those in need continues. We are working towards a future where we hope to impact the underlying causes of the epilepsies with disease modifying therapies which can change the course of the disease. It is important to recognise that there are multiple types of epilepsy. In fact, we now more commonly refer to epilepsy as the ‘epilepsies,’ indicating that it’s not really a single disease. In addition, for many people — particularly those living with certain, rare epileptic syndromes — seizures are not the only impactful symptom. As an industry, we've been very focused on seizures, which for so many patients, continues to be top of mind. But it’s essential that, in our focus on seizure suppression, we don’t neglect what changes in the brain are often underneath those seizures. When we understand the root cause of certain epilepsies, it opens opportunities to target the underlying mechanisms that impact these diseases, through new treatment approaches.  To further increase our understanding, we've been collaborating with universities on building one of the largest analyses of human brain tissues from epilepsy patients in the world, including > 200 samples. This in-depth study allows us to really comprehend the world of epilepsies as a global brain map, to quantify fully the different subtypes of epilepsy, understanding what they may have in common and what could be unique to specific conditions.  For complex epilepsies, where mechanisms are less understood, the situation is more challenging. Here, we are building on genetics to help understand universal disease mechanisms across the epilepsies. In addition, we have also developed an artificial intelligence computational framework in collaboration with various academic partners to investigate disease mechanisms and processes that helps us to identify novel areas for future therapeutic entry points.  Our digital innovations are also key in allowing our patients to have a louder voice, to feel more empowered, and to know and understand their disease more thoroughly. The combination of more diverse treatment options and a more informed patient population will help bring us in an era where the treatments will be much more personalized, impacting patients’ lives in a more significant way.  Our commitment to epilepsy research has never been stronger, and as the science advances so does our curiosity. We are hopeful for the future, and we will learn and refine our thinking as we progress.  

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UCL team develop mental health treatment for children with epilepsy

Grace Wood | The mental health treatment for children with epilepsy was published in The Lancet

The new mental health treatment is for children with epilepsy

The study was published in The Lancet on March 7, 2024.

The Mental Health Intervention for Children with Epilepsy (MICE) is based on recommended treatments for common mental health difficulties such as anxiety and depression, but was modified to help children who have more than one problem.

The study found that children who went through the MICE treatment had fewer mental health difficulties than those who had the usual treatment.

It was delivered over the phone or via video call so that people did not have to travel to hospital and miss time from school or work. It was also integrated into epilepsy services – meaning it could be delivered by non-mental-health specialists.

Patients were given an initial assessment followed by weekly calls with a clinician.

The study was done by researchers at UCL, in collaboration with Great Ormond Street Children’s Hospital, King’s College London and the University of California, Los Angeles, with funding from the National Institute for Health and Care Research.

Lead author Dr Sophie Bennett said: “This treatment breakthrough means we have a new way to help children and young people with epilepsy who also have mental health difficulties.

“The treatment can be delivered from within epilepsy services to join up care. It doesn’t need to be delivered by specialist mental health clinicians such as psychologists.

“Integrating the care can help children with epilepsy and their families more effectively and efficiently. We were particularly pleased that benefits were sustained when treatment ended.”

Researchers trialled the treatment with 334 children and young people aged three to 18. Of these, 166 received MICE treatment and 168 received the usual treatment for mental health problems in children with epilepsy.

Co-chief investigator, professor Roz Shafran, said: “These groundbreaking findings not only promise brighter futures for children with epilepsy but also pave the way for a revolutionary shift in mental healthcare practices.

“The collaborative efforts of scientists, patients and healthcare professionals have brought forth a new era of treatment of mental health challenges associated with epilepsy, offering a beacon of hope for families in the face of mental health challenges associated with epilepsy.”

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Research & Funding

About Research and Funding at Epilepsy Foundation

Epilepsy Foundation Chief Medical/Innovation Officer Jacqueline French MD talks about new epilepsy research, funding, and clinical trials.

Epilepsy is broadly underfunded and especially so in the translation of research insights into new therapies.

The Epilepsy Foundation has dedicated a significant part of our mission since our formation in 1968 to ensuring the best and the brightest young investigators get a chance to become involved in epilepsy and seizure research. Our goal was to make sure they built their careers with a focus on epilepsy. As a result, most of the top epilepsy researchers working today have received an Epilepsy Foundation grant early in their careers.

The Board of the Epilepsy Foundation has taken a broad lens to support the best new therapy ideas we could find anywhere in the world. Thus, together with our Board and a long list of amazing parents, friends, and professionals, the Foundation, with the strength of our recent merger with the Epilepsy Therapy Project, has become an organization dedicated to “accelerating ideas into therapies for people living with epilepsy.”

We do not work alone. In addition to contributions from families affected by epilepsy and their friends, partnerships with other organizations focused on improving the lives of people with epilepsy have been critical to the Epilepsy Foundation and the Epilepsy Therapy Project's ability to fund new therapies and have impact. These include critical funding and support from the Milken Family Foundation, from NYU/FACES, and also from unrestricted educational grants from industry to support the development of content on epilepsy.com.

For 10 years prior to the merger, Epilepsy Foundation and Epilepsy Therapy Project partnered to provide 50/50 funding of our New Therapy Grants program.

Our investment in new therapies and research is 100 percent about bringing better treatments in a timeframe that matters to people living with epilepsy and seizures. It is part of our unwavering commitment to the epilepsy community, and we invite you to help us make a difference.

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Angelini Pharma partners with Wazoku to improve target identification in epilepsy

12-Mar-2024 - Last updated on 12-Mar-2024 at 11:12 GMT

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Partnering with scale-up Wazoku, Angelini Pharma announced yesterday (March 11) that it aims to make use of the collective wisdom and expertise of its 700,000-strong crowd to drive innovation in epilepsy treatment.

The challenge, titled ‘New Digital Approaches to Target Discovery in Epilepsy,’ marks the beginning of a strategic collaboration between Angelini Pharma and Wazoku. The initiative seeks to uncover new digital approaches to target identification in epilepsy, with a specific emphasis on collecting and integrating multiple data types and developing new algorithms and tools for target selection.

Epilepsy, a neurological disorder affecting over 50 million individuals worldwide, presents a significant challenge, and according to the research undertaken by the company, approximately one-third of patients experience drug-resistant seizures.

Urgent need for more epilepsy treatments

Recognizing the urgent need for more effective therapies, Angelini Pharma aims to tackle innovative digital solutions to enhance target identification in drug-resistant epilepsy, thereby bolstering its early-stage pipeline and supporting its commitment to brain health.

Rafal Kaminski, chief scientific officer at Angelini Pharma, said he was optimistic about the potential impact of the open innovation challenge.  

Open innovation - a powerful tool

The company, known for its dedication to researching, developing, and commercializing health solutions, particularly in the field of brain health, views this collaboration with Wazoku as a significant step in driving innovation in epilepsy treatment.

This marks the company's first Open Innovation Challenge with Wazoku, signaling its commitment to embracing innovative approaches to address unmet medical needs.

Open innovation has emerged as a powerful tool in the pharmaceutical sector, facilitating accelerated drug discovery and development while addressing challenges such as rising operational costs and talent shortages.

Benefits of collective intelligence

Dino Ribic, innovation consultant at Wazoku, highlighted the transformative potential of open innovation, saying: “Progressive companies like Angelini Pharma have recognized the benefits of collective intelligence, and I can't wait to see what the Wazoku Crowd delivers in this instance.”

The Wazoku Crowd, comprising a diverse network of expert problem solvers, boasts a success rate of over 80% in solving more than 2,500 challenges, delivering over 200,000 innovations in the process. With cash prizes totaling up to $25,000 available for the "New Digital Approaches to Target Discovery in Epilepsy" challenge, Angelini Pharma anticipates receiving groundbreaking proposals from the Wazoku Crowd. The challenge is set to close on Monday next week, (18 March 2024). 

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The NINDS Topics in Clinical Research Seminar Series Presents: Negotiating Contracts for Your Next Job

This presentation will address:

  • Financial architecture of the health care system to understand how monies flow and what sources can provide income in different settings,
  • Payment models and funding mechanisms in the private practice and privademic settings,
  • How to negotiate contracts beyond money (i.e., other resources such as time, help, equipment), &
  • What responsibilities to expect when becoming an attending and how to leverage these responsibilities towards career growth. 

…with Q&A to follow!  

Please log on for the next  Topics in Clinical Research  meeting on  March 2 2 nd , 2024 via Microsoft Teams (link provided below) where we welcome  Rahul Dave, MD, PhD, Director, Neuroimmunology & MS Center, Inova Neurosciences Institute, University of Virginia College of Medicine & Omar Kahn, MD Director, Epilepsy Center of Excellence Baltimore Veterans Health Center, and Co-chair, ECOE Education Committee at the National VHA as t he y present on Negotiating Contracts for Your Next Job .

Dr. Rahul Dave

Bio:   Dr. Rahul Davé is a specialty care physician board certified in neurology at Inova Health System. He joined Inova Neurology in 2017. He specializes in the treatment of multiple sclerosis, neuromyelitis optica (NMO), optic neuritis, transverse myelitis, neuro-sarcoid, neurologic complications of rheumatologic diseases, autoimmune encephalitis, PML, inflammatory neuropathies, myasthenia gravis, paraneoplastic disorders, vasculitis and neuro-infectious diseases (including Lyme). He is the Medical Director of the interdisciplinary  Inova Multiple Sclerosis and Neuro-Immunology Center , which is recognized by the Consortium of Multiple Sclerosis Centers and is also a recipient of a "Partner" award by the National Multiple Sclerosis Society.

Dr. Omar Khan

Bio: Dr. Khan received his MBBS . from Baqai Medical & Dental College in Karachi, Pakistan in 1999 and received his ECFMG certificate in 2001. He completed his post¬graduate Residency training at Virginia Commonwealth University, Medical College of Front Royal, Virginia in 2006; a Neurology Residency at Dartmouth-Hitchcock Medical Center at Lebanon, New Hampshire in 2009; and a Clinical Neurophysiology and a Clinical Epilepsy Fellowship in  NINDS  in 2010 and 2011, respectively. Dr. Khan served as the Director of both the Neurology Consultation Service and the Medical Education Unit at NINDS from 2014 to 2019.   He is a board-certified ABPN neurologist, a board-certified ABPN Clinical Neurophysiology, as well as a board-certified ABCN Clinical Neurophysiologist with a specialty track in Epilepsy Monitoring. He currently serves as the Director of the epilepsy center of excellence at the Baltimore veterans health centre and also co-chairs the ECOE Education Committee at the national VHA. 


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Match Day 2024 – UNC Neurosurgery Welcomes Two New Neurosurgery Residents

March 15, 2024

By Makenzie Hardy

The UNC Health Department of Neurosurgery is pleased to announce that two new neurosurgery residents matched with our program and will join our neurosurgery residency program this summer. Zoe Robinow from California Northstate University College of Medicine and Deveney Franklin from the University of North Carolina at Chapel Hill School of Medicine will graduate from medical school in May and will begin their 7-year neurosurgical training over the summer.

We look forwarding to welcoming our two new neurosurgery residents and preparing them for successful careers in neurosurgery. Congratulations on your match!

UNC Neurosurgery Residents - Match Day 2024

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  23. Mental health treatment for children with epilepsy developed at UCL

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    Administrative Office: Physicians Office Building 170 Manning Drive, Campus Box 7060 Chapel Hill, NC 27599 (919) 966-8804 Clinics: (984)-974-4175