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Science News

Here are some of the biggest medical advances in 2023.

New treatments include the first CRISPR gene-editing therapy, an Alzheimer’s drug and RSV vaccines

In March, the U.S. Food and Drug Administration said the nasal spray Narcan, which can reverse the effects of an opioid overdose, could be sold over the counter.

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By Erin Wayman

December 14, 2023 at 7:00 am

Weight-loss drugs stole much of the spotlight in 2023, but these medical advances treating other conditions are also worthy of attention ( SN: 12/13/23 ).

Green light for CRISPR gene editing

On December 8, the U.S. Food and Drug Administration approved the world’s first CRISPR/Cas9 gene-editing therapy ( SN: 12/8/23 ). The treatment, called Casgevy, targets sickle cell disease by helping patients produce healthy hemoglobin. In people with the disease, hemoglobin is abnormal, causing red blood cells to become hard and crescent shaped, which can block blood flow. By March 2024, the FDA will decide whether the same therapy can be used to treat beta-thalassemia, a disorder that reduces hemoglobin production.

Slowing down Alzheimer’s

The Alzheimer’s drug lecanemab (brand name Leqembi) won full FDA approval in July. Like the drug aducanumab approved in 2021, lecanemab removes the amyloid plaques that build up in the brains of people with Alzheimer’s. The drug doesn’t stop the disease, but in a clinical trial, lecanemab slowed cognitive decline by about 30 percent over 18 months compared with a placebo ( SN: 8/12/23, p. 9 ).

A gene therapy for muscular dystrophy

In June, the FDA approved the first gene therapy for children with Duchenne muscular dystrophy. Due to a faulty gene, people with this muscle-wasting disease don’t make the protein dystrophin, which helps keep muscle cells intact. The therapy helps the body produce a version of the missing protein ( SN: 6/22/23 ).

Guarding against RSV

Several ways to protect against respiratory syncytial virus arrived this year. In May, the FDA approved the first RSV vaccine, called Arexvy, in the United States , for adults age 60 and older ( SN: 6/17/23, p. 8 ), and then in August, a vaccine for pregnant people , called Abrysvo ( SN: 8/25/23 ). A monoclonal antibody — a lab-made antibody that mimics immune system proteins — won approval in July to protect children 2 and younger from the virus, which sends as many as 80,000 young children to U.S. hospitals each year (S N: 4/27/23 ). But in October, limited supplies of the therapy led the U.S. Centers for Disease Control and Prevention to recommend reserving it for babies at highest risk for complications from RSV.

A pill for postpartum depression

Until August, the only medication in the United States specifically targeting postpartum depression required a 60-hour intravenous infusion in a hospital ( SN: 3/22/19 ). With FDA approval of zuranolone (brand name Zurzuvae), those afflicted with postpartum depression can take an oral medication at home and experience improvement in as little as three days .

Birth control, no prescription required

In July, the FDA ruled that the oral contraceptive norgestrel, first approved in 1973, be available without a prescription. It’s the first OTC daily birth control pill in the United States. Some public health experts argue that reducing barriers to contraception is especially important to reproductive autonomy now that state bans have limited access to abortion ( SN: 5/19/23 ).

A shot against chikungunya

The chikungunya virus can cause fever and severe joint pain, and be fatal to newborns. In November, the FDA approved the first vaccine against the virus , which is transmitted by mosquitoes. The virus is most prevalent in tropical regions, but the FDA warns that it’s spreading to new parts of the globe.

Narcan over the counter

The nasal spray Narcan, aka naloxone, can reverse the effects of an opioid overdose within minutes. In March, the FDA ruled this life-saving drug can be sold over the counter . Officials hope that easier access to Narcan can help fight the opioid epidemic, which claimed the lives of nearly 645,000 people from 1999 to 2021 due to overdoses.

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Lecanemab, the New Alzheimer’s Treatment: 3 Things To Know

BY CARRIE MACMILLAN July 24, 2023

Yale researcher discusses the recent FDA approval of a new Alzheimer's disease treatment.

[Originally published January 19, 2023. Updated: July 24, 2023.]

The Food and Drug Administration (FDA) recently granted full approval to a new Alzheimer’s treatment called lecanemab, which has been shown to moderately slow cognitive and functional decline in early-stage cases of the disease.

Alzheimer’s disease is a progressive disorder that damages and destroys nerve cells in the brain. Over time, the disease leads to a gradual loss of cognitive functions, including the ability to remember, reason, use language, and recognize familiar places. It can also cause a range of behavioral changes.

In January, the FDA gave the medication an accelerated approval based on amyloid plaque clearance. Christopher van Dyck, MD , director of Yale’s Alzheimer’s Disease Research Unit, was the lead author of a study published in the Jan. 5 issue of The New England Journal of Medicine that shared results of a Phase III clinical trial of lecanemab. (Dr. van Dyck is also a paid consultant for the pharmaceutical company Eisai, which funded the trials.)

Sold under the brand name Leqembi™ and made by Eisai in partnership with Biogen Inc., the drug is delivered by an intravenous infusion every two weeks. Lecanemab works by removing a sticky protein from the brain that is believed to cause Alzheimer’s disease to advance.

“It’s very exciting because this is the first treatment in our history that shows an unequivocal slowing of decline in Alzheimer’s disease,” says Dr. van Dyck.

This is the first time in two decades that the FDA has granted full approval to a drug for Alzheimer’s, but there is also a “black box” warning on the medication—the agency’s strongest caution—because of safety concerns.

We talked more with Dr. van Dyck, who answered three questions about the new treatment.

How effective is lecanemab for Alzheimer’s disease?

In a trial that involved 1,795 participants with early-stage, symptomatic Alzheimer’s, lecanemab slowed clinical decline by 27% after 18 months of treatment compared with those who received a placebo.

“The antibody treatment selectively targets the forms of amyloid protein that are thought to be the most toxic to brain cells,” says Dr. van Dyck.

Study participants who received the treatment had a significant reduction in amyloid burden in imaging tests, usually reaching normal levels by the end of the trial. Participants also showed a 26% slowing of decline in a key secondary measure of cognitive function and a 37% slowing of decline in a measure of daily living compared to the placebo group.

“Would I like the numbers to be higher? Of course, but I don’t think this is a small effect,” says Dr. van Dyck. “These results could also indicate a starting point for bigger effects. The data appear encouraging that the longer the treatment period, the better the effect. But we’ll need more studies to determine if that’s true.”

They also beg the question about still-earlier intervention, adds Dr. van Dyck. Lecanemab is already being tested in the global AHEAD study for individuals who are still cognitively normal but at high risk of symptoms due to elevated levels of brain amyloid.

Yale currently has the largest number of participants in the AHEAD study, which is funded by the National Institutes of Health (NIH) and Eisai and is enrolling participants as young as 55. “We may see a larger benefit if we intervene before significant brain damage has occurred,” he says.

Is lecanemab safe?

The most common side effect (26.4% of participants vs. 7.4% in the placebo group) of the treatment is an infusion-related reaction, which may include transient symptoms, such as flushing, chills, fever, rash, and body aches. The majority (96%) of these reactions were mild to moderate, and 75% happened after the first dose.

“We can medicate those individuals in advance if we find they have those side effects repeatedly,” says Dr. van Dyck. “We can use medications such as diphenhydramine or acetaminophen. But this is generally not an issue.”

Another potential side effect associated with lecanemab was amyloid-related imaging abnormalities with edema, or fluid formation on the brain. This occurred in 12.6% of trial participants compared to 1.7% in the placebo group. “It’s usually asymptomatic when it occurs, but we can detect it on MRI scans. We often don’t stop dosing if we see it, unless there are symptoms, in which case we would pause infusions until it fully resolves,” Dr. van Dyck says.

It’s important to note that the studies with lecanemab show substantially lower rates of this side effect than do published trials of other, similar drugs such as aducanumab—they're at about a third of the rate, explains Dr. van Dyck. “So, for drugs in this class, I think lecanemab has a favorable safety profile,” he says.

Lastly, 17.3% of trial participants experienced amyloid-related imaging abnormalities with brain bleeding compared to 9% in the placebo group.

“Most of the time we're really talking about microhemorrhages that are in the order of millimeters,” says Dr. van Dyck. “People with Alzheimer's disease are more prone to these events because of the amyloid deposits in their blood vessels, but a catastrophic bleed is quite rare.”

The medication’s label includes warnings about brain swelling and bleeding and that people with a gene mutation that increases their risk of Alzheimer’s disease are at greater risk of brain swelling on the treatment. The label also cautions against taking blood thinners while on the medication.

When will lecanemab be available for Alzheimer’s disease treatment?

Eisai set the price for Leqembi at $26,500 per year, and it has reportedly been largely unavailable while FDA full approval was pending. That may change now that Medicare has said it will cover 80% of the cost.

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Nih research matters.

December 21, 2023

2023 NIH Research Highlights - Promising Medical Findings

Results with potential for enhancing human health.

With NIH support, scientists across the United States and around the world conduct wide-ranging research to discover ways to enhance health, lengthen life and reduce illness and disability. Groundbreaking NIH-funded research often receives top scientific honors. In 2023, these honors included  two NIH-supported scientists who received Nobel Prizes . Here’s just a small sample of the NIH-supported research accomplishments in 2023. Also see this year's  Human Health Advances  and  Basic Research Insights .

Printer-friendly version of full 2023 NIH Research Highlights

Immune and hormonal features of Long COVID

About one in eight people who survive an acute SARS-CoV-2 infection go on to have persistent symptoms. The processes that give rise to this syndrome, known as Long COVID, remain unclear. Researchers found several immune and hormonal differences between people with Long COVID and those without. Another study found that infection with a common cold virus may predispose some people to develop Long COVID . This year, researchers also discovered how COVID-19 may damage cells’ energy production and potentially cause some symptoms of Long COVID.

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Protein may be linked to exercise intolerance in ME/CFS

People with myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) live with debilitating symptoms. These including exhaustion, exercise intolerance, cognitive problems and worsening of symptoms after even mild exertion. A study suggested that high levels of a protein called WASF3 may reduce energy production in the muscle cells of people with ME/CFS. Blocking this protein in cells in the laboratory restored energy production, suggesting a potential new strategy for treating the condition.

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Engineering skin grafts for complex body parts

Advances in bioengineering have allowed researchers to grow new patches of skin in the lab. But these skin patches have been small and limited in shape. Using new techniques, scientists grew strong skin in the shape of a full human hand. This technology has the potential to help heal burns and other damage to complex body parts with less trauma and scarring.

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Blood test for early Alzheimer’s detection

One of the first stages of Alzheimer’s disease involves the formation of toxic aggregates of a protein called amyloid beta (Aβ). The ability to detect these early would let scientists test new treatments before irreparable brain damage occurs. Researchers developed a blood test that could detect the toxic Aβ aggregates before Alzheimer’s symptoms appeared. This is one of several promising approaches to early diagnosis of Alzheimer’s and other dementias.

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Erythritol and cardiovascular events

Artificial sweeteners can help people reduce their sugar and calorie intake. But little is known about the long-term health consequences. Researchers found that elevated blood levels of the artificial sweetener erythritol were associated with increased risk of heart attack and stroke. When used as a sweetener, erythritol is typically added at levels more than 1,000-fold higher than those found naturally in foods. The results highlight the need to further study erythritol’s long-term effects on cardiovascular health.

Read more 2023 NIH Research Highlights: Basic Research Insights

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Our comprehensive coverage encompasses various medical fields, including genetics, neuroscience, mental health, nutrition, and more. By staying informed on the latest medical advancements, we empower you to take control of your health and well-being.

Learn more about the latest research into Antibiotics , Medicine , Cancer , Diet , Stem Cells , Nutrition , Immunobiology , Alzheimer’s , Psychiatry , Metabolism , Pregnancy , and Cardiology . Join us as we explore the future of healthcare and unravel the complexities of the human body.

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Ozempic v. Mounjaro? There's a clear winner for weight loss, study finds.

One next-generation weight loss drug jumps ahead of the other in terms of effectiveness, but remains extremely hard to access..

People lost substantially more weight when they took Eli Lilly's diabetes drug Mounjaro than Novo Nordisk's Ozempic, with roughly the same side effects, drop-out rate and benefit for diabetes, according to the first major head-to-head study.

Both drugs are in the same class, known as GLP-1s, but Lilly's drug, tirzepatide, sold under the brand names Mounjaro for diabetes and Zepbound for weight loss, includes a second action that seems to boost its effectiveness. Separate studies had suggested tirzepatide led to more weight loss than semaglutide, which Novo Nordisk sells as Ozempic, for diabetes and, Wegovy, at a higher dose, for weight loss.

The new study, published Monday in JAMA Internal Medicine , compared data on more than 18,000 patients who began taking one of the two medications between May 2022 and September 2023. The study relied on electronic health records from Truveta, a collective of health systems across the country with access to data on more than 100 million patients.

At the time, tirzepatide hadn't yet been approved for weight loss, just diabetes, so the study looked only at people who had prescriptions for the two drugs for type 2 diabetes, although not everyone had the disease.

By November of last year, more than half of the people on both medications ‒ 56% of those taking tirzepatide and 53% of those taking semaglutide ‒ had stopped taking them. Although the study didn't confirm a reason, many have reported side effects from the drugs, typically gastrointestinal, including vomiting and nausea.

The study didn't report whether the people who dropped out regained any weight they lost. Research has shown that so-called yo-yo dieting can cause more health problems than simply carrying extra pounds.

But GLP-1s are by far the most effective and safest class of weight loss drugs ever developed. More than 70% of Americans meet the medical definition for being overweight and 40% for obesity.

"As we've tracked GLP-1 use over the last couple of years, we've just seen these dramatic increases of use," said Tricia Rodriguez, who led the study for Truveta Research, and both drugs are "really revolutionizing the treatment of both diabetes and obesity."

Clear winner? Not so fast, Novo says

Novo Nordisk disputed the study's conclusion.

"The ideal way to compare two treatments is an adequately powered head-to-head randomized clinical trial (RCT) in obesity. Currently, no head-to-head trials have been completed comparing tirzepatide and semaglutide 2.4 mg," the company said in a statement sent by Allison Schneider, the company's director of media relations and issues management.

The 2.4 mg dosage is the highest of semaglutide typically used for weight loss. The highest dose used to address diabetes is lower, so the dosage used by people in the study, which focused on people diagnosed with diabetes, would have been below the optimal dose for weight loss.

For tirzepatide, the highest dosage is the same, whether for weight loss or diabetes.

Specific takeaways from the study

People taking tirzepatide were nearly twice as likely to lose 5% of their body weight as those taking semaglutide; 2.5 times as likely to lose 10% of their body weight; and more than three times as likely to lose 15% of their body weight, the study showed.

After three months on each drug, those taking tirzepatide had lost an average of about 6%, while those taking semaglutide had lost just under 4%. At six months, those on tirzepatide had lost 10%, while those on semaglutide had lost 6%. And at a year, those on tirzepatide had lost 15% of their body weight, compared with 8% for those on semaglutide.

As has been found in other studies, people with diabetes lost less weight on the medications than people who had obesity but not diabetes.

Among the 18,386 people studied, 70% were female, 77% were white, 11% were Black and just over half had type 2 diabetes. Their average body-mass index was 39 and their average age was 52. More than 1 in 5 also had been diagnosed with depression.

Obesity, diabetes and depression often overlap, in part because they are all so common, in part because medications for mental health conditions are associated with weight gain and diabetes onset, and in part because they contribute to one another, said Dr. Katherine Saunders, an obesity medicine expert at Weill Cornell Medicine in New York.

"Obesity and diabetes can worsen depression, and depression can worsen obesity and diabetes," she said.

Why they're dropping out

Many patients stop taking these extremely effective medications because of side effects and trouble accessing them, say doctors who prescribe them.

The side effects can typically be controlled with adequate medical oversight and proper ramping up of the medication, experts say.

"Patients require more than a prescription for a GLP-1 medication in the last few minutes of a busy appointment," said Saunders, also co-founder of Intellihealth, which provides medical obesity treatment.

Personalized care, time and "tons of education and support" are needed to help people start and stick to these medications, she said.

Both drugs are meant to be taken in steps, starting at a low dose and advancing slowly to higher doses. If a patient cannot tolerate a higher dose, they are typically left at a lower one indefinitely or until side effects lessen, said Dr. Fatima Cody Stanford, an obesity medicine specialist at Massachusetts General Hospital in Boston.

Cody Stanford said her only patients who discontinue a GLP-1 for medical reasons are the 1 in 1,000 or so who develop pancreatitis, a painful inflammation of the pancreas, and the 15% or so who don't see any substantial weight loss after a significant period of time.

For those people, Cody Stanford said, she often has to convince them the drug others describe as a "miracle" simply isn't going to work for them.

"This isn't a 'try harder' situation," she said. "If it works, it works."

Cost and access problems

A much more common reason for discontinuing a GLP-1, Cody Stanford said, is an inability to get access to a reliable and consistent supply.

Both drugs have been hard to access because of supply constraints .

Novo Nordisk says it has provided Wegovy to more than 1 million Americans since 2021, and the highest dose strengths of 1.7 mg and 2.4 mg are now fully available, "which aligns to our goal of doing our best to ensure existing patients have continuity of care as they dose escalate per the label," according to a company statement.

"At this time, we can’t speculate when Wegovy will become fully available at all dose strengths but we are doing everything we can to build manufacturing capacity and supply to meet patient needs," the statement said.

Supply updates are available at WegovySupply.com .

Lilly, which declined to comment saying they were not involved in the new research, has struggled to keep up with demand for tirzepatide, particularly since it was approved for weight loss under the brand name Zepbound.

For her patients, Fatima Cody Stanford said she now has been able to access Novo's semaglutide but not Lilly's tirzepatide.

Both drugs cost about $1,000 a month for the highest dose, and Wegovy, Novo Nordisk's semaglutide formulation for weight loss, costs more than Ozempic, its diabetes drug, because the highest dose is higher.

Cody Stanford said the high price and access problems mean people who need the medications most have the hardest time getting them.

Most of the study participants were middle-class white women ‒ and those prescribed tirzepatide were even more likely to be white and female, Rodriguez said. There's a difference between the patients on tirzepatide and those on semaglutide, and some patients can't access either one, she said.

Black, Hispanic and Native women have the highest rates of obesity and diabetes but typically don't get coverage for GLP-1s. "There's a mismatch in who gets coverage with these more novel therapies and who doesn't," Cody Stanford said.

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Scientists create first mouse model with complete, functional human immune system

by University of Texas Health Science Center at San Antonio

A breakthrough for biomedical research promises new insight into immunotherapy development and disease modeling. Scientists at The University of Texas Health Science Center at San Antonio have created a humanized mouse model with a human immune system and a human-like gut microbiome that is capable of mounting specific antibody responses.

The scientists were led by Paolo Casali, MD, University of Texas Ashbel Smith Professor and Distinguished Research Professor, Department of Microbiology, Immunology and Molecular Genetics in the Joe R. and Teresa Lozano Long School of Medicine. Casali has five decades of biomedical research experience in immunology and microbiology and is a leading researcher in molecular genetics and epigenetics of the antibody response.

The aim of the multi-year project, which appears in the August 2024 issue of Nature Immunology , was to overcome limitations of currently available in vivo human models by creating a humanized mouse with a fully developed and functional human immune system .

Mice are widely used in biological and biomedical research because they are small, easy to handle, share many immune elements and biological properties with humans and are easily genetically modified.

Many of the more than the 1,600 immune response mouse genes, however, are incongruent with their human equivalents, resulting in divergencies or deficiencies of mice as predictors of human immune responses. This made availability of a "humanized" mouse model that faithfully reproduces human immune responses a high priority.

The first humanized mice were created in the 1980s to model human HIV infection and the human immune response to HIV. Humanized mice were, and have been created since, by injecting immunodeficient mice with human peripheral lymphocytes, hematopoietic stem cells or other human cells.

Previous and current models, however, do not develop a fully functional human immune system, have a brief lifespan and do not mount efficient immune responses. This makes them unsuitable for development of in vivo human immunotherapies, human disease modeling or human vaccine development.

Casali's team began with injecting immunodeficient NSG W41 mutant mice intracardiacally (left ventricle) with human stem cells they purified from umbilical cord blood.

After a few weeks, once the graft has been established, the mice are hormonally conditioned with 17b-estradiol (E2), the most potent and abundant form of estrogen in the body. Hormonal conditioning by estrogen was prompted by previous research by Casali and others suggesting that estrogen boosts the survival of human stem cells, boosts B lymphocyte differentiation and production of antibodies to viruses and bacteria.

The resulting humanized mice, called TruHuX (for truly human, or THX), possess a fully developed and fully functional human immune system, including lymph nodes, germinal centers, thymus human epithelial cells, human T and B lymphocytes, memory B lymphocytes, and plasma cells making highly specific antibody and autoantibodies identical to those of humans.

THX mice mount mature neutralizing antibody responses to Salmonella Typhimurium and SARS-CoV-2 virus Spike S1 RBD after vaccination with Salmonella flagellin and the Pfizer COVID-19 mRNA vaccine, respectively. THX mice are also amenable to developing full-fledged systemic lupus autoimmunity after an injection of pristane, an oil that triggers an inflammatory response.

Casali said the THX mouse discovery opens the possibilities for human in vivo experimentation, for development of immunotherapeutics such as cancer checkpoint inhibitors, development of human bacterial and viral vaccines, as well as the modeling of many human diseases. He also hopes the new approach could make obsolete the use of non-human primates for immunological and microbiological biomedical research.

As prior research on the effect of estrogen and the immune system is sparse, Casali hopes this discovery prompts further research into the topic.

"By critically leveraging estrogen activity to support human stem cell and human immune cell differentiation and antibody responses, THX mice provide a platform for human immune system studies, development of human vaccines and testing of therapeutics," Casali said.

With the THX model, the Casali lab is now investigating the in vivo human immune response to SARS-CoV-2 (COVID-19) at the systemic and local levels, and human memory B lymphocytes, the dependence on nuclear receptor RORα for their generation and the events that lead to RORα expression and dysregulation.

They are also exploring epigenetic factors and mechanisms that mediate generation of human plasma cells, the cell factories that make antibodies—literally thousands per second—to bacteria, viruses or cancer cells.

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New compound could supercharge naloxone in fight against opioid overdoses

In a Stanford Medicine-led study, researchers combed through billions of compounds to find one that could enhance naloxone’s ability to fend off more potent opioids, with promising results in mice.

July 3, 2024 - By Nina Bai

Naloxone (orange) treats opioid overdose by kicking out opioids (pink) from the opioid receptor (teal). The newly discovered compound 368 (purple) strengthens the binding of naloxone to the opioid receptor, making it a more effective life-saving medicine. Emily Moskal

Every great superhero needs a sidekick. Now, scientists may have found a drug-busting partner for naloxone.

Naloxone is an opioid antidote that has saved tens of thousands of lives by rapidly reversing opioid overdoses in more than 90% of cases in which it is used. But its powers are temporary, lasting only 30 to 90 minutes. The rise of potent, long-acting opioids such as fentanyl means that someone brought back from the brink can still overdose after the naloxone wears off.

In a new study, Stanford Medicine scientists and collaborators have discovered a novel compound that can work alongside naloxone, supercharging its life-saving effects.

When tested in mice, adding the compound to a miniscule dose of naloxone made it as powerful as the conventional dosage, with the added benefit of milder withdrawal symptoms.

Naloxone, which is given as a nasal spray or injection, works by seizing opioid receptors, kicking out opioids and taking their place. (Naloxone has no addictive properties of its own.) The researchers found that the new compound — known for now as compound 368 — binds next to naloxone on opioid receptors and helpfully holds naloxone in place.

The findings were published July 3 in Nature .

“Naloxone binding to an opioid receptor turns it mostly off, but not all the way,” said Evan O’Brien , PhD, a postdoctoral scholar in molecular and cellular physiology and the lead author of the new study. “Our data shows that compound 368 is able to increase the binding of naloxone and turn the receptor off more completely.”

A new type of drug

The new compound belongs to an unusual class of drugs that don’t directly target the active site on receptors. Instead, they bind elsewhere on the receptor but trigger a structural change that alters the active site. Known as allosteric modulators (allos meaning “other” in Greek), they create new possibilities in drug development, but are trickier to identify, O’Brien said. 

Evan O'Brien

“Allosteric modulators are not common yet, and they’re a lot more difficult to discover and to work with,” he said.

Compound 368 is the first known allosteric modulator that can help turn off opioid receptors.

The researchers picked out compound 368 from a library of 4.5 billion compounds. Using advanced high-throughput techniques, they were able to screen the entire molecular library in just two days. To identify potential allosteric modulators that could cooperate with naloxone, they selected for compounds that bind only to receptors already saturated with naloxone.

Compound 368 — an otherwise rather unremarkable compound, O’Brien said — stood out for its ability to tightly bind to opioid receptors only in the presence of naloxone. Like a loyal sidekick, it doesn’t work with other drugs, and it doesn’t work alone.

Powers combined

When researchers exposed cells with opioid receptors to compound 368, they found that the compound alone made little difference. But when cells were given the compound with naloxone, the combination was a powerful deterrent against opioid binding.

The more compound 368 they added, the better naloxone was able to block opioids, including morphine and fentanyl.

“The compound itself doesn’t bind well without naloxone,” O’Brien said. “We think naloxone has to bind first, and then compound 368 is able to come in and cap it in place.”

Indeed, using cryoEM imaging to visualize frozen molecular structures, the researchers found that compound 368 docks right next to naloxone on the opioid receptor, forming bonds that secure the drug in place and slow its natural degradation by the body.

Boosting naloxone

Next, collaborators in McLaughlin’s lab tested the new compound in mice that had been given morphine. Because opioids reduce pain sensation, the researchers observed how quickly a mouse removed its tail from hot water. The stronger the opioid antidote, the faster a mouse would take its tail out of the water.

When mice on morphine were treated with compound 368 alone, nothing changed.

“The compound in mice, at least from the assays we’ve run, does nothing on its own,” O’Brien said. “We don’t observe any off-target effects. We don’t see anything happen to the mice even when we inject a massive amount of compound 368.”

This was exactly what the researchers had predicted from their molecular work and a good sign of the compound’s safety, he added.

The more tools at our disposal, the better we’ll be able to fight this epidemic of fentanyl overdoses.

When they also gave the mice a small dose of naloxone — an amount that typically would have no effect — the pairing with compound 368 dramatically improved naloxone’s effects.

“When we start to give them more and more of compound 368 with that low dose of naloxone, they take their tail out of the water pretty quickly,” O’Brien said.

Other effects of opioids, such as respiratory depression (the usual cause of death in opioid overdoses), were also reversed by a small dose of naloxone enhanced with the new compound.

Remarkably, the combination of compound 368 with a half dose of naloxone was strong enough to counter fentanyl, which is about 100 times more potent than morphine and the main culprit of overdoses in the United States.

By requiring less naloxone, the new compound could also ease the withdrawal symptoms that opioid users experience after overdose treatment. These symptoms — including body aches, shivering, nausea and diarrhea — are immediate and can be extremely uncomfortable, O’Brien said.

The researchers found that a low dose of naloxone plus compound 368 could reverse the effects of opioids with much milder withdrawal symptoms — in mice, this meant less teeth chattering, jumping and diarrhea.

Saving lives

The team, with the Majumdar lab’s expertise in medicinal chemistry, is now tweaking compound 368 so it can help naloxone counter strong opioids for longer durations.

“We’re still working on optimizing the compound’s properties for those longer-lasting effects,” O’Brien said. “But first showing that it works cooperatively with these low doses of naloxone suggests that we’re on the right track.”

O’Brien is optimistic that this track will lead to trials in humans. Overdoses from synthetic opioids, primarily fentanyl, continue to surge, killing nearly 74,000 Americans in 2022. “The more tools at our disposal, the better we’ll be able to fight this epidemic of fentanyl overdoses,” he said.

Researchers from Kurume University, SLAC National Acceleration Laboratory, Princeton University and University of Copenhagen also contributed to the work.

The study received funding from an American Diabetes Association Postdoctoral Fellowship, an American Heart Association Postdoctoral Fellowship, the National Institute of Health (grant RO1DA057790) and the Chan Zuckerberg Biohub.

About Stanford Medicine

Stanford Medicine is an integrated academic health system comprising the Stanford School of Medicine and adult and pediatric health care delivery systems. Together, they harness the full potential of biomedicine through collaborative research, education and clinical care for patients. For more information, please visit med.stanford.edu .

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Immunotherapy for NSCLC: Potential Biomarkers for Resistance Are Identified

Immunotherapy can be a highly effective treatment for non-small cell lung cancer (NSCLC), but some patients are resistant to the therapy or develop intolerable side effects. Now, researchers from Yale School of Medicine (YSM) have identified two ribosomal RNA genes that could serve as potential biomarkers to assess whether patients will respond to immunotherapy for NSCLC, though further research is needed. NSCLC is the most common form of lung cancer, often triggered by smoking.

The study was published on June 10 in the Journal for ImmunoTherapy of Cancer . Researchers screened over 18,000 genes from tissue samples of NSCLC patients who had undergone a cancer treatment called immune checkpoint inhibitor therapy (ICI). ICI works by preventing a cellular interaction that prevents the immune system from destroying tumor cells. Out of the thousands of genetic candidates, researchers identified two that were significantly associated with poor outcomes after ICI.

While ICI has been a transformative treatment for lung cancer, some patients can become resistant to the therapy, and researchers aren’t exactly sure how or why. Finding a gene that correlates with resistance to the treatment could help physicians guide patients in deciding whether to use ICI, potentially preventing an unnecessary risk of side effects, especially in the early cancer setting. According to David Rimm, MD, PhD , Anthony N. Brady Professor of Pathology, professor of medicine (medical oncology), member of Yale Cancer Center, and principal investigator of the study, one in five patients undergoing ICI treatment loses thyroid function and must take thyroid replacement pills, and as many as one in 100 patients will die from other complications of ICI therapy.

And when there’s a possibility that the tumor will be resistant to immunotherapy . Rimm says, the risk of these daunting side effects isn’t worth it. “[ICI] is not a drug that you want to give lightly,” says Rimm. “In early cancer, we want to make sure that the patient is going to benefit before they get the drug.”

[ICI] is not a drug that you want to give lightly. In early cancer, we want to make sure that the patient is going to benefit before they get the drug.” David Rimm, MD, PhD

Rimm’s current research may help identify patients who are best suited for immunotherapy. “You can imagine someday a patient might be tested for these biomarkers, and then if they’re negative they could have immune checkpoint inhibitors in the adjuvant setting,” says Rimm, meaning treatment given after initial cancer treatments like chemotherapy and surgery. “If they’re positive, we might want to opt for other adjuvant options and not expose them to the risk of an immune checkpoint inhibitor.”

ICI is transforming lung cancer treatment

ICI therapy was first used in 2011 to treat melanoma . Since then, ICI drugs have been used to treat a variety of cancers, such as breast cancer, colon cancer, and lung cancer.

“Immune checkpoint inhibitors are probably the most important new drug in oncology in the last 15 years,” says Rimm.

Immune checkpoints are naturally occurring proteins that help prevent the immune system from destroying healthy tissue. Immune cells called T cells contain checkpoint proteins, and healthy cells contain checkpoint inhibitors. When a T cell encounters a healthy cell, its checkpoint protein interacts with the checkpoint inhibitor. This essentially “turns off” the T cell, leaving the healthy cell alone.

But sometimes cancer cells will also display checkpoint inhibitors and turn off the T cell when it should be active. ICI drugs may work by blocking the binding of the checkpoint protein and the checkpoint inhibitor, leaving the T cell active and free to attack the cancer, or they may control immune regulatory cells.

Rimm’s team used tissue samples from NSCLC patients to search for biomarkers that could identify whether a patient’s cancer would become resistant to ICI therapy. The two biomarkers that the team found were ribosomal RNA genes. The researchers were surprised by this finding and were able to only speculate about how these RNA genes might contribute to immunotherapy resistance. More research will be necessary, Rimm says.

“This discovery was the result of what they sometimes have called in our field a ‘fishing expedition,’ because we have no underlying hypothesis, thus, at this point, we do not know how these genes work to regulate the immune system,” he adds. “This is the very first piece of evidence. I would need a whole pile of evidence before we take this to patients.”

A “spatially informed” approach

While future studies are needed to understand the mechanisms of these biomarkers and to determine if the same biomarkers show up in other cohorts of NSCLC patients, Rimm maintains that the most important takeaway from the study was the “spatially informed” approach that the researchers took to find these genes.

Typically, scientists looking for cancer biomarkers will take tumor tissue and mix it up, combining tumor cells, immune cells, and inflammatory cells. They will then look for biomarkers in this cellular soup, making it difficult to identify the specific cell type from which a certain biomarker was found.

But in this study, the researchers took a more delicate approach. Using a new technology called digital spatial profiling, they sampled biomarkers specifically from tumor cells, and not from surrounding inflammatory or immune cells.

“If you use a technology that takes biomarkers from specific regions of the tumor … you get more information than if you just mix up the whole thing,” Rimm says. “Ultimately, we believe that we need to measure those biomarkers from tissue in ways that are specific to specific cells.”

The field is already moving toward this type of approach, Rimm says, which may allow researchers to discover new biomarkers that have not been identified.

Featured in this article

• David Rimm, MD, PhD Anthony N. Brady Professor of Pathology and Professor of Medicine (Medical Oncology); Director, Yale Cancer Center Tissue Microarray Facility, Pathology; Director, Yale Pathology Tissue Services, Pathology; Director, Physician Scientist Training Program, Pathology Research

The Bulletin on Health

Affiliates' Research in Medical Journals, Summer 2024

Many NBER-affiliated researchers publish some of their health-related findings in journals that preclude pre-publication distribution, and thus do not post them as NBER Working Papers. This is a partial listing of recent papers in this category by NBER affiliates.

Clinical Outcomes after Admission of Patients with COVID-19 to Skilled Nursing Facilities McGarry BE,  Gandhi AD , Chughtai MA, Yin J, Barnett ML.  JAMA Internal Medicine,  June 2024.

To assess the association between the admission of COVID-19-positive patients to Skilled Nursing Facilities (SNFs) and subsequent COVID-19 cases and death rates among residents, this study used survey data from the National Healthcare Safety Network of the Centers for Disease Control and Prevention. In a cohort of SNFs in the US from June 2020 to March 2021, 264 exposed facilities (with initial admission of COVID-19-positive patients) were matched to 518 control facilities (without such admissions) in the same county and with similar preadmission case counts. A stacked difference-in-differences approach was used to compare outcomes for 10 weeks before and 15 weeks after the first admission. Exposed SNFs had a cumulative increase of 6.94 additional COVID-19 cases per 100 residents compared with control SNFs, a 31.3 percent increase compared with the sample mean (SD) of 22.2 (26.4). Exposed facilities experienced 2.31 additional cumulative COVID-19-related deaths per 100 residents compared with control facilities. Exposed facilities experiencing potential staff shortages and a personal protective equipment shortage had larger increases in COVID-19 cases per 100 residents (an additional 10.97 and 14.81 cases, respectively) compared with those without such shortages. Thus, during the prevaccine phase of the pandemic, postacute care provided to patients who were discharged from a hospital while still requiring transmission-based precautions due to COVID-19 could have served as a vector for early outbreaks in SNFs. 

Officer–Involved Killings of Unarmed Black People and Racial Disparities in Sleep Health Venkataramani AS , Bair EF, Bor J, Jackson CL, Kawachi I, Lee J, Papachristos A, Tsai AC.  JAMA Internal Medicine  184(4), April 2024, pp. 363–373.

Short sleep duration is a risk factor for many chronic and mental health conditions, and is more prevalent among Black than among White individuals in the US. Unequal exposure to police violence is examined in this study as a contributor to differences in short sleep, using data from three sources: the US Behavioral Risk Factor Surveillance System (BRFSS, 2013, 2014, 2016, and 2018), the American Time Use Survey (ATUS, 2013–2019), and the Mapping Police Violence database. An exposure was quantified as the occurrence of any police killing of an unarmed Black person in the state, county, or commuting zone of the survey respondent’s residence in each of the four 90-day periods prior to interview, or occurrence of a highly public, nationally prominent police killing of an unarmed Black person anywhere in the US during the 90 days prior to interview. Short sleep (<7 hours) was reported by 46 percent of Black respondents to the BRFSS and 33 percent of White respondents, and for very short sleep (<6 hours) the rates were 18 and 10 percent respectively. Difference-in-differences analyses found statistically significant increases in the probability of short sleep and very short sleep among Black respondents to the BRFSS and the ATUS when officers killed an unarmed Black person in their state of residence during the first two 90-day periods prior to interview. Magnitudes were larger in models using exposure to a nationally prominent police killing occurring anywhere in the US. These effects were not found for officer-involved killings of armed Black people, or among White respondents (for killings of unarmed Black or White individuals). Estimates were equivalent to 7 percent to 16 percent of the sample disparity between Black and White individuals in short sleep and 13 percent to 30 percent of the disparity in very short sleep.

The Impact of Telemedicine on Medicare Utilization, Spending, and Quality, 2019–22 Nakamoto CH,  Cutler DM , Beaulieu ND, Uscher-Pines L, Mehrotra A.  Health Affairs  43(5), May 2024, pp. 691–700.

Clinical and Healthcare Use Outcomes after Cessation of Long Term Opioid Treatment Due to Prescriber Workforce Exit: Quasi-experimental Difference-in-Differences Study Sabety AH , Neprash HT, Gaye M, Barnett ML.  BMJ  385, May 2024.

Loss of Medicaid Coverage during the Renewal Process Dague L , Myerson R.  JAMA Health Forum  5(5), May 2024.

Recreational Marijuana Laws and Teen Marijuana Use, 1993–2021 Anderson DM , Fe HT, Liang Y, Sabia JJ.  JAMA Psychiatry,  April 2024.

Comparison of Hospital Mortality and Readmission Rates by Physician and Patient Sex Miyawaki A,  Jena AB , Rotenstein LS, Tsugawa Y.  Annals of Internal Medicine  177(5), April 2024, pp. 598–608.

State Paid Sick Leave Mandates Associated with Increased Mental Health Disorder Prescriptions among Medicaid Enrollees Maclean JC , Golberstein E, Stein B.  Health Affairs Scholar  2(5), April 2024.

Measuring Local-Area Racial Segregation for Medicare Hospital Admissions Akré EL, Chyn D, Carlos HA, Barnato AE,  Skinner J .  JAMA Network Open  7(4), April 2024.

Paternal Depression in the Postpartum Year and Children’s Behaviors at Age 5 in an Urban US Birth Cohort Schmitz K, Jimenez ME,  Corman H , Noonan K, Reichman NE.  PLOS ONE  19(4), April 2024.

How Does Telehealth Expansion Change Access to Healthcare for Patients with Different Types of Substance Use Disorders? Tilhou AS, Burns M, Chachlani P, Chen Y, Dague L , . Substance Use & Addiction Journal 45(3), March 2024, pp. 473–485.

Public versus Private Care in the Military Health System: Evidence from Low Back Pain Patients Leggett CG, Schmidt RO,  Skinner J , Lurie JD, Luan WP.  Military Medicine,  March 2024.

Effects of Medicaid Expansions on Coverage, Prenatal Care, and Health among American Indian/Alaska Native Women Strully KW,  Chatterji P , Liu H, Han S, Schell L.  Health Affairs  43(3), March 2024, pp. 344–353.

Racial and Gender Disparities in the Effect of New Drug Approvals on US Cancer Mortality Lichtenberg FR .  Academia Medicine , February 2024.

Chronic Health Conditions and Adolescents’ Social Connectedness James C,  Corman H , Noonan K, Reichman NE, Jimenez ME.  American Journal of Orthopsychiatry  94(3), January 2024, pp. 235–245.

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• Published: 10 December 2021

11 clinical trials that will shape medicine in 2022

• Carrie Arnold 1  

Nature Medicine volume  27 ,  pages 2062–2064 ( 2021 ) Cite this article

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Nature Medicine asks leading researchers to name their most anticipated clinical trial for 2022, from CRISPR and psilocybin to oncolytic viruses.

Despite the ongoing disruptions from COVID-19, 2021 saw some major results from clinical trials.

2022 looks to be a bumper year too, so Nature Medicine asks leaders in their field what they think will be the most important clinical trial results in the coming year.

Universal influenza vaccines

Neil P. King: Like existing vaccines against seasonal influenza, FluMos-v1 (Table 1 ) is meant to create antibodies to several different influenza virus strains. Unlike existing influenza shots, the new vaccine uses a mosaic nanoparticle immunogen that co-displays antigens from multiple influenza viruses on the same nanoparticle surface. In our pre-clinical work, we found that the nanoparticle immunogen elicited a greater diversity of protective antibodies than did current vaccines against influenza. We are particularly interested in learning about how the vaccine will interact with immune systems that have encountered influenza viruses or vaccines before. This pre-existing immunity may alter vaccine performance, and this is something we couldn’t get a read on pre-clinically.

Neil P. King is an assistant professor at the Institute for Protein Design, University of Washington .

Antisense oligonucleotides for Huntington’s

Claudia Testa: This trial for Huntington’s disease closed early, due to futility, in March 2021. Despite this setback, the full results are not yet out and are critically important. This antisense oligonucleotide (ASO) targets both mutant and wild-type huntingtin mRNA, which is completely different from other ASOs on the market. The trial results will be a first real look at fundamental aspects of RNA-lowering strategies, including how the drug impacted various biomarkers across hundreds of participants. The results could transform the clinical-trial landscape for other conditions treatable by ASOs. This trial perfectly illustrates how well-designed and well-run trials do not entirely fail — the efforts of the trial participants will be crucial to accelerating the timeline to a safe and effective Huntington’s disease–modifying treatment, even though the trial outcome will not lead to an agent approved by the US Food and Drug Administration.

Claudia Testa is the division chief of Precision Medicine and Neurogenetics, UNC Chapel Hill .

CRISPR for amyloidosis

Julian Gillmore: I have been looking after patients with transthyretin amyloidosis for 25 years, and until 5 years ago, I just watched their health decline. Some years ago, gene silencers became available and improved outcomes for patients, which was a very substantial advance. But patients on gene silencers need lifelong repeated treatments, and although most patients’ disease stabilizes, some continue to deteriorate. Preliminary data from this trial have shown for the first time that CRISPR-based gene therapies can be administered by intravenous infusion to edit a specific gene in liver cells. Full results are expected next year. This new gene-editing therapy offers patients the prospect of meaningful clinical improvements after a single dose of the drug.

Julian Gillmore is a reader in medicine and an honorary consultant nephrologist at Royal Free London Hospital .

Oral therapy for kala-azar

Monique Wasunna: I am excited to see the results of our phase 3 clinical trial looking for a better regimen to treat visceral leishmaniasis, also known as ‘kala-azar’. This trial tests new combination regimens of miltefosine and paromomycin in Ethiopia, Sudan, Kenya and Uganda in eastern Africa. The aim is to replace the toxic sodium stibogluconate component of the current injectable treatment for kala-azar with the orally administered miltefosine. This is a much-needed improvement patients have been waiting for and we have been working toward for years, as oral treatments can be administered at local health centers, closer to affected communities. Patients diagnosed with kala-azar will die if they are not treated. The new, less toxic and more easily administered regimen will also be good for children, who are disproportionately affected by visceral leishmaniasis. Children under 12 represented 60% of clinical-trial patients, and preliminary results show a promisingly high efficacy for them.

Monique Wasunna is the director of the Drugs for Neglected Diseases Initiative Africa Regional Office, Nairobi .

Targeting sigma 1 receptor in neurodegenerative disease

Blair Leavitt: There are several ongoing studies of pridopidine that target disease progression in neurodegenerative disorders. Pridopidine is an oral small-molecule therapeutic that selectively targets the sigma 1 receptor and has a well-established safety record from previous human clinical trials. Over the past several years, a role for sigma 1 receptor in neurodegeneration has been established, and there has been quite a bit of promising pre-clinical data to suggest that pridopidine has protective effects in Huntington’s disease and in other neurodegenerative disorders such as amyotrophic lateral sclerosis. Pridopidine is the only drug being tested in a phase 3 clinical trial that will assess the clinical progression of Huntington’s disease. There is an important phase 2/3 trial of pridopidine in amyotrophic lateral sclerosis that is also currently underway, and the results of these ongoing studies are eagerly awaited by the community.

Blair Leavitt is a consulting neurologist and director of research at the University of British Columbia Centre for Huntington’s Disease .

Exon skipping in muscular dystrophy

Kevin M. Flanigan: A small number of boys with Duchenne muscular dystrophy have a disease caused by a duplication of exon 2 of the DMD gene (which encodes dystrophin). This exon duplication can be removed through the use of an adeno-associated virus 9–based vector, which carries copies of the non-coding small nuclear RNA U7 with a sequence directed toward splice donor and acceptor sites within the duplicated exon. This exon-skipping approach is a first in-human trial and results in exclusion of a copy of DMD exon 2, and expression of the full-length, wild-type dystrophin. Preliminary data have been presented showing successful expression, for the first time, of full-length dystrophin in response to this viral gene therapy. There are four more gene therapies for Duchenne muscular dystrophy being tested, but each of these requires delivery of a gene encoding an engineered microdystrophin, which is not present in nature and which is expected to result in substantial functional improvement.

Kevin M. Flanigan is an attending neurologist at Nationwide Children’s, the Robert F. and Edgar T. Wolfe Foundation Endowed Chair in Neuromuscular Research and a professor of pediatrics and neurology at The Ohio State University College of Medicine .

Psychedelic therapy

David Nutt: Psilocybin, a psychedelic derived from fungi, is showing great promise in the treatment of mental-health conditions. I’m excited about this psilocybin trial for three main reasons. First, it tackles the huge problem of depression that is resistant to existing treatments. Second, it’s a multi-center and multi-country trial, which will let researchers test the psychedelic therapy in a diverse group of people. Finally, it will also give some readouts on the dose–effect relationship, as they are testing three greatly differing doses of psilocybin, at 1 mg, 10 mg and 25 mg.

David Nutt is the Edmond J. Safra Professor of Neuropsychopharmacology and director of the Neuropsychopharmacology Unit in the Division of Brain Sciences, Imperial College London .

Oncolytic viruses

Yoshikazu Yonemitsu: This is a phase 1 open-label study of ASP9801 administered by intratumoral injection in patients with advanced or metastatic solid tumors. ASP9801 is a genetically engineered oncolytic vaccinia virus that expresses two cytokines, IL-7 and IL-12, that help stimulate antitumor immune responses. ASP9801 was effective in multiple immunocompetent mouse models and showed promising results in both directly treated tumors and distant tumors. This study will for the first time assess the safety, tolerability and antitumor activity of this oncolytic vaccinia virus in patients with cancer.

Yoshikazu Yonemitsu is a professor in the Graduate School of Pharmaceutical Sciences, Kyushu University .

Long-lasting monoclonal antibodies for RSV

Ruth A. Karron: Respiratory syncytial virus (RSV) is a leading cause of severe respiratory infections in infants and children, but there is currently no vaccine against this. Nirsevimab is a monoclonal antibody that targets the F protein on the virus’s surface. It carries a YTE mutation, which extends its half-life; this means that a single dose of the antibody could protect an infant throughout their first RSV season. This is a big improvement over the current monoclonal antibody to RSV, palivizumab, which lasts about a month and therefore is used only for high-risk infants. The final results will be interesting, because they will determine if vaccine-policymaking bodies are willing to approve a monoclonal antibody, rather than a vaccine, as a prophylactic product.

Ruth A. Karron is a professor in the Department of International Health, Johns Hopkins Bloomberg School of Public Health, the director of the Center for Immunization Research, and the founding director of the Johns Hopkins Vaccine Initiative .

Adjuvant immunotherapy for breast cancer

Jennifer Litton: People with triple-negative breast cancer have fewer treatment options than others with this type of cancer. Although pembrolizumab (marketed as Keytruda) was originally developed to treat advanced melanoma, it has since been approved to treat a variety of cancers. This trial will answer important questions about the use of adjuvant pembrolizumab for patients with high-risk triple-negative breast cancer. The Keynote 522 study provided important information about the use of neoadjuvant pembrolizumab for these patients, but it also left many questions about the length of treatment and the role of this drug in the post-operative space. This study will shed important light on these questions as a guide for moving forward.

Jennifer Litton is the vice president of clinical research and a professor in the Breast Medical Oncology department, MD Anderson Cancer Center .

COVID-19 treatments

Edward Mills: The TOGETHER trial has been groundbreaking, as it has evaluated nine different interventions in 2021 and managed to drop six of them very early due to lack of efficacy, thereby saving time and money. Most importantly, and what I am most excited about, TOGETHER is the first trial to demonstrate that a low-cost repurposed medicine, fluvoxamine, can prevent hospitalizations for COVID-19. We are now moving into combinations of drugs, and I think that is where the future of COVID-19 treatment is. In the next year, we should learn about the efficacy of several combinations, including fluvoxamine or fluoxetine plus an inhaled steroid, as well as fluvoxamine plus the direct-acting antiviral molnupiravir.

Edward Mills is a professor of health sciences at McMaster University .

Editorial note These interviews have been edited for length and clarity.

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Arnold, C. 11 clinical trials that will shape medicine in 2022. Nat Med 27 , 2062–2064 (2021). https://doi.org/10.1038/s41591-021-01601-5

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I N APRIL HILARY CASS , a British paediatrician, published her review of gender-identity services for children and young people, commissioned by NHS England. It cast doubt on the evidence base for youth gender medicine. This prompted the World Professional Association for Transgender Health ( WPATH ), the leading professional organisation for the doctors and practitioners who provide services to trans people, to release a blistering rejoinder. WPATH said that its own guidelines were sturdier, in part because they were “based on far more systematic reviews”.

Systematic reviews should evaluate the evidence for a given medical question in a careful, rigorous manner. Such efforts are particularly important at the moment, given the feverish state of the American debate on youth gender medicine, which is soon to culminate in a Supreme Court case challenging a ban in Tennessee. The case turns, in part, on questions of evidence and expert authority.

Court documents recently released as part of the discovery process in a case involving youth gender medicine in Alabama reveal that WPATH ’s claim was built on shaky foundations. The documents show that the organisation’s leaders interfered with the production of systematic reviews that it had commissioned from the Johns Hopkins University Evidence-Based Practice Centre ( EPC ) in 2018.

From early on in the contract negotiations, WPATH expressed a desire to control the results of the Hopkins team’s work. In December 2017, for example, Donna Kelly, an executive director at WPATH , told Karen Robinson, the EPC ’s director, that the WPATH board felt the EPC researchers “cannot publish their findings independently”. A couple of weeks later, Ms Kelly emphasised that, “the [ WPATH ] board wants it to be clear that the data cannot be used without WPATH approval”.

Ms Robinson saw this as an attempt to exert undue influence over what was supposed to be an independent process. John Ioannidis of Stanford University, who co-authored guidelines for systematic reviews, says that if sponsors interfere or are allowed to veto results, this can lead to either biased summaries or suppression of unfavourable evidence. Ms Robinson sought to avoid such an outcome. “In general, my understanding is that the university will not sign off on a contract that allows a sponsor to stop an academic publication,” she wrote to Ms Kelly.

Months later, with the issue still apparently unresolved, Ms Robinson adopted a sterner tone. She noted in an email in March 2018 that, “Hopkins as an academic institution, and I as a faculty member therein, will not sign something that limits academic freedom in this manner,” nor “language that goes against current standards in systematic reviews and in guideline development”.

Not to reason XY

Eventually WPATH relented, and in May 2018 Ms Robinson signed a contract granting WPATH power to review and offer feedback on her team’s work, but not to meddle in any substantive way. After wpath leaders saw two manuscripts submitted for review in July 2020, however, the parties’ disagreements flared up again. In August the WPATH executive committee wrote to Ms Robinson that WPATH had “many concerns” about these papers, and that it was implementing a new policy in which WPATH would have authority to influence the EPC team’s output—including the power to nip papers in the bud on the basis of their conclusions.

Ms Robinson protested that the new policy did not reflect the contract she had signed and violated basic principles of unfettered scientific inquiry she had emphasised repeatedly in her dealings with WPATH . The Hopkins team published only one paper after WPATH implemented its new policy: a 2021 meta-analysis on the effects of hormone therapy on transgender people. Among the recently released court documents is a WPATH checklist confirming that an individual from WPATH was involved “in the design, drafting of the article and final approval of [that] article”. (The article itself explicitly claims the opposite.) Now, more than six years after signing the agreement, the EPC team does not appear to have published anything else, despite having provided WPATH with the material for six systematic reviews, according to the documents.

No one at WPATH or Johns Hopkins has responded to multiple inquiries, so there are still gaps in this timeline. But an email in October 2020 from WPATH figures, including its incoming president at the time, Walter Bouman, to the working group on guidelines, made clear what sort of science WPATH did (and did not) want published. Research must be “thoroughly scrutinised and reviewed to ensure that publication does not negatively affect the provision of transgender health care in the broadest sense,” it stated. Mr Bouman and one other coauthor of that email have been named to a World Health Organisation advisory board tasked with developing best practices for transgender medicine.

Another document recently unsealed shows that Rachel Levine, a trans woman who is assistant secretary for health, succeeded in pressing wpath to remove minimum ages for the treatment of children from its 2022 standards of care. Dr Levine’s office has not commented. Questions remain unanswered, but none of this helps WPATH ’s claim to be an organisation that bases its recommendations on science. ■

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Coupling food pantry access with primary care offers new recipe for preventative medicine, Northeastern research says

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“Primary care partnerships with food pantries help stabilize health outcomes,” says John Lowrey, assistant professor of supply chain and information management at Northeastern University.

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In a world awash with mass-produced, highly processed high-caloric food, the cheapest option is often an unhealthy one . Add rampant wealth inequality , mix in the many factors plaguing the U.S. health care system, and you have a recipe for obesity and other diet-related illnesses.

But a new study from Northeastern University shows that adding regular food pantry access to primary clinical care can stop the progression of some diet-related chronic illnesses. 

“Partnerships with food banks could be the next preventative medicine model,” says John Lowrey, assistant professor of supply chain and information management at Northeastern’s D’Amore-McKim School of Business and Bouvé College of Health Sciences. “Primary care partnerships with food pantries help stabilize health outcomes.”

Approximately 44 million Americans are food insecure, meaning they don’t have enough food to eat or don’t have access to healthy food. 

At the same time, two in five American adults, and one in five American children, are obese . 

To look into both issues, Lowrey partnered with the Mid-Ohio Farmacy, a collaboration across the Mid-Ohio Food Collective, a network of over 650 affiliated food pantries and a large federally qualified health center. The health center offers primary and preventative health care services across eight free clinics that are co-located with the food pantries. Patients were screened for food insecurity during their clinic visit and, if positive, were referred to the Farmacy.

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Lowrey and colleagues found that, overall, those who made at least one visit to the food pantry a year after referral stabilized their body mass index. However, patients who did not visit the pantry over that time period saw their BMI increase, according to the research. 

There is also a possible “dose-response relationship” between the frequency of food pantry visits and health benefits,” Lowrey says. Those who visited the food pantry at least 11 times in the year and had multiple comorbidities had a greater reduction in BMI and reduction in blood sugar levels, according to the research.

“We find that they have the greatest change in health outcomes,” Lowrey says.

Finally, the data suggest that these regular food pantry users with comorbidities changed their behaviors following referral to emphasize their health.

For one, they visited pantries more consistently and traveled further to visit pantries, for instance, and visited more food pantry locations.

“The way they were using the pantry system suggests more “food is medicine” behaviors, Lowrey says. 

The food insecure patients who used the pantry most often also changed their health care visits. 

Lowrey explains that research has found a “treat or eat” tradeoff, where the food secure generally visit health care providers earlier in the week than the food insecure. This suggests the former group can better prioritize health care needs when they don’t have to worry about food.  

But in the study, the pattern shifted following referral.

“By looking at clinic visit patterns by food security and by compliance status, we see that compliers after referral have visit patterns that are more suggestive that they are like food secure patients,” Lowrey says. “This suggests that patients are prioritizing their health care visits earlier in the week, even if these visits compete with occupational demands.”

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Med schools face a new obstacle in the push to train more black doctors.

Lauren Sausser

Jerrian Reedy, left, a student at the University of Mississippi School of Medicine, assists Dorothy Gray, a student at Northside High School in the Mississippi Delta, as she practices intubation in a simulation lab. Gray, who is interested in pursuing a career in the mental health care field, attended the University of Mississippi School of Medicine’s annual African American Visit Day in April. Lauren Sausser for KFF Health News hide caption

JACKSON, Miss. — Jerrian Reedy was 9 when his father was admitted to the hospital in Hattiesburg, about two hours northeast of New Orleans, after sustaining three gunshot wounds. Reedy recalled visiting his dad in the intensive care unit that summer in 2009, even though children weren’t typically permitted in that part of the hospital.

“Just seeing him laid up in bed, in a hospital bed, it was traumatizing, to say the least,” Reedy said.

His father died within a week of being admitted, in the middle of a nine-month span when Reedy also lost an aunt and a grandmother. “They say death comes in threes,” he said.

That chain of events prompted him to pursue a career in medicine, one that might help him spare other children from losing loved ones too soon.

Fifteen years later, Reedy has completed his first year at the University of Mississippi School of Medicine — a remarkable feat, and not only because his career path was born of grief and trauma. Reedy is among a small share of Black medical school students in a state where nearly four in 10 people — but only one in 10 doctors — identify as Black or African American.

Of the 660 medical school students enrolled in the same four-year program as Reedy, 82 students, or about 12%, are Black.

Black Americans expect to face racism in the doctor's office, survey finds

Medical schools around the country are trying to recruit Black, Hispanic, and Native American students, all of whom remain disproportionately underrepresented in the field of medicine. Research has shown that patients of color prefer seeing doctors of their own race — and some studies have shown that Black patients who see Black doctors experience better health outcomes.

But a recent swell of Republican opposition threatens to upend those efforts, school administrators say, and could exacerbate deep health disparities already experienced by people of color.

Since 2023 — the year the Supreme Court voted to outlaw affirmative action in higher education — more than two dozen states, including Alabama, Florida, Mississippi, North Carolina, and Texas, have introduced or passed laws to restrict or ban diversity, equity, and inclusion, or DEI, programs.

“I don’t expect this movement of anti-DEI legislation to slow down or stop at all,” said Anton Gunn, a health care consultant and former head of the Office of External Affairs at the U.S. Department of Health and Human Services. “And it likely will exacerbate if Donald Trump gets the opportunity to be president of the United States again.”

Diversity programs face pushback

In 2023, Florida and Texas became the first states to pass laws that banned DEI efforts in higher education. Several other states, including Idaho, North Carolina, and Wyoming, passed laws targeting such programs this year.

In Mississippi, state Rep. Becky Currie and state Sen. Angela Burks Hill, both Republicans, introduced separate bills that would have restricted how colleges and universities could spend money on DEI initiatives. Both bills died in legislative committees and were not brought before the 2024 legislature for a vote.

Health Care

Following in her mom's footsteps, a doctor fights to make medicine more inclusive.

In a statement, Hill said that Mississippi needs more doctors of all kinds, not just more Black doctors, and that she thinks money spent on DEI salaries and programs should be reallocated to initiatives benefiting all students.

“Qualifications should determine who gets into medical school not color or socioeconomic status,” she said. “Can’t we just be happy with more highly qualified doctors no matter their skin color? I thought a color blind society was the goal.”

Nationally, the movement to ban DEI programs has broad conservative support.

Jay Greene, a senior research fellow at the Heritage Foundation, a conservative think tank, said he believes diversity programs “fail for a hundred reasons.” He cited research he conducted with a conservative medical advocacy group called Do No Harm refuting the premise that access to Black doctors improves health outcomes among Black patients.

“That doesn’t mean there’s no potential benefits for having greater diversity in the doctor workforce,” Greene said. Having more Black doctors, for example, might encourage more Black children to consider a career in health care, he said. “But that benefit is not health outcomes.”

Meanwhile, school administrators are closely watching the progress of such laws.

In March, the University of Florida eliminated all DEI programs and terminated jobs related to those efforts. In Alabama, lawyers and school leaders are grappling with a bill signed the same month by Republican Gov. Kay Ivey that bans DEI programs in public schools, state agencies, and universities starting Oct. 1.

“We have to be very, very careful,” said Richard deShazo, who teaches at the University of Alabama’s Marnix E. Heersink School of Medicine in Birmingham and used to chair a committee that raised money for Black medical school students.

“You cannot raise money for Black kids. You have to raise money for medical students,” he said.

Kids of color get worse health care across the board in the U.S., research finds

A bitter history.

A shortage of Black doctors isn’t unique to Mississippi. The same story could be told in many other places, especially across the South, where more than half of all Black Americans live and where health outcomes regularly rank among the worst in the United States.

But a look at Mississippi, one of the unhealthiest states in the country, shows how the roots of systemic racism continue to shape the nation’s health care workforce.

“A lot of the Black physicians in the state have a bitter taste in their mouth about our medical school,” said Demondes Haynes, associate dean of medical school admissions at the University of Mississippi Medical Center, where he graduated in 1999 as one of four Black students in his class.

An estimated 1.1 million Black people live in Mississippi, where there are fewer than 600 Black doctors. Research suggests health outcomes would improve if there were more, counter to what Greene concluded. One study published last year in the medical journal JAMA Network Open found that life expectancy was longer among Black patients in counties with higher ratios of Black primary care physicians. In a study based in Oakland, Calif., that involved more than 1,300 Black men, those who were assigned a Black doctor were more likely to agree to screening tests for diabetes, cholesterol and other health concerns, according to the findings published in 2018 by the National Bureau of Economic Research.

“We absolutely are not saying every Black patient has to have a Black doctor,” Haynes said. But because the patient population in Mississippi is diverse, "they should at least have the right to say, ‘This is what I want,’” he added.

However, most Black patients aren’t afforded that choice. Nearly two dozen of Mississippi’s 82 counties had no Black doctors, while four counties claimed no doctors at all, according to a physician workforce report published by the state in 2019.

For more than a century, dating to its founding in the mid-1800s, the University of Mississippi didn’t admit Black students — and that policy applied to its medical school. In 1972, nearly 10 years after the Civil Rights Act of 1964 banned racial segregation in higher education, the first Black physician graduated from the medical school in Jackson. Even then, very few Black students were admitted to study medicine there each year.

Before the federal government banned the school from rejecting Black applicants because of their race, aspiring Black doctors who applied were diverted to one of the historically Black colleges and universities, or HBCUs, such as Meharry Medical College in Nashville, Haynes said.

Many older Black physicians in Mississippi still remember getting those rejection letters, he said, pointing out composite photographs of physician graduates that line the walls of the medical school building in Jackson. Many of the earliest composites, dating to the 1950s, showcase classes of all-white, and almost all-male, students.

“Mississippi history — everybody remembers it,” Haynes said. “And those people who experienced it, it’s hard for them.”

‘Shaping the possibilities’

On a damp Saturday morning in mid-April, 17-year-old Dorothy Gray, a high school junior, stepped up to a hospital bed at the medical school in Jackson to intubate a mock patient in a simulation lab.

Gray was one of more than 100 high school and college students who attended the University of Mississippi School of Medicine’s annual African American Visit Day, established more than 10 years ago to foster interest among prospective Black students. The administrators, who also host special visiting days for Hispanic and Native American students, said anyone, regardless of race or ethnicity, may attend. They acknowledge that most attendees won’t become doctors, and their purpose isn’t to extend preferential treatment to minority applicants.

Marlee Washington (left) and Jon Trayvious attend the African American Visit Day at the University of Mississippi School of Medicine. Trayvious, a recent graduate of Northside High School in Shelby, Mississippi, inspects a human lung in a classroom at the medical school. Lauren Sausser for KFF Health News hide caption

“This is about shaping the possibilities of what could be,” said Loretta Jackson-Williams, the school’s vice dean for medical education. “These kids are at that precipice where they can choose to do something that's really hard for their future or they can choose an easier pathway. That choice doesn't come about overnight.”

Besides African American Visit Day, medical school leaders in Mississippi also offer a test prep program for applicants from underrepresented backgrounds who have been rejected from medical school.

The school recently identified 16 applicants, 12 of whom are Black, who were not accepted to the medical school during the last admissions cycle because their MCAT scores were too low. This year, those applicants will receive a test prep course designed by The Princeton Review — free of charge — and will have a chance to meet with administrators to learn how their medical school applications might be strengthened.

“So many students have never had someone tell them, ‘You can do this. I believe you can do this,’” said Dan Coleman, the medical school’s outreach director.

For Jerrian Reedy, who wants to become an orthopedic surgeon, the path to medical school was years in the making. He took advantage of the University of Mississippi Medical Center’s PROMISE program — short for Promoting Recruitment Opportunities in Medicine with Individual Study Experiences — which assures acceptance to students from disadvantaged backgrounds who meet certain eligibility requirements, including a 3.0 GPA in their undergraduate science classes.

During his sophomore year as an undergraduate, Reedy saw an opportunity to learn more about medical school when Haynes, the assistant dean, visited the Ole Miss campus in Oxford to interview students.

“I saw some open slots, put my name down,” he said. “The rest is history.”

KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF — an independent source for health policy research, polling, and journalism.

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• Mississippi
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