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How one research pioneer plans to revitalize gene therapy

August 22, 2024
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Morning Rounds Writer and Podcast Producer
Good morning! We've got a risk-filled issue for you today, as my editor called it. And women seem to be particularly at risk. With that, read on, and I hope you all have a nice, safe Thursday.

chronic disease

What we know about long Covid in kids

Adobe

It's estimated that up to 5.8 million kids in the U.S. could have long Covid. But what does that look like? There's still so much about the chronic condition that scientists don't understand. But a study published yesterday in JAMA analyzed how its symptoms present in kids and found the experience varies by age groups.

For younger school-age kids (6 to 11 years old) with symptoms, headaches and abdominal pain were common, as was trouble with memory, focus, and sleep. Adolescents (12 to 17) suffered more from daytime fatigue, sleepiness, trouble with memory and focus, and body, muscle, or joint pain.

Figuring out why some kids experience long Covid differently depending on their age is the next question that researchers are hoping to answer. "Children of these different ages are growing, their brains are developing, there's lots of changes," said Rachel Gross, a pediatrician and study author. Read more from STAT's Liz Cooney on the study and researchers' lingering questions about long Covid.


biotech

Can this research pioneer revitalize gene therapy after the 'great abandonment'? 

If you're a reader of STAT's biotech newsletter, The Readout, you probably already know the name Jim Wilson. If you're not, here's a primer: He is the guy in gene therapy. He's been a pioneer in the field for decades, focusing on treatments for rare disease. His work at the University of Pennsylvania's Gene Therapy Program was the basis for dozens of clinical trials and three gene therapies approved by the FDA. (Two years ago, STAT published an investigation into the toxic and abusive workplace culture that current and former employees said existed in Wilson's program.)

Now, Wilson is leaving Penn to found a pair of companies focused on developing gene therapies and offering supportive services to other biotechs. It's a big move that has caught the attention of other academic experts in the field, who say their research budgets have been reduced. STAT's Jonathan Wosen spoke to Wilson and other experts about the move and what it represents for the relationship between academia and industry.


health care workers

Female doctors are at increased risk for suicide

That's both compared to the general public, and compared to male doctors, according to a new study published yesterday in The BMJ. Resarchers performed a systematic review of 39 studies that included data on more than 3,300 suicides in 20 countries between 1935-2020. Male doctors had a slightly increased risk of suicide compared to the general population. But female doctors had a 76% higher risk.

The bright side: For all doctors, the risk has decreased over time. But there was a lot of variability in the data among different populations, likely due to differences in training and work environments, the study authors wrote. 

While there has long been increased focus on high suicide rates among doctors, recent research has found higher rates among all female health care workers, including nurses. "There's something about the gender roles and occupational experiences of women as health care workers that's putting them at risk of suicide in a way that is not putting the men so much at risk," researcher Mark Olfson told STAT's Nalis Merelli last fall. 

If you or someone you know may be considering suicide, contact the 988 Suicide & Crisis Lifeline: call or text 988 or chat 988lifeline.org. For TTY users: Use your preferred relay service or dial 711 then 988.



first opinion

Diversity matters in preclinical research, too

Adobe 

The FDA started reporting on racial representation in clinical trials in 2015. The idea was that raising awareness of the lack of diversity would drive change. Years later, the FDA is still reporting, and the reality hasn't shifted. But in a First Opinion essay, life sciences consultant Sophie Zaaijer points to a rarely discussed representation problem even earlier in the pipeline: preclinical risk assessments. 

These early evaluations are often based on human cell lines. And while the first and most famous cell line came from a Black woman (Henrietta Lacks), the majority of human cell lines come from people of European descent. "This ongoing monochromatic approach in preclinical R&D fails to capture the rich tapestry of human genetic variation, painting a portrait of humanity with a limited palette," Zaaijer writes. Read more


cardiovascular health

Women are more likely to die of postoperative heart complication

When it comes to the most common complication after heart surgery, atrial fibrillation, a new study found that women were more likely than men to die from the condition. The research, published yesterday in JAMA Network Open, is based on data from more than 21,500 patients who developed postoperative atrial fibrillation after open heart surgery at two care centers in Massachusetts.

Postoperative atrial fibrillation occurs when a patient experiences an irregular, rapid heart rhythm. Some previous research has found that men have a greater risk of developing the complication than women, while other studies have found no difference. The lack of clarity may be due to an overall small sample size, the authors wrote. It also remains unclear why any differences in incidence or survival of the condition may differ by sex.


disability

New Alzheimer's drugs may be dangerous for this at-risk group

About 30% of people with Down syndrome in their 50s have Alzheimer's — it's the population with the highest prevalence of the disease. But anti-amyloid drugs like lecanemab and donanemab still largely aren't prescribed to them. 

Ultimately, that may be for the best. In a study testing lecanemab on post-mortem brain tissue published this week, researchers determined that the population might face higher risk factors. 

The conclusion comes more than a year after the FDA approval of the drug. (Donanemab was approved earlier this summer.) People with Down syndrome have historically been excluded from Alzheimer's clinical trials. More than 18,000 people have participated in trials for these types of drugs, but none of them had Down syndrome. Read more from STAT's Timmy Broderick on what makes people with this condition more susceptible, and what researchers are trying to do about it.


More around STAT
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What we're reading

  • LGBTQ+ asylum seekers detail harsh conditions and abuse in ICE custody, The 19th

  • Merck Ebola vaccine Ervebo shown to offer substantial protection, STAT
  • Yes there is famine in Sudan. So why isn't 'famine' being declared? NPR
  • Gene therapy is in crisis. For nine hours, the field's leading minds looked for a solution, STAT

Thanks for reading! More tomorrow,


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