Breaking News

Polio's missed goal, a psychedelic for PTSD, & evidence for gender-affirming care

September 15, 2023
Reporter, Morning Rounds Writer
Good morning. Today we have a mix of good news (on a gene therapy for one boy), potentially good news (on a PTSD treatment trial), and not-yet-good news (on polio eradication). In any case, happy Friday.

global Health

Another goal for ending polio is slipping out of sight

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Rizwan Tabassum/AFP via Getty Images

Missing deadlines for ending polio transmission is nothing new, but the most recent report about eradication efforts is particularly discouraging. The Global Polio Eradication Initiative had set 2023 as its goal for stamping out both wild polio viruses and vaccine-derived viruses that together have paralyzed children for centuries. While there have been only been seven wild polio cases recorded this year — in Afghanistan and Pakistan — the clock will run out in 2023 before ongoing spread is stopped, the polio program's Independent Monitoring Board concluded. 

"The complexity of the continuing barriers to interrupting wild poliovirus transmission is too great to be resolved in an 18-week period," the committee said. It was blunt in its assessment, blaming the initiative for "a turbulent journey marred by rigid attitudes, missed opportunities, lack of foresight, and an inability to adapt swiftly to evolving circumstances." STAT's Helen Branswell explains.


good news 

A 6-year-old boy gets his gene therapy after all

Here's a happy development, brought to us by STAT's Jason Mast: Cash Huber, a 6-year-old Duchenne muscular dystrophy patient, received a gene therapy from Sarepta Therapeutics this week. It was notable because, as STAT wrote last month, Huber's insurance, Highmark Delaware, initially denied him access to the $3.2 million drug, deeming it "experimental." It was an early test case in how insurers would approach a medicine that might alter the lives of patients with the fatal muscle-wasting disease but has only limited data and a sky-high price tag.  

But after a public campaign from his family and advocates, Highmark reversed its decision, putting it in line with other payers, which have largely chosen to cover the treatment. By that point, Cash had technically aged out — it's approved only for 4- and 5-year-olds —  but doctors were still willing to administer it.  The effects shouldn't kick in for a few weeks, but the boy's dad, Phil Huber, reports Cash is doing well so far. Highmark said it could not comment, citing patient privacy.


mental Health

MDMA continues to show promise in treating PTSD

A new study testing the psychedelic MDMA as a treatment for PTSD in combination with psychotherapy reports two wins. First, the therapy improved symptoms after 18 weeks to a greater degree than a placebo used with psychotherapy. Second, it did so in a racially and ethnically diverse population, which has higher rates of PTSD but lower representation in research studies. The results, described in Nature Medicine, echo previous research, possibly bringing the psychedelic closer to FDA approval.

"The results are exciting," Thomas Insel, a neuroscientist and former director of the National Institute of Mental Health, told STAT's Annalisa Merelli. He was not involved with the study. "These effects are notably greater than reported for the two SSRIs that have been approved by FDA for PTSD."  Insel noted that the placebo group did improve as well, perhaps due to psychotherapy — something that could raise doubts for the FDA about MDMA's effectiveness. Read more.



Closer Look

The debate over what constitutes 'enough' evidence on gender-affirming care

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It's been an unfortunate record year for anti-LGBTQ+ efforts, particularly when it comes to gender-affirming care. Attacks often hinge on the argument that the existing evidence — made up mainly of observational studies — is insufficient, and the care is "experimental." But the viability of "high-quality" evidence like randomized controlled trials is often limited by both practical and ethical concerns.

"I think the people who are calling for RCTs on whether or not we should provide gender-affirming care are simply not serious," said Ayden Scheim, an assistant professor of epidemiology at Drexel University's Dornsife School of Public Health. "Not serious in terms of their understanding of basic principles of epidemiology and public health research." STAT's Theresa Gaffney has more.


global health

More striking ProMED staffers have been fired 

The number of remaining staff of the global disease surveillance program ProMED continues to shrink, STAT's Helen Branswell reports, with additional moderators and other employees having been fired by the staff of the International Society for Infectious Diseases, which oversees the program. A number of long-time moderators and the administrative staff of ISID have been at odds since mid-summer over a plan to put much of the information ProMED collects behind a paywall, a move the ISID says is required to sustain the program. The ISID has struggled to pay the moderators, who earn a $7,000 a year stipend.

Many of the moderators and support staff went on strike in early August but most had agreed to return to work at the beginning of this week. That plan changed when ISID CEO Linda MacKinnon terminated the leaders of the strike last week. Some moderators who remained out on strike in protest have been informed their services are no longer required. Among those now listed on the ProMED website as "past team members" are long-time anthrax expert Martin Hugh-Jones, a professor emeritus at Louisiana State University, and viral diseases moderator Laura Kramer, a professor emeritus at the University of New York at Albany's School of Public Health.


science

And the winners are …

In their 10th year, the Breakthrough Prizes — created by Sergey Brin, Priscilla Chan and Mark Zuckerberg, Julia and Yuri Milner, and Anne Wojcicki — deliver kudos to laureates in the life sciences, fundamental physics, and mathematics. The 2024 winners in the life sciences are celebrated for their discoveries in cancer, cystic fibrosis, and Parkinson's disease. AKA the "Oscars of Science," the honors include $3 million prizes for each team: 

  • Carl June of the University of Pennsylvania  and Michel Sadelain of Memorial Sloan Kettering Cancer Center for developing CAR-T therapy, in which key immune cells are genetically engineered to recognize the cancer cells of individual patients. 
  • Sabine Hadida, Paul Negulescu, and Fredrick Van Goor of Vertex Pharmaceuticals for inventing the first effective treatment for the underlying cause of cystic fibrosis. 
  • Thomas Gasser of the University of Tübingen, Ellen Sidransky of the National Human Genome Research Institute, and Andrew Singleton of National Institute on Aging for discovering the two most common genetic causes of Parkinson's disease.

All winners get the chance to walk the red carpet April 13 at a ceremony in Los Angeles.


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What we're reading

  • First, the loss of a baby, then the loss of legal rights, Washington Post
  • The cost of straight hair, The Cut
  • Dana-Farber, in break with Brigham and Women's, will build new cancer center with Beth Israel, Boston Globe
  • Pig kidney works a record two months in donated body, raising hope for animal-human transplants, Associated Press

  • Opinion: How the Wegovy shortage is making life impossible for my patients — and for me, STAT

Thanks for reading! More Monday, but before you go, say farewell to this  scripted logo:
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