April 13, 2023
Reporter, Morning Rounds Writer
Good morning. We have some breaking news from overnight on the legal fight around the abortion pill. Plus, Michael J. Fox, arguably the face of Parkinson’s as well as funding for research on the disease, tells his story in a First Opinion and talks to Matthew Herper about a discovery that could speed detection and possibly diagnosis and treatment.
DAhealth
With court ruling, abortion medication remains available for now

Under a decision from a federal appeals court late yesterday, a common abortion medication will remain available as a legal fight around its approval wages, but with some additional restrictions. The decision, to be in place until the appellate court hears the case, will allow the drug, mifepristone, to continue to be dispensed, but no longer through the mail and only up to seven weeks into pregnancy, not 10. 

The panel's order comes after a federal judge in Texas last week ruled that the FDA had improperly approved the drug in 2000, threatening access to the medication around the country. The judge, Matthew Kacsmaryk, paused his ruling for one week to allow for appeals. The case could wind up before the U.S. Supreme Court. 

Health

'This is the big trophy': Research points to biomarker for Parkinson's and a possible path to treat it

Portrait of Michael J. Fox.
Courtesy Mark Seliger

Michael J. Fox has Emmys and an Oscar (for philanthropy), but he’s more excited about science published yesterday in Lancet Neurology: “This is the big reward. This is the big trophy,” he told STAT’s Matthew Herper. Research funded by the Michael J. Fox Foundation for Parkinson’s Research paved the way for the clearest evidence yet that testing for a misfolded protein called alpha synuclein can determine if people have Parkinson’s, the disease Fox has had for more than half his life. The discovery could also speed much-needed treatments.  

“It’s fair to say that when we were starting out, no one expected much from a ‘celebrity charity.’ No matter — we put our heads down and committed to tackle the arduous (or tedious) challenges that other stakeholders wouldn’t or couldn’t,” Fox also writes in a STAT First Opinion. “Today, few would deny that we’re moving the needle.” Read his essay here and Matt’s story here.

exclusive

FDA staff leaned toward rejecting a gene therapy until a top official stepped in

How the FDA approves drugs has come under the microscope lately, and not just for abortion pills. The accelerated approval pathway, first used to speed cancer and HIV treatments to patients while clinical trials continued, has been proposed for gene therapy, too. In an exclusive story, STAT’s Adam Feuerstein and Jason Mast report that agency reviewers thought Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy should be rejected, until a top official stepped in. 

The FDA staff concluded as far back as last summer that Sarepta’s proposed application did not meet the criteria for accelerated approval, sources said about internal deliberations not previously reported. But Peter Marks, a top FDA official and vocal advocate for faster gene therapy approvals, directed FDA staff to schedule a public hearing on the therapy on May 12. At the hearing, outside experts will review Sarepta’s gene therapy data and offer their opinion. Read more.

in the lab

Genome sequencing expands diagnostic power for children with developmental disorders

Rapid advances in genome sequencing have helped identify disease-causing mutations, but a new study takes that approach one step further by bringing that information to patients. Researchers report in NEJM how they diagnosed severe developmental disorders and discovered new genetic conditions in children from thousands of families across the U.K. and Ireland who hadn’t been diagnosed using standard methods. 

The Deciphering Developmental Disorders project, which launched in 2011, has led to some 300 research papers, but yesterday’s sums up how it led to about 5,500 children being diagnosed with conditions caused by mutations on 800 different genes, as well as 60 new genetic conditions. The diagnostic rate was comparatively low for children of African descent, a common result given the lack of diversity in genomic databases. And a diagnosis didn't mean there’s a treatment, but it did inform families. STAT’s Andrew Joseph has more.

Closer Look

Pear was a trailblazer in digital medicine, but making money proved difficult

When Pear Therapeutics filed for bankruptcy last week, the move prompted scrutiny of digital medicine it helped pioneer. Less attention was paid to how the company misjudged its trajectory, believing it was “poised for commercial scale” despite the modest state of its business. STAT’s Mario Aguilar sifted through regulatory filings and investor presentations circulated before Pear went public in 2021, finding optimistic projections like these: By strength of momentum, Pear would grow its business from $4 million in 2021 to a whopping $125 million by the end of 2023.

“There were definitely some indicators that would have said, ‘OK, this is going to move very quickly and we certainly are optimistic in terms of where those numbers are going to go,’” Alex Waldron, who was chief commercial officer and chief strategy officer at Pear before leaving in February 2021, told Mario. “In retrospect, you could certainly say they were too aggressive.” Read more. 

vaccines

Bivalent Covid boosters 'moderately durable'

The bivalent Covid-19 vaccines offer added protection against severe illness, but fleeting protection against infection, a new NEJM study found. STAT's Helen Branswell asked one of the authors, Dan-yu Lin, a professor of biostatistics at the University of North Carolina’s Gillings School of Global Public Health, about the finding published yesterday.

Is what you're showing how much added benefit — in terms of length of protection — the updated booster conferred to people who got it compared to those only boosted earlier with the monovalent vaccine?

In this article, as well as in our earlier article published in NEJM on February 23, we estimated the effectiveness of one additional dose.

How would you characterize the added protection from the bivalent booster? It seems to wane quite quickly.

The amount of added protection was high and waned gradually over time. In this article, we showed that bivalent boosters were moderately durable and were effective against the currently circulating XBB.1.5 [Omicron] subvariant.

Some countries are allowing high-risk individuals to get a spring booster. Do these results argue for a similar policy in the U.S.?

Our results showed that the effectiveness of boosters declined to a low level after five months. In addition, older adults and people who are immunocompromised have much higher risks of hospitalization and death than young and healthy people. Thus, it would be sensible for those people to get a spring booster.

public health

Declines in deaths from suicide have slowed or reversed for some groups

Screen Shot 2023-04-12 at 10.54.30 AMNational Center for Health Statistics, National Vital Statistics System, Mortality

Two CDC reports on suicide released today — one on deaths from suicide (above) and another on emergency department visits by people considering suicide (below) — chart the falling then rising rates for the 10th highest pre-pandemic cause of death. The researchers highlight differences by sex, age, and race, suggesting possible goals for intervention.  

  • From 2001 to 2018, suicide deaths rose by 33%, fell 14% for two years, but increased 4% in 2021. Suicide rates were highest among American Indian or Alaska Native people in 2021; deaths increased from 2020 to 2021 for American Indian or Alaska Native, Black, and white males and for Black and white females.
  • From 2016 through 2020, ED visits for suicidal thinking, a strong predictor of death by suicide, were highest among youths ages 14 to 18 and higher in female than male teens. Across all ages, they were higher for males than females. 
  • Rates for these ED visits over these five years were highest for Black people, followed by white people; they were lowest for people living in the Northeast compared to those in other regions. 
Screen Shot 2023-04-12 at 10.54.51 AMNational Center for Health Statistics, National Vital Statistics System, Mortality

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.

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What we're reading

  • Exclusive: Sanders plans to haul in insulin executives to testify in Senate, STAT
  • Juul agrees to pay $462 million to 6 states and D.C., and to share documents, STAT
  • White House moves to protect some abortion patients’ records, Associated Press

  • China’s struggles with lab safety carry danger of another pandemic, Washington Post

  • Sickle cell gene therapies could be cost-effective even if priced as high as $1.9 million, STAT

Thanks for reading! More tomorrow,

elizabeth.cooney@statnews.com

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