Breaking News

A historic decision for gene therapy, the latest on medication abortion, & Uber’s new health care pilot

April 17, 2023
Good morning, it's Marathon Monday here in Boston. This is reporter and podcast producer Theresa Gaffney filling in for Liz, as she prepares to run her 13th (!) Boston Marathon. Join the STAT team in cheering her on.

special report

Gene therapy for Duchenne nears historic decision

Alex Haile, a senior scientist at Sarepta Therapeutics, looks at immunofluorescence images of muscle fibers in the lab in Columbus, OH
Maddie McGarvey for STAT

When Emerson Furbee was diagnosed with Duchenne muscular dystrophy at just 3 years old, his pediatrician told his parents that there was nothing the family could do. But that same year, three companies including Sarepta were racing to develop a life-altering gene therapy for Duchenne. The Furbees were determined to enroll Emerson in research, and two years later, he received a groundbreaking treatment in a Sarepta clinical trial.

This past September, Sarepta asked the FDA to conditionally approve the gene therapy that Emerson received for any Duchenne patient who can still walk. The decision, expected by May 29, is perhaps the most consequential moment in the history of one of the most common and debilitating rare diseases — the culmination of 40 years of work by patient advocates, research groups, and rival companies. For Sarepta, it's a story of foresight, savvy dealmaking, scientific puzzle solving, and perseverance. The debate over Sarepta's application has roiled the FDA, as STAT's Adam Feuerstein and Jason Mast reported last week. In a new special report, they have the harrowing story of how we got here.


abortion

SCOTUS likely to rule on abortion pill this week

There's a pivotal week ahead for the legal battle over the abortion pill mifepristone, after more movement on Friday. Access to the drug will remain unchanged until this Wednesday night, after Supreme Court Justice Samuel Alito issued a stay on an April 7 ruling from a conservative Texas judge banning the medicine. It is likely the country's highest court will rule more substantively on mifepristone before then, a decision that will have major ramifications for the FDA's authority and access to the commonly used drug. 

An appeals court had allowed mifepristone to stay on the market during legal processes but banned mail-order deliveries of the drug and ruled that it could only be used up to seven weeks into a pregnancy, not 10. Alito's stay effectively returns mifepristone to FDA's status quo. Read more from STAT's Sarah Owermohle.


cancer

Moderna makes strides toward a personalized cancer vaccine

For years, scientists have worked toward the goal of someday being able to offer cancer vaccines tailored to an individual's tumor. On Sunday, a personalized mRNA cancer vaccine made by Moderna and Merck provided the first evidence that the approach can truly offer clinical benefits to patients. The vaccine, formulated based on the unique mutations in a patient's tumor, greatly reduced the risk of relapse when combined with a Merck immunotherapy drug than when the drug was given alone.

Scientists randomly sorted 157 patients with stage 3 or 4 melanoma to receive the standard immunotherapy alone or the combination. All patients knew whether they were receiving the vaccine or not, so the results will still need to be confirmed in a larger trial. Still, experts are encouraged. "I was really super excited about it. It's very rewarding to see this type of data having worked in the field for more than 10 years and seeing early signs of activity," Patrick Ott of Dana-Farber Cancer Institute told STAT's Angus Chen. Read more.



Closer Look

Uber pushing insurers to cover free rides to prenatal appointments 

For patients of the Community of Hope and Mary's Center clinics in Washington, D.C., getting to medical appointments can be a challenge — many live far from public transit or lack cars. That's why ride-sharing company Uber, which has for years been searching for a viable way into the $4 trillion health care market, swooped in to offer hundreds of pregnant patients in D.C. free rides to appointments in 2021 and 2022. Uber says patients who participated in the pilot were slightly more likely to get prenatal care, and it's shopping the data around to insurers in a bid to get them to pay for the service.

The company is "spending a lot of time talking with Medicaid plans about how to structure that [transportation] benefit design, and how to administer that benefit," its global head of health, Caitlin Donovan, told STAT's Mohana Ravindranath. "Existence of the benefit alone is not enough." Read more.


health

'Astonishing' study finds that Black people live longer in counties with more Black doctors

Health equity experts have long emphasized the benefits of when Black patients are seen by Black physicians. And a new study finds that Black people in counties with more Black primary care physicians live longer — whether they actually see those doctors or not. The study, published in JAMA Network Open, is the first to link a higher prevalence of Black doctors to longer life expectancy and lower mortality in Black populations. 

"That a single Black physician in a county can have an impact on an entire population's mortality, it's stunningly overwhelming," Monica Peek, a primary care physician and health equity researcher at UChicago Medicine, told STAT's Usha Lee McFarling. The study did not address why Black patients fare better, and it isn't a strict cause-and-effect. Still, "to see the impact at the population level is astonishing," Peek said. Read more.


infectious disease

CDC: Bird flu mutations not a cause for concern

Genetic sequence data from H5N1 bird flu viruses that infected a man in Chile shows the virus underwent a couple of changes that are thought to be signs of early adaptation to humans. But scientists from the CDC, who spotted the mutations in the virus' PB2 gene while analyzing the sequence on Friday, told STAT's Helen Branswell they are not overly concerned, noting there were no significant changes in the hemagglutinin, the protein that flu viruses use to attach to human cells they are infecting. Vivien Dugan, who heads CDC's flu division, said the changes may have occurred as the viruses replicated in the unnamed man.

Richard Webby, a flu expert at St. Jude Children's Research Hospital, concurred with the CDC's assessment, saying these changes are ones that seem to be easy for H5 viruses to make, but they aren't enough to allow them to start spreading from person to person.


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What we're reading

  • Exclusive: The Biden administration will hang on to some Covid pandemic emergency powers, STAT
  • A beauty treatment promised to zap fat. For some, it brought disfigurement, New York Times
  • Abortion pills, take two, Radiolab
  • UnitedHealth says medical costs aren't soaring. The reality is murkier, STAT
  • The day 'stop the bleed' entered civilian life, The Atlantic
  • 72 hours: Inside San Diego County's mental health crisis, San Diego Union-Tribune

Thanks for reading! More tomorrow! — Theresa

Theresa Gaffney is a reporter and podcast producer at STAT.


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