Breaking News

Questions on CRISPR therapy's historic approval, obesity drugs' next target, & trouble with FIT tests

November 17, 2023
Reporter, Morning Rounds Writer
Good morning. The dust hasn't yet settled on the first CRISPR therapy being approved or on the rise of obesity drugs, but we're looking into unanswered questions.

biotech

The first CRISPR therapy is here. We have questions

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U.K. regulators made history yesterday when they approved the first CRISPR gene-editing therapy, one that aims its RNA guide and molecular scissors at the blood disorders sickle cell disease and beta thalassemia. The treatment, Casgevy, was developed by CRISPR Therapeutics and Vertex Pharmaceuticals. U.S. approval is expected by Dec. 8, but neither regulatory nod answers all the questions about what is not an easy treatment to receive, requiring patients to spend weeks, even months, in the hospital before and after the therapy is administered. How does it work? Who gets it? What could go wrong?

The two diseases Casgevy treats are daunting, and because sickle cell disease predominantly affects people of African ancestry, it carries the heritage of racism in medicine, too. Then there's the likely millions-plus price for what promises to be curative. "Approval does not mean access," said Teonna Woolford, CEO of the Sickle Cell Reproductive Health Education Directive. STAT's Matthew Herper, Adam Feuerstein, Brittany Trang, and Eric Boodman look at these and other questions here.


the obesity revolution

The next goal in obesity drugs: keeping lean muscle

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What do mighty mice so muscle-bound they look like bodybuilders have to do with the new wave of blockbuster weight loss drugs? Body composition, or the changes in fat versus muscle, aren't typical endpoints for a weight loss drug trial. But new drugs like Ozempic, Wegovy, and Mounjaro (aka Zepbound) tend to reduce not just fat but also lean muscle when body mass can drop by 15% to 21%. That's why drug developers are giving a second look to mouse muscles that ballooned after the protein mysostatin was blocked.

That's just one approach biotech startups are pursuing to create what they believe will be better weight loss medications. They've learned that inhibiting myostatin isn't enough for disorders like Duchenne muscular dystrophy or spinal muscular atrophy. And there's disagreement about whether this is a problem that needs to be solved. STAT's Elaine Chen and Allison DeAngelis have more, including an updated Obesity Drug Tracker.


infectious disease

Measles cases and deaths are climbing as childhood vaccinations wane around the world

A falling rate of childhood vaccinations against measles is giving rise to a surge in cases and deaths from the highly contagious virus, the WHO and CDC said yesterday in a joint report. Year-to-date figures for 2023 are already expected to double the numbers from 2022. "If this carries on the direction it's going in, this is going to be a disaster for children in the most vulnerable settings," Natasha Crowcroft, the WHO's global lead for measles and rubella, told STAT's Helen Branswell.

Measles often causes a nasty flu-like illness with an itchy full-body rash, but it can also kill, especially when children are malnourished or have other underlying health conditions. Vaccination rates have been declining in upper-income countries due to vaccine hesitancy. In lower-income countries, the declines are attributed to weak health care systems, inadequate funding, and security challenges — issues that were all exacerbated by the pandemic. Read more.



closer look

Colon cancer tests had high rate of unusable samples

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Fecal immunochemical tests to screen for colorectal cancer, better known as FIT, are a tool health care leaders hope can help drive down cancer rates, especially in populations with higher death rates compared to white men: American Indian or Alaska Native men (46%) and Black men (44%). A new study performed at a safety-net system in Dallas that serves more than a million uninsured, lower-income patients, including people of color, says the tests' usefulness is undercut by the high percentage containing samples that could not be processed by labs.

More than a tenth of the tests, performed in a clinic or sent through the mail, had problems with the specimen, labeling, or containers. More worrying, researchers write in the journal Cancer Epidemiology, Biomarkers and Prevention, was the finding that only about 40% of those who had an unsatisfactory test received a follow-up test or a colonoscopy within 15 months of the failed result. STAT's Debbie Balthazar has more on why.


 

health equity

FDA asks for feedback on pulse oximeters and their lower accuracy on patients with darker skin

Just two weeks after two dozen state attorneys generals sent a letter prodding the FDA to take more urgent action to remedy pulse oximeters, widely used oxygen-measuring devices that have been shown to be less accurate on patients with darker skin, the agency announced it will convene a second public meeting to study the issue on Feb. 2. The agency also asked for feedback on a discussion paper released yesterday that proposes — as many physicians and researchers called for during a public meeting held in Nov. 2022 — having more patients with darker skin involved in pre-market testing of the devices, and implementing more standardized ways to measure skin pigment in those tests. 

The scheduling of yet another meeting in lieu of new rules will likely be frustrating to those who have linked the inaccuracy of the devices to slower diagnosis and treatment for patients, STAT's Usha Lee McFarling tells us, but Jeff Shuren, director of the FDA's Center for Devices and Radiological Health, said the agency had been working on the issue, including commissioning studies to evaluate how well the devices work in both children and adults and working to analyze existing data on the devices.


reproductive health

Infant and maternal health in the U.S. at 'crisis level'

Preterm births and infant mortality are at alarming levels in the U.S., according to a new March of Dimes report published yesterday. In 2022, 10.4% of all babies were born premature (defined as before  37 weeks of gestation),  higher than the global average of 9.9%. The U.S. has the highest risk of preterm birth among its wealthy peers: In the U.K. the rate is 7.6%; in Italy, it's 6.8%; in Japan, it was around 5%.

"This year's report shows the state of infant and maternal health in the United States remains at crisis-level, with grave disparities that continue to widen the health equity gap," said March of Dimes President and CEO Elizabeth Cherot, in a statement. There are significant racial and ethnic disparities: 14.6% of Black babies were born preterm, compared to 9.4% of white babies and 9% of Asian babies. STAT's Annalisa Merelli has more.


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What we're reading

  • FDA's Janet Woodcock to retire early next year, STAT
  • Why some seniors are choosing pot over pills, New York Times

  • Doctors divide over reforming a secretive panel that determines their Medicare pay, STAT

  • Progress in childhood cancer has stalled for Blacks and Hispanics, report says, Associated Press
  • FDA cites Dr. Reddy's for quality control problems at a biologics plant, STAT

Thanks for reading! More tomorrow,


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