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Bracing for another debate on Duchenne therapy

November 2, 2023
Reporter, Morning Rounds Writer
Good morning. Our story on Duchenne gene therapy is another reminder of the people who feel the impact of forces beyond their control, from biotech companies to federal regulators to a disease itself.

gene therapy

Duchenne therapy: 'Nothing is ever a slam dunk'

Earlier this week, Sarepta Therapeutics reported disappointing results from a clinical trial of its gene therapy for Duchenne muscular dystrophy. The treatment won provisional clearance from the FDA in June, but only for 4- and 5-year-olds, so families and doctors are waiting for more from the agency. While the latest study failed to meet its primary metric, secondary measures looked better. Experts have seen children in their clinics now ride bikes, jump on trampolines, and do other things kids with Duchenne all but never do.

"You know, nothing is ever a slam dunk for us," said Kelly Maynard, the mom of a teenager with Duchenne and head of Little Hercules Foundation. "I think we're used to that." All this comes against a background of questions about how the FDA views Sarepta. A former FDA reviewer said "the FDA seems to have a separate evidence standard for Sarepta." Read more from STAT's Jason Mast and Adam Feuerstein.


health

American Cancer Society urges lung screening for more smokers

Anyone with a significant smoking history should get an annual low-dose CT scan for lung cancer, the American Cancer Society said yesterday, loosening guidance that leaned hard on how much people smoked and when they quit. The new guidelines reduce the smoking history requirement from 30 cigarette pack years to 20 pack years (equivalent to two packs a day for 10 years). The age range widened to 50 through 80, instead of 55 through 74.

The new guidance is in line with what the U.S. Preventive Services Task Force recommends, which is important for private insurance coverage. Under 6% of eligible patients actually get lung cancer screening, estimates suggest. And the new thinking may correct previous guidelines that disproportionately excluded women, Hispanics, Asians, and African Americans, experts said. For the same amount of smoking history, these groups seem to have a higher risk of lung cancer. STAT's Angus Chen has more.


neuroscience and oncology

How tumor cells hijack brain plasticity to drive cancer

Monje_2021_MacArthurFellowCourtesy MacArthur Foundation

Since 2015, Stanford neuro-oncologist Michelle Monje has been revealing how cancer can subvert normal brain plasticity. "Neuroscience is very important as a principle of cancer biology," said Monje. "It's just a very newly recognized one." I asked her what her latest research, published yesterday in Nature, shows.

How do tumors hijack the normal brain?

The tumor cells are doing exactly the same thing as a healthy cell would do, but here, instead of it promoting a memory or improving connections between neurons that strengthen the function of a neural circuit, it is driving cancer growth. 

What could this mean for treating brain cancers?

A lot of the medicines that target what we and others have been discovering already exist for other reasons. But we haven't been giving them to cancer patients. So it opens up a new avenue of potential therapeutics that have to be studied in clinical trials. 

Read the full interview.



closer look

Treating rural America: The telehealth solution

rural america

Hyacinth Empinado/STAT 

There's a stark urban-rural divide in the U.S. when it comes to specialty care. "We know that urban patients typically do better based upon their ZIP code," said David Newman, an endocrinologist based in Fargo, N.D. "We don't want your care to be defined by your ZIP code. We want everyone to have equal access to specialty care."

Part three of STAT's short documentary series on rural health focuses on how Newman and his colleague, Johnna Nynas, are using technology to offset major obstacles to healthcare access in their region. Watch here for the latest from Hyacinth Empinado.


infectious disease

New antibiotic against gonorrhea stacks up well

Promising results from a Phase 3 trial are raising hopes for the first new treatment for gonorrhea in decades, a development that experts say cannot come too soon. "We're on borrowed time," Edward Hook, a professor emeritus at the University of Alabama at Birmingham who was protocol chair for the clinical trial, told STAT's Helen Branswell. The drug comes from the nonprofit Global Antibiotic Research & Development Partnership and Innoviva Specialty Therapeutics.

Since the advent of antibiotics, Neisseria gonorrhoeae has developed resistance to each class of antibiotics wielded against it, leaving ceftriaxone as the sole remaining drug that can reliably cure gonorrhea. But in the past few years, there have been mounting reports of cases of ceftriaxone-resistant gonorrhea. If it is not successfully treated, gonorrhea can cause pelvic inflammatory disease and infertility in women as well as sterility in men and blindness in babies infected during birth. Read more.


mental health

Brain function reflects the impact of adverse experience, study says  

Adversity leaves its traces in the adult brain, a meta-analysis of 83 neuroimaging studies says, changing the way the brain reacts to a variety of challenges. Someone's ability to cope with later stressors could be lessened while their susceptibility to mental health problems could grow. The researchers found greater amygdala reactivity and diminished prefrontal cortex reactivity in response to four specific tasks: emotion processing, memory processing, inhibitory control, and reward processing.

These are regions of the brain that size up potential threats and set in motion defensive behaviors and physical coping. Changes in these brain responses were clearest in people who'd been exposed to severe threats and trauma. "These results might better identify how adversity diminishes the ability to cope with later stressors and produces enduring susceptibility to mental health problems," the study in JAMA Network Open notes.


More around STAT
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Read premium in-depth biotech, pharma, policy, and life science coverage and analysis with all of our STAT+ articles.

What we're reading

  • A modest proposal to save mothers' lives, The Atlantic
  • A 'tropical disease' carried by sand flies is confirmed in a new country: the U.S., NPR
  • With a new center, All of Us tackles health data silos to power precision medicine, STAT
  • Infant mortality in the U.S. rose last year for the first time in two decades, says CDC, Associated Press
  • Nostrum Labs and its CEO — who once defended Shkreli price hikes — to pay up to $50 million over Medicaid rebates, STAT

Thanks for reading! More tomorrow,


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