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Good morning. Don’t miss Brittany Trang’s compelling story about seven-year-old Shelby Campbell’s life — and her family’s — while being treated for beta thalassemia with gene therapy.

What ‘one time’ means when you’re 7 years old and gene therapy takes a grueling year

Shelby and her parents sit together for family pancake breakfast on Saturday morning at home in Mechanicsburg, PA on August 26, 2023. Amanda Berg for STAT

Two gene therapies approved by the FDA last week have been heralded as one-time, essentially curative therapies. But what does that mean? To leave behind beta thalassemia or sickle cell disease, “one time” means months, or for seven-year-old Shelby Campbell, more than one year. That includes seven weeks in the hospital, toxic chemotherapy to wipe out defective cells, hair loss, high fevers and mouth sores, and isolation from friends and school.

STAT’s Brittany Trang chronicled the Campbell family’s journey through Shelby’s treatment for beta thalassemia with Bluebird Bio’s Zynteglo. “It’s definitely not a quick fix or an easy fix,” said her mother, Michelle Campbell. Yet by the end, Shelby was giving advice to another child about feeding-tube insertion: “You might need to get it in a few times but once you get used to it, it’s not that scary and you know what to expect.” Read more.

Humana sued again over algorithm used to deny care

A second class action lawsuit from Medicare Advantage beneficiaries was filed yesterday against Humana, also alleging that the health insurance giant used an algorithm to cut off payment for rehabilitative care needed by patients recovering from serious illnesses and injuries. This one, filed in U.S. District Court in Western Kentucky, argues Humana’s reliance on the algorithm was part of a fraudulent scheme to reap a windfall by systematically denying claims to desperately ill people. An earlier complaint was filed in November against the algorithm’s maker, NaviHealth, and its owner, UnitedHealth Group.

Both suits follow a STAT investigation that found Medicare Advantage insurers nationwide are using the algorithm to cut off patients’ rehab care. Complaints alleging the improper denial of care are common, STAT’s Bob Herman and Casey Ross point out, but these cases are among the first to challenge insurers’ reliance on an internal algorithm. Read more.

Promising news about an under-the-radar sickle cell treatment

Historic approvals of two pricey ($2.2 million and $3.1 million) gene therapies for sickle cell disease have grabbed our attention, but there’s a less expensive, more traditional approach turning heads at the American Society of Hematology in San Diego. Yesterday scientists reported advances in bone marrow transplantation, a decades-old approach that means patients need a well-matched donor and a hefty dose of chemotherapy, two requirements hard for most patients to meet.

In the new mid-stage trial, after sickle cell patients were given a gentler course of chemo and an infusion of half-matched cells, they had less pain, 95% were alive two years after transplant, and only 7% experienced a severe immune reaction, study co-author Adetola Kassim told reporters. “Whatever we do in terms of novel research, if it cannot reach the patients who need it, what’s the whole idea?” study co-author Adetola Kassim said. STAT’s Jonathan Wosen has more. And STAT’s Jason Mast reports on about early animal data from Novartis presented at the same meeting about a potential pill for the disease.

Doctors call out ableism in cancer care, where assumption can harm patients

Cheri Blauwet (right) with a patient in an exam room. -- health coverage from STAT
Spaulding Rehabilitation Network

People who have a disability often need to educate others about what they can and can’t do, including doctors whose assumptions can undermine their care. In cancer, for example, clinicians might assume a woman who uses a wheelchair isn’t sexually active, so doesn’t need to be tested for the virus that causes cervical cancer. After a cancer diagnosis, treatment can follow outdated decision trees that depend on a patient’s ability to walk as a benchmark for tolerating chemotherapy.

“To make an assumption that a wheelchair user must inherently be someone with a significant burden of chronic disease is a very ableist assumption,” Cheri Blauwet, chief medical officer at Spaulding Rehabilitation Hospital (pictured above with David Estrada), told me. It’s perhaps an understatement to say she knows whereof she speaks. She’s a Paralympic gold medalist and two-time winner of both the Boston Marathon and the New York City Marathon in wheelchair racing. I have more here.

For kids with high BMI, task force not ready to recommend weight loss drugs

In its new draft recommendation on high BMI in children and adolescents, the U.S. Preventive Services Task Force has moved from its 2017 guidance suggesting screening to urging intensive, comprehensive behavioral interventions. Today’s recommendations, open for comment until Jan. 16, stop short of weight-loss medications for the roughly 20% of children and teens with high BMI in the U.S., saying “more research is needed to fully understand the long-term health outcomes for medications.”

Instead, the group recommends providing or referring kids age 6 and older who have a BMI above the 95th percentile for age and sex on the growth chart to behavioral interventions such as supervised exercise, counseling for behavior change, and information about healthy eating. The American Academy of Pediatrics has come out in favor of weight-loss drugs for adolescents, but as this New York Times story tells us, many pediatricians are reluctant to prescribe them.

Experimental male birth-control pill enters trial

We haven’t heard too much lately about male birth control, but a San Francisco startup hopes to change that story with a non-hormonal drug candidate that began its Phase 1 clinical trial today in the U.K. to test the safety, tolerability, and functioning of the oral drug in 16 participants. YourChoice Therapeutics is betting on century-old science that discovered mice, rats, and monkeys with vitamin A deficiency were infertile, which dozens of papers have since confirmed. The experimental drug is designed to stop the metabolism of vitamin A in the testicles, thus blocking both the production and the release of sperm — completely eliminating the chances of fertilizing an egg.

Ultimately, the team behind YCT-529 hopes that if their product is successful, it might help change the dynamics of contraception, and make it more of a shared responsibility. STAT’s Annalisa Merelli has more, including another male contraceptive product currently being studied.

What we’re reading

  • Pharmacies share medical data with police without a warrant, inquiry finds, Washington Post

  • CPR can be lifesaving for some, futile for others. Here’s what makes the difference, NPR
  • A targeted therapy shows it can offer a ‘last, great chance’ for some acute leukemia patients, STAT
  • Thousands of patients may be undergoing vascular procedures too soon or unnecessarily, ProPublica
  • Maze CEO ‘personally disappointed’ in Sanofi decision to terminate partnershipSTAT





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