CAR-T showing more promise in autoimmune disease

A once-radical idea — repurposing CAR-T cancer therapy to reset the immune system — is rapidly gaining traction in autoimmune disease. Five years ago, researchers found that a lupus patient achieved durable remission with a cellular therapy, and a newer report shows a critically ill woman recovered from three overlapping conditions with CAR-T — autoimmune hemolytic anemia, immune thrombocytopenia, and antiphospholipid antibody syndrome.

Now, STAT’s Isabella Cueto writes, researchers are racing to refine the approach, targeting B cells through markers like CD19 and BCMA. Drugmakers are pouring investment into this approach, and hospitals are building in-house CAR-T programs for autoimmune disease.

“There's a huge level of interest in this area now from pharma” based on pre-clinical data, one Massachusetts General Hospital CAR-T researcher told STAT.

That said, some patients relapse as B cells return, it’s still unclear which diseases are optimal targets, and the therapy carries real risks — from toxicity to cancer. And the price tag that can exceed $500,000. Still, it’s a burgeoning area, and there’s no dearth of clinical interest.

autism

GSK says goodbye to leucovorin (again)

From STAT's Lizzy Lawrence: The Food and Drug Administration is withdrawing approval of GSK’s leucovorin to treat cerebral folate deficiency, which is a rare brain disorder that resembles autism.

The announcement, in the Federal Register, is the culmination of a bizarre episode that exemplified the ways political pressure began to shape the FDA’s work last year, as STAT previously reported. In September, the agency asked GSK to revive leucovorin, which the company had discontinued in 1997 due to low sales.

The inquiry appeared to stem from health secretary Robert F. Kennedy Jr.'s and President Trump’s long-standing interest in autism. Though career staff successfully pushed to limit the approval to a rare disorder, as supported by existing studies, FDA Commissioner Marty Makary publicly touted the drug as a cure for autism.

Withdrawing approval was always part of the plan, GSK told STAT, as the company never intended to market or manufacture the product. Rather, the agency asked GSK to submit the drug application so that generic drug companies could also update their labels to reflect the cerebral folate deficiency indication. If GSK held on to the approval, the company would be required to submit safety reports — not in the interest of a company not actively selling a drug.

Patients will still be able to access generic leucovorin, said health department spokesperson Andrew Nixon.

podcast

A new trick for old science, and biotech VCs' scrambled playbook

Why is old exon science getting new traction? What’s unsettling biotech VCs? And who will be the next CEO of PhRMA?

We discuss all that and more on the latest episode of “The Readout LOUD,” STAT’s weekly biotech podcast.

Adam is out, so Elaine and Allison dive into the latest news, including how China and artificial intelligence are straining biotech VC firms, in addition to more M&A. They also discuss how a cancelled Food and Drug Administration meeting led to a biotech’s demise.

Plus, there’s new hope for an old theory on treating Duchenne muscular dystrophy. STAT reporter Jason Mast details the clinical testing underway.

Listen here.

washington

Personal stories power a health tech push at HHS

From STAT's Brittany Trang: It was a family affair at the Department of Health and Human Services yesterday, at least for a couple of officials, including Amy Gleason of CMS and DOGE. Her daughter Morgan, whose chronic illness and medical record feature prominently in virtually every speech the elder Gleason gives, spoke at a public event about how a more seamless patient portal experience could help her manage her 51 logins. Former U.S. chief technology officer Aneesh Chopra's mother also came onstage to talk about how importing her medical records into Claude made her more reassured about a health scare.

Yesterday's event was an opportunity for health tech companies to show off what they've built in response to last July's "pledge" to Medicare that they would make health records more portable and build apps that would help people manage diabetes and obesity and answer questions about a patient's own medical records with AI. But how much will a voluntary tech pledge change Americans' health outcomes and costs?