Medicare rolls out plans for more drug negotiations

Medicare officials this morning announced plans to negotiate the prices of blockbuster diabetes and weight loss drugs Ozempic and Wegovy, further complicating big decisions the Trump administration will have to make about how to cover the medications.

The drugs are on a list of 15 whose prices will be negotiated this year in a program created by one of President Biden’s signature accomplishments, the Inflation Reduction Act, STAT's Rachel Cohrs Zhang reports. The inclusion of these high-profile medications could raise the public profile of the negotiation program, which has struggled to break through. In a poll conducted by the nonpartisan health policy research and polling firm KFF published in September, 45% of voters surveyed had heard “nothing at all” about the law.

diabetes

Experts see early promise in Sana's type 1 diabetes treatment

When Sana Biotechnology announced last week that it had successfully implanted insulin-producing cells in a type 1 diabetes patient without prompting an immune rejection, the news might have seemed underwhelming. The study was only conducted on one patient, with just one month's data.

But the results sparked a 200% stock surge for the company. Researchers were optimistic, too: “It’s actually very exciting,” one endocrinologist not involved in the study told STAT. “I think it’s much closer today than yesterday. It is an exciting result, a real exciting result.”

Sana plans on scaling the technology using lab-grown stem cells, but a lot of concerns still swirl about safety, scalability, and long-term efficacy. One worry is whether these injected cells — which are designed to evade the immune system — could become cancerous and slip past the notice of immune cells.

RARE disease

Shifting focus on Huntington's disease

There’s a seismic shift underway in understanding Huntington’s disease. Before, researchers focused almost exclusively on the slow buildup of toxic proteins produced by the mutant HTT gene, believing that was the cause for the brutally neurodegenerative disease. Now, however, they’re looking at the gene’s somatic expansion — specifically, the base pairs C, A, and G repeat an incredibly high amount in the mutant HTT gene. The number of these CAG repeats determine when in life a person’s symptoms start, if ever, and they are what underpin neuronal death.

Specifically, research from the lab of Harvard’s Steve McCarroll shows that once medium spiny neurons in the brain reach a threshold of 150 CAG repeats, the cells rapidly deteriorate. Symptoms only occurred after enough neurons had died. Now, pharmaceutical companies are increasingly interested in developing therapies focused on this somatic expansion, not only for Huntington’s but also diseases like fragile X syndrome and spinocerebellar ataxia.

alzheimer's disease

Xenon gas shows promise for Alzheimer's

Might inhaling xenon gas one day help treat Alzheimer’s disease? A new study in mice, published in Science Translational Medicine, found that the gas activated immune cells in the brain called microglia to break down beta-amyloid plaques and reduce brain inflammation. The xenon improved cognitive function and showed reduced levels of Alzheimer’s proteins in treated mice, even in models with the high-risk APOE4 gene.

Xenon’s already used as an anesthetic and to treat brain damage that results from a lack of oxygen, which suggests this simple, inhalation-based treatment could have a solid safety profile for Alzheimer’s disease. The study has already prompted FDA-approved Phase 1 safety trials.

“It’s a really out-of-the-box approach,” one of the study’s authors, a Harvard neuroimmunologist, told STAT’s Anil Oza. “The final verdict will be after a Phase 2 clinical trial. If it reaches its end point with a positive effect on biomarkers and cognition, this is going to be very big.”

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