Marathon runners are shown running from their legs down.

Physical activity like running a marathon may do more good than researchers imagined — like potentially informing new ways to treat multiple sclerosis.

Credit: iStock.com/tibor5

Weekly Rundown: Myelin may act as a fuel source during marathons 

A landmark UTI drug approval, new hope for the amyloid hypothesis in Alzheimer’s disease, a brewing vaccine study controversy, and more led the news this week.
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Welcome to the Weekly Rundown! If there’s anything in particular you’d like to see in this new column, send us an email at: editor@drugdiscoverynews.com

Myelin may act as a fuel source during marathons 

Myelin — the fatty substance that insulates electrical signals in nerve cells — may act as a source of fuel during prolonged physical activity. Brain scans of marathon runners revealed that after a race, myelin was reduced in brain areas responsible for motor coordination and sensory and emotional integration (1). However, cognitive function remained unaffected, and myelin levels returned to normal within two months. These findings could inform new approaches for restoring myelin levels in conditions like multiple sclerosis, where the immune system attacks myelin in nerve tissues, disrupting communication between the brain and the rest of the body. – Dika Ojiakor

FDA approves novel oral antibiotic to treat urinary tract infections

About 50 percent of all women will experience a urinary tract infection (UTI) at some point in their lives, and about 30 percent will have a recurrent episode. On Tuesday, the FDA approved GSK’s gepotidacin (Blujepa) as the first-in-class oral triazaacenaphthylene antibiotic for uncomplicated UTIs. Blujepa works by inhibiting two different type II topoisomerase enzymes that bacteria use to replicate. The approval is based on multiple Phase 3 clinical trials in more than 3,000 female adults and children 12 years and older, which showed that Blujepa was as or more effective in treating uncomplicated UTIs than commonly prescribed antibiotics. – Allison Whitten

Roche’s discontinued Alzheimer’s disease drug provides hope for symptom prevention

People with a genetic form of Alzheimer’s disease (AD) called dominantly inherited Alzheimer's disease (DIAD) account for less than one percent of people diagnosed with AD, but they will eventually develop the disease — usually by the time they reach 30 to 50 years old. In 2020, Roche released topline results from their Phase 2/3 trial testing their anti-amyloid drug, gantenerumab in this population, and while the drug reduced amyloid levels in the brain, it didn’t significantly slow cognitive decline. But, based on the promising amyloid reduction data, researchers at Washington University School of Medicine continued testing the drug in an open-label extension study. In a new paper in The Lancet Neurology, they’ve reported that gantenerumab reduced the risk of developing AD symptoms from 100 percent to 50 percent in these patients (2). – Stephanie DeMarco

An antibody associated with lupus could boost cancer treatment

Researchers at Yale University discovered that 4H2, a lupus-derived antibody, could successfully penetrate into brain, lung, and breast tumors and activate an inflammatory signaling response that killed cancer cells in culture (3). Produced by the immune system in patients with lupus, the researchers found that 4H2 binds to a cell’s own RNA and specifically activates cyclic GMP-AMP synthase (cGAS) to set off the cancer-killing attack. Their work also showed that in mice with glioblastomas, 4H2 improved the efficacy of immune checkpoint inhibitors — suggesting the potential for the antibody to be added to future cancer treatment regimens in patients. – Allison Whitten

Cassava discontinues Alzheimer’s disease drug after another Phase 3 failure

Cassava Sciences, a neuroscience-focused pharmaceutical company, has halted the development of simufilam, its AD drug candidate, after it failed in a second Phase 3 clinical trial. Simufilam is an oral small molecule drug designed to stabilize filamin A, a structural protein that helps build the cell’s cytoskeleton. In a press release, Cassava reported that simufilam did not significantly reduce cognitive decline in patients with mild-to-moderate AD when compared to a placebo, prompting the drug’s discontinuation. The announcement comes months after the first Phase 3 trial failure for simufilam, marking the end of Cassava’s development efforts for the drug, which have been plagued by scandal and controversy from the basic research underlying the drug’s initial development to trial results. The company, however, is interested in studying simufilam in other neurological disorders, including tuberous sclerosis complex-related epilepsy. – Dika Ojiakor

Vaccine skeptic reportedly hired to oversee controversial vaccines and autism study

The US Department of Health and Human Services has hired David Geier — a discredited researcher and vaccine skeptic — to analyze vaccine data from four previously conducted studies on the link between immunizations and autism, The Washington Post  reported. None of these studies had found any link between vaccines and autism. Geier has published multiple papers on thimerosal — an old vaccine preservative that is rarely present in currently available vaccines — and autism, including one study that was retracted in part due to Geier’s failure to disclose his conflicts of interest (4). He is not a physician and has been charged by the Maryland State Board of Physicians for practicing medicine without a license. Scientists are concerned by Geier’s reported involvement in the study and how his apparent conflicts of interest may affect the study results. – Stephanie DeMarco

References

  1. Ramos-Cabrer, P. et al.  Reversible reduction in brain myelin content upon marathon running. Nat Metab  (2025).
  2. Bateman, R.J. et al.  Safety and efficacy of long-term gantenerumab treatment in dominantly inherited Alzheimer's disease: an open-label extension of the phase 2/3 multicentre, randomised, double-blind, placebo-controlled platform DIAN-TU trial. Lancet Neurol  24, 316-330 (2025).
  3. Chen, X. et al.  A lupus-derived autoantibody that binds to intracellular RNA activates cGAS-mediated tumor immunity and can deliver RNA into cells. Sci Signal  18, eadk3320 (2025).
  4. Kern, J.K. et al.  RETRACTED ARTICLE: Systematic Assessment of Research on Autism Spectrum Disorder and Mercury Reveals Conflicts of Interest and the Need for Transparency in Autism Research. Sci Eng Ethics  23, 1689-1690 (2015).
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