July 28, 2025

The need-to-know this morning

Celcuity said its experimental cancer drug, gedatolisib, delayed tumor progression in women with a certain type of breast cancer, achieving the primary goals of a Phase 3 clinical trial.
GSK said it would pay $500 million upfront for rights to an experimental COPD drug from Jiangsu Hengrui Pharmaceuticals, the latest example of a pharma company turning to a Chinese firm to build out its pipeline.

chronic disease

Did this treatment prevent ALS for one man?

After losing multiple relatives to ALS, molecular biologist Jeff Vierstra volunteered for an experimental therapy targeting the FUS gene before showing any symptoms — becoming the first known attempt to prevent the fatal disease. While his sisters, who also carried the gene and were also treated, both died from falls in 2023 and 2024, Vierstra remains symptom-free nearly five years later — raising hope among researchers and gene carriers alike for presymptomatic intervention.

The results, still anecdotal, underscore a growing push to treat neurodegenerative conditions before decline begins, STAT's Jason Mast writes — despite scientific unknowns, ethical risks, and regulatory hurdles. Vierstra, meanwhile, is planning a ski trip to Antarctica.

policy

Proposed NIH cuts would have sprawling effects, analysis predicts

A new JAMA Health Forum paper warns that the Trump administration's proposed 40% cut to NIH funding could dismantle decades of biomedical progress, ultimately costing more than it saves. The authors argue that beyond immediate losses — like axed grants and staff layoffs — the long-term ripple effects include a shrinking scientific workforce, stalled public health gains, and research gaps unlikely to be filled by industry.

"This is an unfortunate reality. If you blow up this system of innovation, it's going to take some time to feel the pain from it, and it's not going to be maybe as salient as the pain from the Medicaid cuts that were just enacted or cutting food assistance," one economist who has studied the benefits of federal funding on R&D told STAT's Anil Oza. "Unfortunately, that means that should the cuts go through, there won't be much accountability for those policymakers because of their actions."

obesity

Lawmakers demand crackdown on surge in counterfeit weight loss drugs

In a letter sent Friday, Rep. Richard Hudson and 81 bipartisan lawmakers urged FDA Commissioner Marty Makary to take immediate action against the flood of illegal, unapproved weight loss medications entering the U.S. They say the products, often counterfeit versions of semaglutide-based drugs like Ozempic and Wegovy, are being smuggled in from unregistered foreign suppliers — primarily in China — and pose serious health risks including overdose, organ damage, and death.

The lawmakers are calling for stricter border enforcement, import alerts, and a coordinated crackdown on compounding pharmacies and online sellers capitalizing on the demand for GLP-1 drugs.

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