hotspot -WSS

hotspot

author：knowledge 2025-09-30 16:26:39 - browse：5

Matts Take Column Illustration — Molly Ferguson for STAT

What do you say to a patient when the FDA rejects “their” experimental drug — one that they believe can alleviate at least some of their suffering?

That question arises in the aftermath of a dramatic moment at the Food and Drug Administration last week. Over the FDA’s protests, a small biotechnology company called BrainStorm Cell Therapeutics had insisted that the agency evaluate its experimental treatment for amyotrophic lateral sclerosis, or ALS, brand named NurOwn. ALS patients made fervent pleas for the medicine, a type of stem cell preparation, based on reports on social media and re-analyses of the data that they thought indicated it might be of some help to those with the fatal disease.

But the FDA methodically dismantled BrainStorm’s application. The medicine couldn’t work the way the company said it would, the agency insisted; the clinical trials didn’t show it was effective; and it was not clear BrainStorm could manufacture the medicine with sufficient quality. Worst of all, more patients died among those who received the drug than those who got placebo. The FDA’s panel of expert advisers voted 17 to 1 that the medicine was not safe and effective, with one person abstaining. The agency doesn’t have to follow the panel’s advice but probably will.

Get unlimited access to award-winning journalism and exclusive events.

Subscribe Log In

Previous article： Wildfire smoke exposes gaps in outdoor worker protections
Next article： More regulation could be coming for drug ads

WSS

Startup Neumora takes a novel depression drug to Phase 3

Astronauts get first look at the spacecraft that will fly them around the moon

Medicare has a new plan to address drug shortages

Hospitals use virtual reality to design more inclusive rooms for kids

Startup Neumora takes a novel depression drug to Phase 3

Wegovy cuts risk of cardiovascular events by 20%, study finds

entertainment

hotspot

leisure time

Affirmative action in medical school literally saved lives

Germany receives 178 compensation applications from gay servicepeople over discrimination

Hepatitis C has a cure — but many Americans still lack access to it

EHR maker NextGen Healthcare to pay $31M to resolve fraud claims

Medical records are filled with copy

A year after a Russian missile took her leg, a young Ukrainian gymnast's spirit endures

Peter Hotez and the public health issue of online harassment

Hospitals use virtual reality to design more inclusive rooms for kids

Sports medicine is finally prioritizing gender equality

Medicare has a new plan to address drug shortages

Wikipedia

Astronauts get first look at the spacecraft that will fly them around the moon

STAT Virtual Event: The State of Biosimilars: What to Watch

Group opposing PBM reform sets lobbying record

CDC launches VaccineFinder tool to locate Covid

Doctors find melanoma drugs effective against rare brain cancer

Cheesesteak restaurant owner explains his secret sauce, the viral Malibu Barbie Dream Drizzle

focus

hotspot

hotspot

focus