explore -WSS

explore

author：fashion 2025-08-11 08:01:00 - browse：19

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： Psychedelics group wrestles with new pharma identity
Next article： Amylyx and the next ALS drug test, plus a chat with CRISPR's CEO

WSS

Latigo launches with $135 million for Vertex

Ebola vaccine can save some who are already sick, per new study

App could help diagnose ear infections more accurately — and at home

5 to watch: Tech startups trying to solve health care's staffing crisis

AMAs digital advisors table RPM coding reform proposal, again

Latigo launches with $135 million for Vertex

focus

explore

Wikipedia

In Memoriam: Notable people who died in 2023

U.K. researchers warn about biases in medical devices, algorithms

As demand for Covid shots wanes, Moderna seeks its next success

Neuroscience’s long legacy of racism

Jerome Adams on the need for new antivirals for Covid

STAT Boddities: What happens to the brain when we get scared?

Guidelines to prevent youth baseball injuries need more muscle

Study gives hint on South Asians’ higher risk of cardiovascular disease

New Eli Lilly Alzheimer’s data poses Medicare coverage conundrum

Having children helps parents live longer, especially after age 80

entertainment

Latigo launches with $135 million for Vertex

Health AI adoption puts security, privacy startups on investors' radar

UnitedHealth increases loan offers after Change Healthcare outage

Leading voice in opioid treatment defends methadone clinic system

Too many hospitalized Covid

Abbott's new heart device gets FDA advisory panel nod

entertainment

knowledge

knowledge

explore