hotspot -WSS

hotspot

author：comprehensive 2025-09-03 18:40:41 - browse：3

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： In age of alternative facts, a scholarly course on calling out crap
Next article： What does generative AI mean for health care? We asked experts

Wikipedia

WSS

Alabama IVF ruling threatens families facing fatal genetic disease

In utero gene editing: an explainer

Affirmative action in medical school literally saved lives

Medical records are filled with copy

Neurocrine Biosciences, biotech based in San Diego, joins PhRMA

United colonoscopy coverage change 'may cost lives,' doctors say

knowledge

hotspot

explore

What does generative AI mean for health care? We asked experts

Medical leaders decry Supreme Court decision on affirmative action

Bright Health sells Medicare Advantage business to Molina

R1 RCM gets $5.8 billion buyout offer from private equity firm

Drug repurposing or repositioning? The language matters

A new nitrogen execution bill also undermines medical licensing

Indiana Supreme Court upholds abortion ban, says state constitution gives only limited protections

Alabama frozen embryo ruling has major implications for IVF clinics

Walensky prepares to leave CDC — and Congress — behind

Alabama frozen embryo ruling has major implications for IVF clinics

hotspot

Anesthesiologist group: stop taking Ozempic before surgery

A new nitrogen execution bill also undermines medical licensing

The Supreme Court will review a ruling striking down a domestic violence federal gun ban

Continuity nursing helped my family in our darkest PICU moments

Alabama IVF ruling threatens families facing fatal genetic disease

Readout Newsletter: Wegovy, dialysis, Humira, Vir, and more

entertainment

explore

Wikipedia

hotspot