hotspot -WSS

hotspot

author：fashion 2025-09-30 14:08:23 - browse：36

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： Guidelines to prevent youth baseball injuries need more muscle
Next article： How digital therapeutics firms are pivoting after Pear's bankruptcy

WSS

Ukraine and a new approach to mental health in war

FDA clears AI tool for detecting skin cancer

Think you might have Covid

Thermo Fisher plant for manufacturing gene therapies to expand

NIH director takes cautious approach to Biden drug pricing priority

Biotech venture capitalists face new demands from investors

focus

hotspot

explore

Apple is now the first public company to be valued at $3 trillion

Gilead’s Trodelvy fails in lung cancer, raises questions on ADCs

Medical leaders decry Supreme Court decision on affirmative action

Elizabeth Holmes banned from government health programs

Apple is now the first public company to be valued at $3 trillion

FDA panel to weigh approval of NurOwn, controversial ALS drug

Drug repurposing or repositioning? The language matters

Biotech lab space demand falls sharply

Food as medicine: CMS rules hamper 'prescribing' of fruits, veggies

Senate HELP committee spars over health care workforce policies

entertainment

Parkinson’s finding may lead to tests and, someday, treatments

Research misconduct: Proposed new rules face resistance

AI generated plenty of interest at JPM but not a lot of trust

Independent physicians launch a new lobbying group

Gilead’s Trodelvy fails in lung cancer, raises questions on ADCs

AstraZeneca, Daiichi breast cancer treatment succeeds in trial

leisure time

explore

focus

focus