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author：knowledge 2025-08-12 08:26:09 - browse：55

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.

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