FDA advisers vote against experimental ALS treatment pushed by patients

abcnews.go.com:

Federal health advisers have voted overwhelmingly against recommending approval of an experimental treatment for Lou Gehrig's disease, the fatal muscle-wasting disease

WASHINGTON — Federal health advisers voted overwhelmingly against an experimental treatment for Lou Gehrig’s disease at a Wednesday meeting prompted by years of patient efforts seeking access to the unproven therapy.

The panel of Food and Drug Administration experts voted 17-1 that drugmaker Brainstorm’s stem cell-based treatment has not been shown effective for patients with the fatal, muscle-wasting disease known as ALS, or amyotrophic lateral sclerosis. One panel member abstained from voting.

While the FDA is not bound by the vote, it largely aligns with the agency’s own strikingly negative review released earlier this week, in which staff scientists described Brainstorm’s application as “scientifically incomplete” and “grossly deficient.”

«Creating false hope can be considered a moral injury and the use of statistical magic or manipulation to provide false hope is problematic,” said Lisa Lee, a bioethics and research integrity expert from Virginia Tech, who voted against the treatment. The lone positive vote came from a panel member representing patients.

Wednesday's public meeting was essentially a longshot attempt by Brainstorm and the ALS community to sway FDA’s thinking on the treatment, dubbed NurOwn.

Brainstorm’s single 200-patient study failed to show that NurOwn extended life, slowed disease or improved patient mobility. But FDA agreed to convene the panel of outside advisers after ALS patients and advocates submitted a 30,000-signature petition seeking a public meeting.

In the last year, the FDA has approved two new drugs for ALS, after a