Tag Archives: als

Amylyx may get rare second chance with FDA panel

From left to right: Amylyx Co-founders and Co-CEOs Josh Cohen and Justin Klee (Photo courtesy of Amylyx)

A biotechnology company expects to get a rare second chance to try to win the support of a key federal panel for an experimental treatment, in this case, a medicine intended to treat a devastating neurological condition, amyotrophic lateral sclerosis, commonly known as ALS.

Amylyx Pharmaceuticals Inc. on July 5 announced that it expects the Food and Drug Administration (FDA) to hold an advisory committee meeting on Sept. 7 about the company’s experimental ALS treatment. The Cambridge, Massachusetts-based company said it expects discussion at this meeting to focus on additional analyses of data from research done on the treatment or AMX0035. (AHCJ has created a tip sheet for covering FDA advisory committee meetings held about experimental medicines or new uses of approved drugs. 

The FDA has a target date of Sept. 29 for deciding on whether to clear AMX0035. This deadline was extended from June 29 to allow more time for the FDA to review additional analyses of data, Amylyx said. The company suffered a setback in March when the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 6-4 to say that evidence gathered to date had not proven the treatment works. The FDA considers the recommendations of its advisory panels, but it is not bound by them.

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Think of artificial intelligence as city planning, not building construction, panel says

Artificial intelligence (AI) is one of the hottest topics in health care and it has the potential to transform care delivery in hospitals, clinics and in the home.

The Health Journalism 2019 panel “Hope vs. Hype: Reporting on AI” offered some examples where AI is being applied to medical care today. It also tapped the brakes on some of the hype. Continue reading

Concussion-related trauma masquerades as ALS

The New York TimesAlan Schwarz reports on what he says is “the first firm pathological indications that brain trauma results in motor-neuron degeneration.” The headline behind that conclusion, of course, is that researchers say some  athletes with concussion and impact-induced brain injuries may have been misdiagnosed as ALS victims.


Photo by peterjr1961 via Flickr

In interviews, the study’s authors even speculate that Lou Gherig, who gave the disease its popular name, may have instead suffered from a similar disease caused in part by brain injuries.

The finding was not unexpected, given that ALS seemed to occur at much higher rates in concussion-heavy populations like athletes and soldiers.

Schwarz’s summary of the study:

Doctors at the Veterans Affairs Medical Center in Bedford, Mass., and the Boston University School of Medicine, the primary researchers of brain damage among deceased National Football League players, said that markings in the spinal cords of two players and one boxer who also received a diagnosis of A.L.S. indicate that those men did not have A.L.S. at all. They had a different fatal disease, doctors said, caused by concussionlike trauma, that erodes the central nervous system in similar ways.

It’s in the emergence of that second disease that really seems to have attracted Schwarz’ attention. It behaves similarly to ALS, but shows a distinct protein pattern that only seemed to emerge in patients with a history of head injury. There is not, however, a 1:1 relationship. Other factors seem to also be at play, Schwarz writes.

Gary Schwitzer of HealthNewsReview.org examines the story more closely and concludes that, while this is an “important and fascinating area of research,” the story “did not exhibit the best of health/medical/science journalism.” He lists seven points of criticism and includes comments from one of the site’s medical editors.

John Gever of MedPage Today offers more scientific coverage of the study and points out that there was no mention of Gehrig in the study but that “a New York Times reporter coaxed McKee into suggesting that Gehrig may have been among those misdiagnosed – even though, as a first baseman, he did not routinely experience violent collisions. (He was, however, beaned at least twice during his 14-year career with the New York Yankees.)”