ALS may soon be treatable with new drug combo dreamed up by a college student

death with dignity main kh c a fc d a ee ba cf b b f
Credit: Benjamin Rasmussen/NBC

Patients who took [an experimental medication for ALS] — initially dreamed up over beers and obsessive internet searching in a Brown University dormitory — retained a higher level of certain motor functions than those given a placebo, according to the researchers’ study, published [September 2] in the New England Journal of Medicine. The company developing the drug, Cambridge, Mass.-based biotech Amylyx, released outlines of the data in December, but the new paper details how effective the treatment was in slowing progression of the disease.

While researchers involved in the study said it marked a watershed moment in the fight against ALS, an accompanying editorial in NEJM called the data from the Phase 2/3 study only “tantalizing.” It said the benefit appeared to be modest — and stressed a Phase 3 trial would be important to validate the conclusions.

Related article:  Viewpoint: Trump's proposed drug price cap would threaten gene therapies, cell-based therapies by stifling innovation

Any additional treatments would be welcome by the ALS community. There is no cure for the disease, and only a couple of available treatments.

Follow the latest news and policy debates on agricultural biotech and biomedicine? Subscribe to our newsletter.

“When they first wrote to me in June 2013, they were just kids,” [Alzheimer’s researcher Rudy] Tanzi recalled. “I thought it was a pretty naive idea, to be honest, but thought I’d go through this exercise with them — throw them some hard-to-do science. And now here we are, today, with a paper coming out.”

Read the original post

Outbreak
Outbreak Daily Digest
Biotech Facts & Fallacies
Genetics Unzipped
Infographic: How dangerous COVID mutant strains develop

Infographic: How dangerous COVID mutant strains develop

Sometime in 2019, probably in China, SARS CoV-2 figured out a way to interact with a specific "spike" on the ...
Untitled

Philip Njemanze: Leading African anti-GMO activist claims Gates Foundation destroying Nigeria

Nigerian anti-GMO activist, physician, and inventor pushes anti-gay and anti-GMO ...

Most Popular

News on human & agricultural genetics and biotechnology delivered to your inbox.
glp menu logo outlined

Newsletter Subscription

Optional. Mail on special occasions.
Send this to a friend