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:  California man invites scientists to experiment with CRISPR on his rare disorder

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
Talking Biotech
Genetics Unzipped
Video: Test everyone – Slovakia goes its own way to control COVID

Video: Test everyone – Slovakia goes its own way to control COVID

As Europe sees record coronavirus cases and deaths, Slovakia is testing its entire adult population. WSJ's Drew Hinshaw explains how ...
mag insects image superjumbo v

Disaster interrupted: Which farming system better preserves insect populations: Organic or conventional?

A three-year run of fragmentary Armageddon-like studies had primed the journalism pumps and settled the media framing about the future ...
dead bee desolate city

Are we facing an ‘Insect Apocalypse’ caused by ‘intensive, industrial’ farming and agricultural chemicals? The media say yes; Science says ‘no’

The media call it the “Insect Apocalypse”. In the past three years, the phrase has become an accepted truth of ...
globalmethanebudget globalcarbonproject cropped x

Infographic: Cows cause climate change? Agriculture scientist says ‘belching bovines’ get too much blame

A recent interview by Caroline Stocks, a UK journalist who writes about food, agriculture and the environment, of air quality ...
organic hillside sweet corn x

Organic v conventional using GMOs: Which is the more sustainable farming?

Many consumers spend more for organic food to avoid genetically modified products in part because they believe that “industrial agriculture” ...
benjamin franklin x

Are most GMO safety studies funded by industry?

The assertion that biotech companies do the research and the government just signs off on it is false ...
favicon

Environmental Working Group: EWG challenges safety of GMOs, food pesticide residues

Known by some as the "Environmental Worrying Group," EWG lobbies for tighter GMO legislation and famously puts out annual "dirty dozen" list of fruits and ...
m hansen

Michael Hansen: Architect of Consumers Union ongoing anti-GMO campaign

Michael K. Hansen (born 1956) is thought by critics to be the prime mover behind the ongoing campaign against agricultural biotechnology at Consumer Reports. He is an ...
News on human & agricultural genetics and biotechnology delivered to your inbox.
Optional. Mail on special occasions.
Send this to a friend