- Amyotrophic lateral sclerosis (ALS) is a neurologic disease in which neurons die off and all muscles in the body fail, eventually leading to death. It affects roughly 30,000 people in the U.S. and there is currently no cure.
- There have been numerous failures in ALS R&D, including Biogen's drug, despramipexole, which failed in early 2013.
- The goal of a new collaboration between Biogen, which is based in Cambridge, MA, and the Columbia University Medical Center (CUMC) in New York City is to better understand the genetic underpinnings of ALS.
Biogen's CEO, George Scangos, called the demise of the despramipexole program a "spectacular failure," but that hasn't stopped this influential biotech's determination to continue addressing the unmet medical need associated with ALS.
The collaborative research project between Biogen and CUMC, which is slated to last three years, is focused on studying the genetic sequences of current ALS patients. The goal is to uncover the mutations driving the disease.
It just so happens that August is the month when the annual ALS Ice Bucket Challenge takes place. Last year, despite heavy skepticism, the effort (which involves making a donation and/or "challenging" others to dump buckets of ice water over their heads) successfully raised a staggering $220 million worldwide. About $3.5 million out of that total will be used to fuel the Biogen/Columbia collaboration.