BioCentury
ARTICLE | Targets & Mechanisms

TBK1 and beyond

Biogen and partners link mutant TBK1 to autophagy and neuroinflammation in ALS

March 12, 2015 7:00 AM UTC

Biogen Idec Inc.'s partnership with Columbia University and the HudsonAlpha Institute for Biotechnology has yielded a new target in ALS - TBK1 - that provides a link between autophagy, neuroinflammation and the pathology of the disease. While TBK1 mutations only occur in about 1% of cases, the partners believe targeting the enzyme will address degeneration caused by other defects in its pathway, and could end up helping a bigger section of the ALS population.

"The percentage of patients with TBK1 mutations is only part of the story," said David Goldstein. "There's every reason to believe that treatments developed for molecularly defined subsets can affect other patients without that specific mutation." ...