Newly discovered ALS gene may lead to new drug targets

A new gene associated with sporadic amyotrophic lateral sclerosis—or Lou Gehrig's disease—has been identified by an international consortium of scientists and clinicians including Biogen Idec, Columbia University Medical Center and HudsonAlpha Institute of Biotechnology, Biogen announced Thursday. The gene—TBK1, or TANK-Binding Kinase 1—appears to play a critical role in two significant cellular pathways: inflammation and autophagy. The gene was discovered through advanced DNA sequencing methods. A study documenting the findings was published Thursday in Science.


Lucie Bruijn, chief scientist of The ALS Association, said of the findings: “The fact that TBK1 accounts for 1% of ALS adds significantly to our growing understanding of the genetic underpinnings of the disease. This study, which combines the efforts of over two dozen laboratories in six countries, also highlights the global and collaborative nature of ALS research today.” Researchers are currently using stem cells to study the disease and vet drug candidates. “Several compounds that affect TBK1 signaling have already been developed for use in cancer, where the gene is thought to play a role in tumor cell survival,” according to Biogen's media release.