New ALS Gene Discovered with UMass Medical Leadership

Researchers have identified a new ALS gene called KIF5A. This important gene discovery, funded by The ALS Association through Ice Bucket Challenge donations, will help researchers understand ALS disease pathways and represents a potential new ALS therapeutic target.

This was a large collaborative effort involving over 250 researchers, led by John Landers, Ph.D., Professor of Neurology at University of Massachusetts Medical School in Worcester and Bryan Traynor, M.D., Ph.D., senior investigator in the Laboratory of Neurogenetics at the National Institute on Aging at the NIH in Bethesda, M.D. The discovery was published in the March 21 edition of the journal Neuron.

ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within five years of diagnosis. For unknown reasons, veterans are twice as likely to develop ALS as the general population. There is no cure.

“It was truly a pleasure to see the collaborative effort of so many groups coming together to help with our goal of identifying a novel ALS gene,” said Dr. Landers. “It demonstrates that any single group may be able to make significant findings on its own but together we can accomplish boundless advances.”

“This collaborative discovery of KIF5A gives greater insight into the cellular mechanisms that lead to motor neuron degeneration and identifies important new targets for future gene therapies,” said Dr. Traynor. (Source: ALS Association Website, 21 March, 2018)