Team identifies potential target pathway for ALS

In a paper published in Science Translational Medicine, researchers at Kyoto University (Kyoto, Japan) identified the Src/ABL proto-oncogene 1 non-receptor tyrosine kinase (ABL; ABL1) pathway as a potential target to treat amyotrophic lateral sclerosis (ALS).

The group used a phenotypic screening assay to evaluate existing drugs' effect on survival of motor neurons generated from induced pluripotent stem (iPS) cells derived from an ALS patient. More than half of the compounds identified as "hits" targeted the pathway. Src/ABL inhibitors and siRNA knockdown of Src or ABL protected against motor neuron degeneration in vitro...