Amyotrophic lateral sclerosis (ALS) is a severe, fatal neurodegenerative disorder causing loss of motor neurons and voluntary muscle action. While mouse studies have identified potential treatments, these drugs have typically done very poorly in human trials. Researchers at Boston Children’s Hospital, working in collaboration with Pfizer, now report a high-throughput target and drug discovery platform using motor neurons made from ALS patients. Using the platform, they confirmed two known targets and identified an existing class of drugs — agonists to the dopamine D2 receptor — as potential novel treatments.
The researchers, led by Clifford Woolf, MD, PhD, director of the F.M. Kirby Neurobiology Center at Boston Children’s, and first authors Xuan Huang, PhD, and Kasper Roet, PhD, in Woolf’s lab, describe the platform and their findings June 8 in the journal Cell Reports.