New ALS Therapy in Clinical Trial Shows Promise

Washington University School of Medicine (WUSM) St. Louis a recently released in Science Daily, points to a promising amyotrophic lateral sclerosis (ALS) study.  Also known as “Lou Gehrig’s disease,” few survive more than a few years after diagnosis.  After a successful pre-clinical study on mice and rats, WUSM published their findings in the Journal of Clinical Investigation. As reported in the press release, the results “have led to a phase one/two clinical trial to investigate whether the drug could benefit people with ALS whose disease is caused by mutations in a gene called SOD1.”

Presently, the FDA has only approved two treatments for ALS and according to the WUSM release “both only modestly slow the course of the disease.”  WUSM collaborated with Ionis Pharmaceutical on the research effort.

TrialSite News refers readers to the listing:

The actual study drug BIIB067 is the focus of the phase I study sponsored with Biogen with Ionis Pharmaceuticals as a collaborator. WUSM and dozens of other investigation sites will be involved with the actual study.  For more on BIIB067 see

Research/Investigative Site

Washington University School of Medicine, et al

Lead Research/Investigator

Timothy Miller, MD, PhD, the David Clayson Professor of Neurology of Washington University



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