Timothy Miller, MD, PhD, the David Clayson Professor of Neurology at Washington University School of Medicine in St. Louis and director of the university’s ALS Center, and a group of his colleagues have received the inaugural Healey Center International Prize for innovation in amyotrophic lateral sclerosis (ALS) research from the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital.
The prize celebrates excellence in research by an individual or team that has made exceptional discoveries leading to a transformative advance in therapy development in ALS, also known as Lou Gehrig’s disease.
The prize was awarded to Miller and colleagues Don Cleveland, PhD, of the Ludwig Institute at the University of California at San Diego; Richard Smith, MD, of the Center of Neurological Study in La Jolla, Calif.; Toby Ferguson, MD, of Biogen; and Frank Bennett, PhD, of Ionis Pharmaceuticals for their discovery and development of a new way to treat a genetic form of ALS caused by a mutation in the gene SOD1. The team used DNA-like molecules called antisense oligonucleotides to reduce levels of dangerous SOD1 proteins. The therapy proved effective in animals, and clinical trials in people are now underway.
The team’s pioneering work with an oligonucleotide-based therapy has led to similar experimental therapies for other forms of ALS and other neurological disorders such as spinal muscular atrophy and Huntington’s disease.