The ALS Association's Translational Research Advancing Therapies for ALS (TREAT ALS™) program provided funding at each stage of development for this therapy, beginning with establishing the proof of concept in pre-clinical work. That work was led by Don Cleveland, Ph.D., and Richard Smith, M.D., both of the University of California at San Diego, along with Dr. Miller, Dr. Cleveland's lab, and Frank Bennett, Ph.D., of Isis Pharmaceuticals, developer of the antisense molecules used in the research program. Merit Cudkowicz, M.D., of Massachusetts General Hospital, was co-investigator of the clinical trial.This first-in-human trial of an antisense therapy for a neurodegenerative disease is the culmination of years of work by scores of people from academia and biotechnology, working in partnership to stop ALS," Dr. Bruijn said. "We still have much work to do before we know whether antisense therapy can offer benefit to patients with SOD1 mutations, but the evident safety of the procedure is very encouraging and should allow larger and longer trials in the near future that can tell us more about the potential of this form of treatment."
Therapy Funded By The ALS Association Involving Injection Of Antisense Molecules Into Spinal Cord Area Of People With Lou Gehrig's Disease Deemed Safe In Recent Study
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