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."
The ALS Association is grateful to have received funding to support this new groundbreaking research from the following:
The Kanter Family ALS Research Fund and The ALS Association Greater Philadelphia Chapter The Jeff Kaufman Fund and The ALS Association Wisconsin Chapter The Wallace Genetic Foundation and Mrs. Jean Wallace DouglasThe George Yardley Company, the family of George Yardley, and The ALS Association Orange County Chapter Contributors to the Lou Gehrig Challenge campaign of The ALS AssociationAbout The ALS AssociationThe ALS Association is the only national non-profit organization fighting Lou Gehrig's Disease on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure. For more information about The ALS Association, visit our website at www.alsa.org. SOURCE The ALS Association