The United States Patent Office issued a key patent that describes the development of a novel therapy for a treatment of one form of familial ALS. The Center was instrumental in conceptualizing this method of treating ALS and similar ailments. The multi-year effort to demonstrate the utility of this approach was undertaken in collaboration with Ionis Pharmaceutical Corporation and the University of California, San Diego. Currently, BioGen Pharmaceuticals is conducting a Phase I study to test the safety and pharmacologic parameters of this drug in a group of ALS patients with a mutation that affects the function of an enzyme that has been associated with one form of ALS. The patent can be found here.
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On September 28, 2016, Dr. Smith presented Grand Rounds at Scripps. He discussed his work on the development of therapeutics for the treatment of neurologic disease.
Publication of ALS: Is the Spinal Fluid Pathway Involved in Seeding and Spread? in Medical Hypotheses, 85(5), 576-583.
Dr. Smith, along with colleagues John Ravits (UCSD) and Robert Bowser (Barrows Neurologic Institute), recently authored a paper that advanced the idea that the spinal fluid pathway may play a key role in the development and spread of ALS. The article was published in Medical Hypotheses and can be found here.
Nuedexta Treatment Trial
CNS is pleased to report that the recently completed Nuedexta ALS treatment trial was a resounding success. The nationwide study, conducted at seven NEALS research sites, convincingly demonstrated that Nuedexta can enhance speech and swallowing in ALS patients and facilitate their ability to control their oral secretions. The results of the trial will be presented at the upcoming International ALS meeting.
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