July 2017 – Publication in Neurotherapeutics

Publication of Enhanced Bulbar Function in ALS: The Nuedexta Treatment Trial in Neurotherapeutics, 14(3), 762-772.

The recently published article is noteworthy in that the placebo-controlled multi-centered study demonstrated enhanced speech and swallowing in response to treatments with Nuedexta which was approved in 2011 for the treatment of labile emotionality that occurs in ALS and kindred disorders. To our knowledge, this is the first controlled study to show improvement of function in ALS patients using a therapeutic agent. If confirmed in a longer and larger study, Nuedexta is likely to play an important role in the treatment of ALS in addition to its effect on labile emotionality.

October 2016 – Antisense Patent for the Treatment of Familial ALS

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.

September 2016 – Scripps Clinic and Research Foundation Grand Rounds

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.