Center for Neurologic Study

RESEARCH: WORK UNDERTAKEN AT CNS PLAYED A ROLE IN THE INTRODUCTION OF INTERFERON FOR THE TREATMENT OF MULTIPLE SCLEROSIS. THE CENTER PARTICIPATED IN NUMEROUS NATIONAL, EXPERIMENTAL TREATMENT TRIALS INVOLVING AMYOTROPHIC LATERAL SCLEROSIS, ALZHEIMER'S DISEASE, NEUROPATHY WHICH LED TO THE DISCOVERY OF NUEDEXTA WHICH HAS BEEN APPROVED FOR USE IN THE UNITED STATES AND EUROPE. REMARKABLY, THE DRUG HAS IMPROVED FUNCTION IN BOTH ALZHEIMER'S DISEASE AND ALS PATIENTS, WITH STUDY RESULTS PUBLISHED IN MAJOR SCIENTIFIC JOURNAlS. PIONEERING WORK DONE AT CNS IN COLLABORATION WITH THE UNIVERSITY OF CALIFORNIA AND IONIS PHARMACEUTICALS IS WIDELY BELIVED TO BE THE MOST PROMISING STRATEGY FOR TREATING NEURODEGENERATIVE DISEASES SUCH AS HUNTINGTON'S DISEASE AND FAMILIAL AMYOTROPHIC LATERAL SCLEROSIS. THIS STRATEGY INVOLVES THE SYNTHESIS OF SMALL MOLECULES THAT DOWN REGULATE THE EXPRESSION OF MUTANT PROTEINS IN THE BRAIN. THE LATTER RESULTS WERE PRESENTED AT THE 2018 ANNUAL MEETING OF THE AMERICAN ACADEMY OF NEUROLOGY.

Public education: research conducted at cns has been presented publically at medical and scientific meetings and published in leading scientific journals. These activities have been reported upon in local and national media, including nbc and cnn television, the san diego union tribune, the los angeles and new york times and scientific american. Cns also provides information about neurological diseases to patients, physicians and the general public via it's website (cnsonline.org). Additionaly cns is involved in organizing an annual conference on amyotrophic lateral sclerosis in collaboration with academic institutuions in California (California als summit).

The Center for Neurologic Study is a leader in translational medicine. The approval of qalsody for the treatment of the sod1 familial variant of als is a recent example, leading to a healey prize for the director's drug discovery efforts and leading to the publication of neurotherapeutics in the era of translational medicine. The seminal discovery of dmq at cns, ultimately formulated by avanir pharmaceuticals corporation as nuedexta, was approved by fda in 2010 for the treatment of emotionality that occurs with numerous neurological disorders. Its therapeutic effect on speech and swallowing was recently published in neurotherapeutics, the first therapy to enhance any function in this disease. The Study was funded by the als association. This year the european journal of neurology published the Center's finding of a self-replicating protein in the spinal fluid of approximately fifteen percent of als patients. This surprising result could lead to a breakthrough treatment.