The Center for Neurologic Study, a non profit 501C3 corporation, was founded in 1979 with the intent of helping patients and families who have been affected by incurable neurologic diseases, such as amyotropic lateral sclerosis (ALS). To accomplish this, we focus on experimental treatment and patient/family support and education. Along with others, CNS was instrumental in the development of Interferon as a treatment modality. This is now one of the standard therapies for multiple sclerosis. Furthermore, CNS has been a leader in an area now referred to as Translational Medicine; for example, the Center was the first to test a growth factor as a potential treatment for ALS. For those who have followed CNS over the years, we are pleased to announce a new direction for the foundation: the establishment of an Angel Investing fund. The purpose of the fund is to invest in seed stage preclinical drug, biomarker, and device development in the area of neurodegenerative disorders.
While supporting the research of others, we continue to work at the forefront of neurologic therapies through collaborations with biotech companies and universities. Most recently, we have been involved with the development of antisense molecules (ASOs) that could represent a major opportunity to treat heritable diseases of the nervous system, such as familial amyotrophic lateral sclerosis and Huntingtons disease. Remarkably, an ASO was just approved to treat spinal muscular atrophy, an ALS-like disease that occurs in infants. This work, undertaken by Dr. Adrian Krainer and Ionis Pharmaceuticals, is a medical milestone. If you read the scientific publications regarding this development, you would find that CNS is credited with doing some of the foundational work that led to the drug's development. Moreover, as I update this message, Biogen and Roche Pharmaceutical Corporations are conducting clinical trials for both familial ALS and Huntington's disease, again, based on early work undertaken by CNS with Ionis Pharmaceuticals and UCSD. Both I and my collaborators, Dr. Don Cleveland from UCSD and Frank Bennett from Ionis Pharmaceuticals, were recognized for this work by the ALS Association (Essey award).
The seminal discovery of DMQ at CNS resulted in the award of 5 U.S. patents. This drug combination was ultimately formulated by Avanir Pharmaceutical Corporation, leading to a product now known as Nuedexta. In clinical trials, this drug was demonstrated to be highly effective for the treatment of emotional lability that occurs in association with neurological conditions, including ALS and multiple sclerosis. In October 2010, Nuedexta was approved by the Food and Drug Administration for sale in the United States, and in 2013, it was approved in the European Union. Recently, Nuedexta was tested for its effects on behavior in persons with Alzheimer's disease. The positive results were published in the Journal of the American Medical Association (JAMA). In 2012, the ALS Association awarded CNS a grant to study the effect of Nuedexta on speech and swallowing on patients affected by ALS. The results of this study, which was conducted at 7 academic centers in collaboration with the Northeast ALS Consorteum (NEALS) and Massachussetts General Hospital (MGH), was just published (July 2017) in the Journal of Neurotherapeutics. To our knowledge, this placebo-controlled trial is the first to demonstrate the enhancement of any function in Amyotrophic Lateral Sclerosis.
Our website is the latest in a long-standing effort to provide patient and family education and support. As early as 1979, CNS sponsored monthly educational programs for patients and their interested family members and friends. While this program has been terminated, individual counseling is available for patients who visit CNS. In special circumstances, CNS will provide online counseling. We hope to provide useful guidance for patients impacted by neurodegenerative disorders.
Our success is due in part to the support we have received from individuals, corporations and foundations. We welcome the opportunity to discuss how your support will advance the treatment of presently incurable diseases such as Amyotrophic Lateral Sclerosis and kindred disorders.