The Center for Neurologic Study, a nonprofit 501c3 corporation, is a leader in an area now referred to as Translational Medicine. Our most recent success is the approval of Qalsody/Tofersen for the treatment of the SOD1 familial variant of ALS. This is considered a medical milestone in the history of this disorder. Prior to this, our director and colleagues were awarded the international Healey Prize for their ALS antisense drug discovery efforts. Further, we are pleased to announce the publication of Neurotherapeutics in the Era of Translational Medicine, which Dr. Smith co-edited with Clive Svendsen and Brian Kaspar (see publications).
The seminal discovery of DMQ at CNS resulted in the development of Nuedexta. The drug, ultimately formulated by Avanir Pharmaceuticals Corporation, was approved by FDA for the treatment of labile emotionality that occurs in association with numerous neurological disorders. Recently, we demonstrated that Nuedexta enhances speech and swallowing in patients with ALS. These results were published in the Journal of Neurotherapeutics (see publications). To our knowledge, this placebo-controlled trial is the first to demonstrate the enhancement of any function in this disease. The study was funded by an award to Dr. Smith (ALS Association) who conceived and managed the study as principal investigator.
In collaboration with Amprion Inc., we have identified a self-replicating protein in the spinal fluid of approximately fifteen percent of ALS patients. While this could be an incidental finding, this subgroup of patients may represent a unique treatment opportunity. The results of this research were published in the European Journal of Neurology early in 2024 (see publications). Considering that the cause of Sporadic ALS which accounts for 90% of cases, our finding may well be as important as the discovery of genetic causes of this dreaded disease.
Our success is due in part to the support we have received from individuals, corporations and foundations. If you’ve come upon us, consider making a donation, if not to us, make a donation to the cause that concerns you.