The ALS Therapy Development Institute (ALS TDI), together with its wholly-owned subsidiary, Anelixis Therapeutics, announced today that it has formed a research partnership with Neurimmune to advance potential treatments for ALS, also known as Lou Gehrig's disease. The research partnership will explore a human monoclonal antibody that targets misfolded superoxide dismutase 1 (SOD1). Misfolded SOD1 accumulates in familial and sporadic ALS, suggesting a generalized role as a drug target for ALS treatments.
"We are excited by the results of Neurimmune's findings," says Steve Perrin, Ph.D., CEO & CSO of ALS TDI. "Recent evidence would suggest that therapies targeting misfolded SOD1 could be important in both sporadic and familial forms of ALS. It is crucial that we use every tool available to us to advance potential treatments toward the clinic, and we are proud to work together with Neurimmune to attempt to do just that."
The collaboration will explore the high-affinity human antibodies developed by Neurimmune that selectively target misfolded SOD1. In preclinical studies with transgenic mice, chronic antibody treatment significantly reduced SOD1 pathology and rescued spinal cord motor neurons, resulting in significantly reduced muscle atrophy, better motor functions and increased survival. Neurimmune will present the key findings of their approach at the International Symposium on ALS/MND Research in Milan, Italy on December 7, 2013.
"ALS is a devastating neuromuscular disease, and there is an urgent need for new and effective therapies that can slow or stop its progression," says Jan Grimm, Ph.D., CSO of Neurimmune. "Our human antibodies are directed against pathologically misfolded SOD1 and the selected lead candidate shows marked efficacy in independent ALS animal models. We believe that there is significant potential for this therapeutic approach for ALS and are enthusiastic to jointly advancing the program towards clinical development together with ALS TDI."
ALS Therapy Development Institute