This technology identifies mitochondria-associated membrane functionality as a promising therapeutic target for amyotrophic lateral sclerosis (ALS).
Amyotrophic lateral sclerosis (ALS) is a hereditary fatal neurodegenerative disorder characterized by progressive muscle atrophy from motor neuron loss. While there are currently seven FDA-approved drugs for ALS treatments, these currently are limited to targeting mutant protein pathways or reactive oxygen species (ROS) buildup and only mildly relieve symptoms without effectively preventing disease. Therefore, there is a need for ALS therapies that effectively block disease progression to improve patient morbidity and quality of life.
This technology proposes restoration of mitochondria-associated membrane (MAM) function as a potential therapy for amyotrophic lateral sclerosis (ALS). MAM dysfunction was identified in mouse models of ALS with the SOD1 mutation, resulting in increased cellular toxicity and leading to motor neuron death. Since MAM dysfunction is a primary event in ALS development and a direct driver of muscle atrophy, therapies that target and restore MAM function can potentially be an effective approach to halt ALS progression in patients.
This technology has been validated in mouse ALS models.
Patent Pending
IR CU23335
Licensing Contact: Kristin Neuman