Kinase for ALS

About

Background: Amyotrophic Lateral Sclerosis (ALS), also termed Lou Gehrig’s disease, is a neurological disease that affects nerve cells in the brain and spinal cords which controls the activity of voluntary muscles. There are several causes of ALS which include gene mutation, disorganized immune response, and chemical imbalance, and symptoms for ALS mainly include fasciculation, respiratory problems, muscles weakness, and cramps. Although ALS disease is rare—only affecting 1 or 2 out of every 100,000 people worldwide—the mortality rate among ALS patients is high with an average patient survival time of 3 years. Current treatments can’t reverse the damage of ALS and can only slow the disease’s progression and reduce patients’ symptoms. The current invention is a novel ALS treatment that has been shown to halt and even reverse the onset of ALS. This invention uses the protein PAS Kinase to block the activity of the protein associated with ALS disease, Ataxin-2, as well as inhibit this protein’s gene from further expression. This technology can be synthesized into a consumer-friendly once daily oral treatment, providing a new and much needed therapy option for ALS sufferers. About the Market: The increasing numbers of patients affected with ALS worldwide will be a major factor driving growth in the ALS treatment market for the next 25 years. In fact, in the developing world the number of ALS cases is expected to increase by an estimated 50% from 2015 to 2040, and cases in developed countries such as the United States are projected to increase by about 24% during the same time. The strong market demand for new ALS treatments and therapies has been popularized by the “ALS Ice Bucket Challenge”—a fundraising initiative that recently went viral on the internet and social media and that has involved over 17 million participants, raising over $115 million for ALS research. Riluzole, manufactured by Rilutek, has long been the only ALS drug on the market approved by the FDA, but since the drug’s patent expiration in 2013, the market has become flooded with generic versions of the drug. However, these drugs’ treatment regimens can only be expected to prolong ALS sufferers’ life expectancies by about 6 months, creating an opportunity for a new and more effective drug using this technology to disrupt the market.