Late stage pre‐clinical development of a small molecule compound for re‐myelination and functional recovery in multiple sclerosis through disruption of the AMPA‐GAPDH complex
While it is the immune system that damages the brain in MS, the way this damage is caused involves many processes. One such process is called excitotoxicity, which is when the chemicals brain cells use to communicate can become toxic to those cells. We have a new drug which stops excitotoxicity without also preventing the ability of brain cells to communicate. This drug can restore movement and myelin in animal models of MS. Animal models for MS include a method called EAE that tricks the animal’s immune system into attacking myelin, and exposure to cuprizone, which causes myelin loss by removing copper from the body. While immune modifying drugs are able to prevent loss of myelin and motor function in animal models when given before symptoms appear, our treatment was effective even after symptoms began progressing in these animals. This makes us hopeful that our drug can help restore function and myelin in people who are at later stages of MS. Our drug represents a new approach for treating MS that targets a different system from existing immune modifying treatments, and could potentially be used in combination with those treatments to help patients with this disease. We aim to perform the necessary experiments to make our drug ready for human clinical trials. Much of this work has been done already. Our goals for the current proposal are to optimize the dose and treatment strategy of our drug, understand how our drug affects the cells which produce myelin, see if our drug can affect more easily measured clinical outcomes in animal models of MS, and test the performance of our drug against known indicators of potential side effects. These experiments will allow us to make a case for approval to do human trials.
