This month, the ALS Association of America awarded a grant to AshaTheraputics: Asha Therapeutics, headquartered in DC, Laboratory Florida, to support the development of a potential therapeutic agent for ALS, ASHA-624, an inhibitor of the protein SARM1. The announcement was made in an ALS-related news article.
SARM1 is a protein that is closely involved in the degeneration of nerve fibers. Previous studies have reported that SARM1 is a risk-causing molecule for solitary ALS (sALS) and is considered a major mediator of neuroaxonal degeneration. This protein normally exists in an inactive state within the cell. However, upon injury or stress, it is activated and triggers a series of events that lead to the self-destruction of neuroaxons. Such degeneration and neuronal death can be significantly inhibited by knocking out (blocking or inhibiting) SARM1. Therefore, the development of a drug that targets SARM1 will help inhibit neuronal cell death. Neuroaxonal degeneration is a central pathological feature not only of ALS, but also of many other neurodegenerative diseases, such as Parkinson’s disease and multiple sclerosis, and therefore, this inhibitor is expected to be effective in a wide range of fields.
Good data have been accumulated in animal studies to date, and the ALS Association’s website and other news articles indicate that the first human clinical trials are expected to begin early next year.
https://www.ashatherapeutics.com/
First-in-human trial of ALS therapy ASHA-624 expected in early 2025
Reported by Nobuko Schlough, P-ALS on Nov 29, 2024
