A pre-clinical pipeline of potential first-in-class brain-penetrant small molecule inhibitors of the mitochondrial permeability pore (mPTP) are to be developed. These will act through a novel mechanism of action and be used in treatment of Parkinson’s and Amyotrophic Lateral Sclerosis (ALS), also known as motor neurone disease (MND).
Mitochondria are seen as the powerhouse or as batteries of cells, as they are essential for maintenance of cell health, and their failure or dysfunction has been noted to be common across many degenerative diseases. Inhibition of the mPTP has been shown, however, to protect neurons, reduce neuroinflammation, and extend survival in pre-clinical disease models.
