Gladstone scientists discover drug candidate for Alzheimer's, Huntington's disease

Scientists at the Gladstone Institutes, an independent and non-profit biomedical-research organization based in San Francisco, USA, have identified a drug candidate that diminishes the effects of both Alzheimer's disease and Huntington's disease in animal models, offering new hope for patients who currently lack any medications to halt the progression of these two debilitating illnesses.

Gladstone investigator Paul Muchowski has identified a new compound called JM6 in experiments done in collaboration with an international team of researchers, and which were published last week in an on-line article in Cell. In laboratory tests involving mice genetically engineered to model one or the other of the two diseases, Dr Muchowski's team found that JM6 blocks kynurenine 3-monooxygenase (KMO), an enzyme that has long been speculated to play a role in neurodegenerative diseases.

In mice modeling Alzheimer's disease, the novel compound prevented memory deficits and the loss of synaptic connections between brain cells - both of which are key features of the human disease. In mice modeling Huntington's disease, JM6 prevented brain inflammation and the loss of synaptic connections between brain cells, while also extending lifespan.