The one-year grant of USD 300,000 was awarded under the Foundation's therapeutics development initiative aimed at supporting pre-clinical development of Parkinson’s disease therapies that have the potential to alter disease course and improve the treatment of symptoms beyond current standards of care.
“Our Foundation is committed to finding a disease-modifying therapy for Parkinson’s disease, the major unmet need for those living with Parkinson’s today,” said Mark Frasier, PhD, director of research programs MJFF. “Domain Therapeutics’ metabotropic glutamate receptor III (mGluR3) program has the potential to provide new insight into the development of treatments that could slow the progression of the disease.”
Parkinson’s disease results from a progressive loss of brain cells that produce dopaminergic neurons. Current marketed drugs for Parkinson’s disease help patients manage many of their symptoms but do not prevent disease progression. Compounds acting as mGluR3 Positive Allosteric Modulators (PAMs) constitute a novel and promising neuroprotective mechanism of action through the activation of glial cell line-derived neurotrophic factor (GDNF), an endogenous protein capable of protecting and promoting the survival of dopamine neurons. In addition to its robust neuroprotective activity, GDNF has already been shown to exert motor improvements both in pre-clinical models and in the clinic. However, as it is a relatively large protein, GDNF’s clinical use has been restricted. The possibility of enhancing striatal GDNF levels with orally bioavailable small molecules could, therefore, be a breakthrough in the treatment of Parkinson’s disease.
"We are grateful for The Michael J. Fox Foundation's support of our mGluR3 PAM program. This award validates the relevance of our innovative approach to developing novel treatments for Parkinson’s disease and our expertise in the field,” said Pascal Neuville, PhD., chief executive officer of Domain Therapeutics. "With no less than three programs in the field of Parkinson’s disease, including one partnered with Merck-Serono, we are committed to bringing forward innovative drug candidates with disease-modifying potential for the benefit of Parkinson’s disease patients."