FMM supports research project to investigate mitochondria-targeted therapeutic approach

The Foundation for Mitochondrial Medicine (FMM) announced today that it is working with The Michael J. Fox Foundation for Parkinson's Research (MJFF) to support a research project led by Wolfdieter Springer, PhD, at the Department of Neuroscience at Mayo Clinic in Florida to investigate a mitochondria-targeted therapeutic approach to treating Parkinson's disease.

Cell death in Parkinson's and other diseases is associated with impediment of the mechanism that degrades damaged mitochondria in cells. The protein parkin plays a role in that degradation, and parkin is affected in some cases of Parkinson's by a mutation in its gene, PARK2 (genes encode proteins). Dr. Springer is researching ways to activate and stabilize the parkin protein, which may allow it to break down damaged mitochondria and thereby prevent cell death.

"Stabilizing parkin will be a positive strike for Parkinson's disease and for mitochondrial disease, and we are pleased to collaborate with MJFF," said Laura Stanley, executive director of FMM. "Our mission is to fund the most promising treatments for mitochondrial disease. Research like Dr. Springer's can unlock potential cures for many related diseases, including Parkinson's, Alzheimer's, Autism and others."

Recent data show a "closed," auto-inhibited conformation of parkin consistent with its low activity seen in Parkinson's disease. Dr. Springer proposes that parkin's auto-inhibition arises sequentially through post-translational modifications and structural rearrangements. Several therapeutic opportunities may exist along this multistep process, offering various targets to maintain or revert to the "open," active parkin conformation.

Dr. Springer's team will combine structural, computational with functional, cell-based methods to identify compounds that promote parkin activation. In subsequent follow-up studies, these could be further developed into neuroprotective agents to treat Parkinson's and mitochondrial diseases.

"Mitochondrial dysfunction is an important focus of research toward a cure for Parkinson's disease. Dr. Springer's project could have great impact, both for people living with Parkinson's and with other mitochondrial diseases," said Marco Baptista, PhD, MJFF associate director of research programs. "We are grateful to FMM for its support."