Mitochon Wins MJFF Grant to Advance Mitochondrial Research in Parkinson’s

Mitochon Pharmaceuticals has received a grant from the Michael J. Fox Foundation for Parkinson’s Research (MJFF) to help develop its mitochondrial targeted compounds for treating Parkinson’s disease.

The grant, whose amount wasn’t disclosed, will fund animal studies to evaluate the benefits of MP101 and MP102 in making up for dopaminergic neuron loss, the hallmark of Parkinson’s. If studies are successful, these compounds could be available to patients as early as 2019.

MP101 and MP201, once-a-day oral drugs, specifically target the mitochondria to provide broad neural protection. Both have been shown to protect cells from damage caused by a host of degenerative processes – genetic, non-genetic, autoimmune and those caused by injury.

In preclinical trials of disease models, these therapies showed protective and functional benefits including brain volume sparing in Huntington’s disease; axonal protection from demyelination (loss of the protective layer around neurons) in multiple sclerosis, and dopaminergic neuron loss in Parkinson’s.

Mitochon, which is based in Radnor, Pennsylvania, plans to launch Phase 1 studies in healthy volunteers in 2018; Phase 2 studies are expected by 2019.

“We are delighted to receive funding from the Michael J. Fox Foundation, the preeminent institution in the field of Parkinson’s research,” Robert Alonso, co-founder and CEO of Mitochon, said in a press release. “By harnessing the power of the mitochondria, our compounds are able to protect cells from the destructive effects of age-related degeneration.”

Added Dr. Shalini Padmanabhan, associate director of research programs at MJFF: “Abnormalities in the mitochondrial processes can have severe consequences for the function and survival of dopamine neurons in people with PD. The Foundation supports Mitochon’s work toward discovery and development of mitochondrial-targeted therapies to speed breakthroughs for patients.”

A special relationship exists between mitochondria and Parkinson’s. Mitochondria are organelles with complex features that play important cellular functions, such as producing energy or controlling programmed cell death. Alterations in mitochondrial biology can lead to neuron dysfunction and cell death.

In addition, researchers at England’s University of Newcastle Upon Tyne discovered that Parkinson’s patients have lower amounts of mitochondrial DNA (mtDNA) in both the blood and the brain. Their study, “Reduced mitochondrial DNA copy number is a biomarker of Parkinson’s disease” — which appeared in the journal Neurobiology of Aging — showed substantially lower mtDNA levels in the white blood cells of Parkinson’s patients than in controls.