NRG Awarded $500K by MJFF to Develop Disease-modifying Therapy

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by Vanda Pinto, PhD |

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NRG Therapeutics has been awarded a $500,000 grant by the Michael J. Fox Foundation for Parkinson’s Research (MJFF) to support the development of its potential disease-modifying therapy for Parkinson’s.

That funding will go toward investigating how small molecules, developed by NRG, inhibit the opening of mitochondrial pores, known as mPTP, and prevent dopamine-producing brain cells from dying — a process that is the hallmark of Parkinson’s disease. Mitochondria are the cellular organelles responsible for the production of energy.

“There is a significant unmet medical need as all current treatments for Parkinson’s control symptoms but do not halt or slow progression of disease,” Neil Miller, PhD, co-founder and CEO of NRG, said in a press release.

“We are delighted to have received funding from MJFF to help us advance our understanding of the molecular target for our mPTP inhibitors,” Miller said. “We are passionate about finding new treatments that offer hope and could change the lives of millions with neurodegenerative diseases.”

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The progressive death of dopamine-producing neurons is one of the key features of Parkinson’s. Loss of these cells causes symptoms such as tremors, slowness, rigidity, and lack of balance. As yet, no effective treatments that can significantly slow the disease course have been discovered.

“Despite recent successes, truly disease-modifying treatments for Parkinson’s and other neurodegenerative diseases remain the holy grail,” Miller said.

Mitochondria have several essential functions in neurons. For example, they produce large quantities of energy and, when active, they also remove calcium from brain cells. In Parkinson’s, mitochondria become overloaded with calcium. This causes a channel found on the mitochondrial membrane, called mPTP, to open and release chemicals that ultimately kill cells.

Preclinical models of Parkinson’s and other neurodegenerative diseases have suggested that targeting mPTP can be neuroprotective. However, first-generation mPTP inhibitors, such as cyclosporin A, are unable to cross the blood-brain barrier — a protective semi-permeable membrane that only allows certain substances to pass from the blood into the brain.

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NRG has discovered new second-generation mPTP inhibitors that have the ability to cross the blood-brain barrier. However, the targets of these agents when they block mPTP are unknown.

The new funding will be used to advance a project called “Novel Inhibitors of Cell Death for the Treatment of People with Parkinson’s Disease.” The project’s investigators, led by Miller and Anthony Richard Rutter, PhD, also an NRG co-founder, will attempt to understand the molecular targets of the company’s small molecules. This research will advance the progression of mPTP inhibitors into early clinical trials in people with Parkinson’s.

“Our mission is to support development of life-changing treatments for people with Parkinson’s disease, and we are pleased to provide funding for this project toward that goal,” said Marco Baptista, PhD, MJFF vice president of research programs.

In August 2020, NRG received a £1 million (about $1.4 million) funding boost from Parkinson’s UK through its drug development arm, Parkinson’s Virtual Biotech. The neuroscience company also plans to secure Series A funding to advance the development and clinical testing of its lead candidates.

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