Cure Parkinson’s funds preclinical research for drug repurposing

Initial focus will be on two types of vitamin B12 and OTC painkiller ibuprofen

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Cure Parkinson’s is funding preclinical research that intends to provide the evidence needed to determine whether three existing compounds are ready to move forward into clinical trials in people with Parkinson’s disease, the U.K. charity announced.

The funding comes as part of the recently launched Pipeline Research Acceleration Programme, which spun off the International Linked Clinical Trials (iLCT) initiative.

Within the framework of this initiative, a panel of Parkinson’s experts comes together each year to rank a list of compounds that are already in the market for other indications, but have the potential to be repurposed to slow or even stop Parkinson’s progression.

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Those that aren’t given top priority “often show potential for Parkinson’s but lack key background information … needed to justify progressing these compounds into clinical trials for people with Parkinson’s,” Cure Parkinson’s stated in the announcement.

The new Pipeline Research Acceleration Programme funds early-stage research that fills knowledge gaps in background information outlined by the expert panel to be necessary before a compound can be advanced into clinical testing for Parkinson’s.

The first research project to be funded is being led by Michael Schwarzschild, MD, PhD, a neurologist and Parkinson’s expert at Massachusetts General Hospital and a professor of neurology at Harvard Medical School.

Schwarzschild and his team are focusing on benfotiamine (a form of vitamin B1), methylcobalamin (a form of vitamin B12), and ibuprofen (a common over-the-counter pain killer and anti-inflammatory medication).

Both vitamin B1 (thiamine) and vitamin B12 help cells use glucose (sugar) for energy. Some studies have suggested they may protect against Parkinson’s, but there isn’t enough evidence to recommend their use.

It’s also been suggested that ibuprofen may reduce the risk for Parkinson’s, but the mechanisms are unclear. Schwarzschild’s team is specifically interested in knowing whether any of these three compounds can reduce alpha-synuclein buildup in Parkinson’s models.

In Parkinson’s, the alpha-synuclein protein misfolds (takes on a wrong shape) and begins to cluster into clumps that can spread from one nerve cell to the next. These clumps are toxic and build up in dopamine-producing nerve cells in the brain.

Dopamine is a chemical messenger that relays messages between nerve cells involved in movement. When alpha-synuclein clumps accumulate, they cause the dopamine-producing nerve cells to gradually die, leading to the hallmark motor symptoms of Parkinson’s.

Project results expected in 2024

The results of this project are expected in the second half of 2024. The 2023 funding round has closed, and the next funding round will open for applications in September 2024, according to Cure Parkinson’s.