NIH grant to Vincere supports work into way of helping mitochondria

Company to test small molecules that promote health of cell's energy source

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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An illustration shows a close-up view of mitochondria, known as the powerhouse of a cell.

Vincere Biosciences has been given $700,000 in seed funding by the National Institutes of Health (NIH) to advance in testing USP30 small molecule inhibitors, its candidate therapy aiming to slow or stop progression in Parkinson’s disease.

Valid for two years, the Phase I Small Business Innovation Research grant by the NIH’s National Institute of Neurological Disorders and Stroke (NINDS) will be used to test these inhibitors in models of the disease.

“We are thankful to the expert reviewers at NINDS for supporting our efforts to test our small molecules in models of Parkinson’s disease,” Bahareh Spring Behrouz, PhD, CEO of Vincere, said in a company press release.

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In Parkinson’s disease, mitochondria sustain damage they’re unable to shed

USP30 is an enzyme — a type of protein — sitting on the membrane of mitochondria, the cells’ powerhouses. The enzyme counters mitophagy, slowing the process that clears damaged mitochondria from cells.

Problems with mitophagy and other mechanisms involved in the control of mitochondrial quality are thought to drive the loss of dopamine-producing nerve cells. The chemical dopamine plays a key role in movement control, and its loss is the main culprit behind Parkinson’s symptoms.

“By inhibiting or reducing USP30, we can enhance the mitophagy process, thereby reducing the amount of damaged mitochondria and resuming cellular health,” Vincere states on a company webpage.

The grant will support studies testing how well Vincere’s USP30 small molecule inhibitors work, as well as their pharmacokinetics, the movement of a medication into, through, and out of a body.

For these studies, the company will team with Michael Henderson, PhD, a neuroscientist with expertise in Parkinson’s, and an assistant professor at the Van Andel Institute’s Department of Neurodegenerative Science in Grand Rapids, Michigan.

Henderson’s team developed lab-made, preformed fibrils of the alpha-synuclein protein to model Parkinson’s. Alpha-synuclein in the disease builds into toxic clumps that damage mitochondria, ultimately killing nerve cells.

Scientists expect that the model can help in understanding how the disease-causing alpha-synuclein protein seeds and spreads through the brain, and in testing investigative medications aiming to protect dopamine-producing nerve cells.

“I’m thrilled to embark on this collaborative research journey with Vincere Biosciences as we work together to unlock new possibilities in the fight against Parkinson’s disease,” Henderson said.

NIH’s small business research grant program supports U.S. companies with 500 or fewer employees in developing their technologies and innovative science, with a goal of bringing new products to the marketplace.