Researchers ID compound that may slow Parkinson’s progression

Researchers at Florida International University (FIU) have identified a compound that boosts the function of mitochondria (cells’ powerhouses) and may slow the progression of Parkinson’s disease.

The scientists found that by reducing the activity of a protein called dynamin-related protein 1 (DRP1), which regulates mitochondrial function, the compound, CTS2444-32, reduced the buildup of toxic protein clumps and inflammation — two processes closely linked to Parkinson’s disease.

“Unlike current treatments that simply replace the missing chemical to relieve symptoms, this compound has the potential to actually protect brain cells and slow down how Parkinson’s disease progresses,” FIU professor Kim Tieu, PhD, said in a university news story.

Parkinson’s is caused by the gradual loss of dopaminergic neurons (nerve cells that produce dopamine, a chemical messenger essential for movement). Researchers believe that the buildup of toxic clumps of alpha-synuclein protein and damage to mitochondria, small structures within the cell that produce energy, contribute to the death of these neurons.

DRP1 helps regulate mitochondrial division and function. When it becomes overactive, it can impair mitochondrial function, trigger inflammation, and cause cell damage.

“DRP1 plays an important role in mitochondrial division,” Tieu said. “But when it gets too active, it causes the mitochondria to split more than they should, which can lead to mitochondrial dysfunction, inflammation and cell death – key factors in how Parkinson’s disease progresses.”

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Tieu’s lab developed a large-scale assay to screen for compounds that could regulate DRP1 activity. By screening a library of 35 million compounds, the team identified CTS2444-32, which reduced DRP1 activity by up to 45% and helped limit damage in preclinical models of brain and lung diseases.

In Parkinson’s models, the treatment lowered brain inflammation and the toxic accumulation of alpha-synuclein clumps. The treatment was also shown to protect mice from ventilator-induced lung injury, in which lungs fill with fluid, making it difficult to breathe.

“We saw reduced levels of inflammatory molecules in lung fluid, which tells us this compound does a great job stabilizing mitochondria and protecting cells from harm,” said Stephen Black, PhD, director of the FIU Center for Translational Science.

The team received a patent for the compound and is now refining it for safe use in humans. Researchers are also testing the compound in larger animals to meet U.S. Food and Drug Administration requirements to initiate human clinical trials, which could begin within a year.

Adel Nefzi, PhD, research professor at FIU Medicine, said CTS2444-32 “targets multiple disease pathways with a single drug, offering hope for conditions beyond Parkinson’s and lung injury, such as Alzheimer’s, heart disease and even certain cancers.”