Researchers are testing FDA-approved compounds that target dynamin-related protein 1 (Drp1), which regulates mitochondrial division, as potential treatments for Parkinson’s disease

Researchers focused on Parkinson’s disease have spent over a decade studying the role of mitochondria—the cell’s powerhouses—in this condition. Their work has uncovered a key protein that could pave the way for new treatments for Parkinson’s and other neurodegenerative diseases. Mitochondria are dynamic, constantly changing in size, number, and location within cells to meet various demands. This flexibility is crucial for both mitochondrial function and overall cellular health. When mitochondrial dynamics are disrupted, it can set off a chain reaction of cellular dysfunction, ultimately leading to cell death. Recent studies have linked disruptions in mitochondrial processes to neurodegenerative diseases, including Parkinson’s. Toxic proteins and environmental neurotoxins can interfere with the balance of mitochondrial fusion and division, leading to impaired cellular function and the buildup of harmful protein aggregates—a hallmark of Parkinson’s disease. The team hypothesized that restoring mitochondrial function by targeting its dynamics could protect against neuronal damage and cell death. They focused on a protein called dynamin-related protein 1 (Drp1), which regulates mitochondrial division. Excessive Drp1 activity leads to fragmented, dysfunctional mitochondria. In their lab models, including neuronal cell cultures and animal models of Parkinson’s, they found that environmental toxins and toxic proteins cause mitochondrial fragmentation and dysfunction, leading to neuron death. However, by reducing Drp1 activity, they restored normal mitochondrial function and protected neurons, which continued to function properly. The 2024 study also revealed that inhibiting Drp1 can protect cells from neurodegeneration by restoring the cell’s waste recycling system, even in the presence of harmful substances like manganese. These findings suggest that targeting Drp1 through multiple pathways could be a promising approach to treating Parkinson’s. They have identified several FDA-approved compounds that target Drp1 and are now testing them as potential Parkinson’s treatments.