Nearly one million people in the U.S. are living with Parkinson's disease, which causes progressive symptoms including tremors, stiffness, and gait and balance issues. Although there are medications to treat symptoms, the disease has no cure.

To date, the California Institute for Regenerative Medicine (CIRM) has invested more than $109 million in helping research for Parkinson's disease progress from a basic or Discovery level through clinical trials. The most recent investment is a $2.5 million award to Jennifer Johnston, PhD, CEO of NysnoBio GT Neurology. The funding will support the development of a gene therapy approach to deliver a healthy version of the Parkin gene to patients whose disease is caused by a mutation in that gene.

In many cases the cause of Parkinson's disease is not known, but almost half of those who have family members with the disease and who develop symptoms before 45 years-old carry a mutation in the Parkin gene.

When it is functioning properly, the Parkin protein helps cells recycle damaged proteins and plays a key role in maintaining the cell's mitochondria, particularly within a group of neurons involved in Parkinson's disease. In people with a Parkin gene mutation, damaged mitochondria accumulate and impair a cell's function. If successful, the gene therapy approach would allow these cells to produce a healthy Parkin protein and reverse effects of the disease.

"This gene therapy approach represents a promising strategy to restore Parkin function and mitochondrial health in this early-onset form of Parkinson's disease," said CIRM Senior Science Officer Lisa McGinley, PhD. "By targeting a genetically validated mechanism, this project has the potential to offer a disease-modifying therapy that goes beyond current standards of care and may also have broader application to other neurodegenerative disorders. CIRM is excited to support the team as they advance toward clinical evaluation."

"NysnoBio is honored to be awarded this prestigious grant from CIRM, whose funding is critical to advance pioneering therapies to cure human disease," said Dr. Johnston. "CIRM is a uniquely steadfast supporter of cell and gene therapies, removing roadblocks and creating efficiencies to ensure integration of new therapeutic modalities into the healthcare arsenal against human disease and suffering."

The funded project is not yet being tested in people. It is considered a translational grant, which means that the funding will help move a promising idea closer to FDA approval for clinical trials.

Written by guest contributor Amy Adams