April is Parkinson’s Awareness Month and the California Institute for Regenerative Medicine (CIRM) is joining the campaign to #Take6ForPD—take 6 minutes to raise Parkinson’s awareness and highlight our commitment to funding research for the disease.
What is Parkinson’s Disease?
Parkinson’s disease (PD) is a neurodegenerative disease that affects nearly 1 million people in the United States and more than 10 million people around the world. In California, more than 106,000 people live with PD.
Symptoms of PD include tremors, slow movement, muscle rigidity, balance issues and lack of facial expressions. Parkinson’s disease occurs when the neurons or nerve cells in the portion of the brain that controls movement die off. These neurons send signals by releasing a chemical called dopamine and are referred to as dopaminergic neurons.
Parkinson’s is the second-most common neurodegenerative disease after Alzheimer’s disease. No cure exists for the disease.
CIRM’s Commitment to Funding Parkinson’s Research
CIRM remains committed to funding regenerative medicine research—including stem cell and gene therapy research—for PD treatments to improve quality of life for patients.
To date, CIRM has invested more than $60 million in helping research for PD progress from a basic or Discovery level through clinical trials.
This year, CIRM awarded $4 million to a late-stage preclinical project by Ryne Bio aiming to improve treatment for idiopathic PD. In the proposed stem cell therapy, investigators at Ryne Bio are aiming to deliver dopamine-producing cells to replace the lost neurons to the brain of PD patients to restore or improve motor function.
“Receiving a Parkinson’s diagnosis is a difficult and life-changing event,” says Ryne Bio CEO Nick Manusos. “This Parkinson’s Awareness Month, we honor patients and their loved ones by reflecting on the clinical progress made so far, and looking forward to a brighter, healthier future.”
Other regenerative medicine approaches CIRM has funded to target PD involve understanding the disease and looking for new drugs to treat it.
CIRM grantees have taken skin cells from people with Parkinson’s disease, reprogrammed them back to an embryonic-like state, turning them into the kind of stem cell that can be transformed into any other cell in the body, then coaxing those cells to become dopaminergic neurons that are lost to the disease.
Those cells showed signs of the disease in the lab dish and were distinctly different from the same cells created from people without PD.
Another approach involves using CRISPR gene editing technology to reduce the levels of a toxic protein called alpha synuclein, which builds up in the dopaminergic brain cells affected by PD.
Overall, CIRM has awarded more than 30 grants aiming to better understand the disease and find regenerative medicine-based treatments.
“CIRM’s commitment to funding research for Parkinson’s is unwavering,” says CIRM President and CEO Maria Millan. “Our goal is to fund the most promising regenerative medicine research to support finding the best treatments to fight this devastating disease that affects millions.”
CIRM’s Task Force on Neuroscience & Medicine
As part of its Strategic Plan, CIRM has also launched a Task Force on Neuroscience and Medicine to set a plan for the $1.5 billion allocated for the support of research and the development of treatments for diseases and conditions of the brain and central nervous system. These include Parkinson’s, Alzheimer’s, stroke, dementia, epilepsy and other diseases and conditions.
The Task Force will host public meetings to gather community input to identify potentially high-impact opportunities in basic neuroscience, neurodegenerative disease, neuropsychiatric disease, neurodevelopment, and normal brain aging.
The goal of the task force is to provide final recommendations to CIRM and its governing board within six months of inception.
To see a listing of upcoming and past Task Force meetings, visit our website.