The Scripps Research Institute is conducting groundbreaking research that holds immense promise for long-term management of Parkinson’s disease, and possibly for MS, ALS, and Alzheimer’s diseases. After learning of Japanese researcher Shinya Yamanaka’s Nobel Prize award-winning findings in 2012 (that four genes could be activated that convert cells taken from a patient’s arm into neurons that could be used to help patients with neurological disorders), Jeanne Loring, PhD, director of the Center for Regenerative Medicine at the Scripps Research Institute, and Melissa Houser, MD, neurologist and director of movement disorders at Scripps Clinic, joined forces, generating interest and support to begin their own Parkinson’s research.
Working in Loring’s lab, Andres Bratt-Leal, PhD is leading the research team and serving as the main contact with the patients and Summit4StemCell, a local nonprofit working with the Parkinson’s Association of S.D. to raise funds and awareness for non-embryonic stem cell research while inspiring people with the disease to move beyond their physical limitations.
According to Bratt-Leal, preclinical animal trials began December 4 by inducing Parkinson’s in rats using a dopamine toxin. “After the toxin does its work to kill off the dopaminergic neurons (as in Parkinson’s disease) we are transplanting doses of neurons from two of our patients’ cells to test for safety and efficacy in restoring the dopamine loss in the rats. It will be about two months before we will be able to see if the cell treatment is working. If it works well in animals, our next step is to work with the FDA to begin planning our patient studies.”
By using the patients’ own cells, Bratt-Leal anticipates that they will avoid immune rejection issues, which can affect how the cells work and what medicines the patients need to take after surgery. “Because what we are doing is personalized medicine aimed at long-term relief and not an off-the-shelf daily pill, we are relying on the Parkinson’s community and philanthropy to make sure this important work has the financial support to move forward as quickly as possible.”
“This is very personal to everyone involved,” says Loring. “It’s a group effort unprecedented in medical research — patients, physicians, and scientists working together every step of the way.”
Funding is entirely grassroots — no companies are involved, and every dollar is measured. It’s the team’s hope to get further needed funding through philanthropy, as well as grant support from the California Institute for Regenerative Medicine.
The treatment will potentially give patients many Parkinson’s disease-free years. “We want to emphasize that we do not call this a cure,” stresses Loring. “This idea of replacing cells that are lost to disease with a patient’s own cells can apply to many other diseases. If we are successful in obtaining permission from the FDA for the clinical trial — and we will, it’s just a matter of time and funding — it will be a landmark for stem cell therapy for many diseases.” (www.summit4stemcell.org) MIA S. PARK