Local residents gather for Summit4StemCell project fundraiser to help battle Parkinson’s disease
Fundraising deadline is Nov. 4 to continue research for groundbreaking treatment
By Stacey Phillips
A small group of San Diego residents diagnosed with Parkinson’s disease have the opportunity to take part in a cutting-edge treatment using cell replacement therapy that could be the first of its kind worldwide and revolutionize the way other life-threatening diseases are treated.
Doctors, scientists, Parkinson’s patients and their friends and families gathered at a private residence in Solana Beach on July 9 for a fundraising event to learn more about the Summit4StemCell project and help raise the $2.5 million needed by Nov. 4 to continue the research.
Nearly $1 million was raised at the event, which was hosted by Jeffrey Strauss, the executive chef and owner of Pamplemousse Grille in Solana Beach, and Ray and Jenifer Raub. Ray said his wife Jenifer was recently diagnosed with Parkinson’s disease and the Summit4StemCell project is giving them hope.
“We believe in the project, we believe in the science. We think it works,” said Ray Raub. “We truly have hope and think this is going to work.”
The Summit4StemCell research is based on taking adult skin cells and transforming them into useful cells that could replace lost or diseased ones. This concept was discovered by Japanese scientist Shinya Yamanaka in 2007 and received the Noble Prize in Medicine in 2012.
The San Diego research team includes Dr. Jeanne Loring, director of the Center for Regenerative Medicine at the Scripps Research Institute in La Jolla; Dr. Andres Bratt-Leal, the senior scientist for the project in association with the Parkinson’s Association; and Dr. Melissa Houser, director of the Movement Disorder Center at La Jolla’s Scripps Clinic.
“Every single patient I’ve seen over nine years get worse,” said Sherrie Gould, a nurse practitioner at the Movement Disorder Center who works with Dr. Houser. “We have an opportunity to change that. What we are doing is going to change history.”
Parkinson’s disease is a progressive disorder of the nervous system. It is mainly due to the loss of a single cell type, dopamine-producing neurons. Dopamine is the chemical that regulates movement and concentration. Current treatments for Parkinson’s include medication and brain surgery, both of which have limitations.
Over the last couple of years scientists have developed methods to use a patient’s own skin cells to produce “pluripotent” stem cells, which are stem cells that can form any cell type in the body. These are matured into dopamine neurons, the same cells lost in Parkinson’s disease. The next step is to inject them back into Parkinson’s patients to treat their symptoms, which requires FDA approval and further clinical studies.
“We could be the first ones in the entire world to use these new types of stem cells so we can actually treat patients with their own cells, their own induced pluripotent stem cells,” said Dr. Andres Bratt-Leal, Ph.D., the senior scientist working on the project with a team of four others in Dr. Loring’s lab. Since the stem cells come from the patient’s own skin cells, there is less chance of rejection by the patient.
Rancho Santa Fe resident Chris Whitmer, who was diagnosed with Parkinson’s disease in 2007, is one of eight Parkinson’s patients of Dr. Houser who have volunteered for the clinical pilot trial at Scripps Clinic. All of the patients are from California and seven are from San Diego. “It’s an honor and a privilege to be one of the eight chosen for the clinical trials,” said Whitmer. “It’s scary and exciting at the same time. This is the future for treatment of Parkinson’s and many other diseases.”
He said that over the last seven years his symptoms, which include shaking of his right hand, arm, leg and foot, stiff muscles, impaired balance, as well as problems sleeping, have become worse. He said that before he learned about the Summit4StemCell project his outlook was pretty bleak. “It will give me and my family my life back free of this crippling disease.”
Scientists have already been able to successfully create the dopamine-producing neurons from eight of Dr. Houser’s patients with Parkinson’s disease and tested the neurons in the lab and in animals. Dr. Bratt-Leal said they have cured Parkinson’s disease in rats in their first preliminary study. “The exciting part is that we’ve done that with our own patients’ cells and now we can move forward and finish the studies that we need to do for the FDA,” he said.
The Summit4StemCell team is looking to raise $2.5 million in order to apply for a matching grant from the California Institute of Regenerative Medicine in La Jolla. Dr. Bratt-Leal said the $5 million would be used to finish the studies to support an application for FDA approval. The institute would also provide funds for overhead costs. All donations are tax deductible and 100 percent of the money will go toward research for this project.
“The sooner we can through the FDA and get our eight patients going, the sooner that the procedure is going to be available to everybody,” said Raub.
The cell replacement therapy will also pave the way to help other untreatable disorders such as diabetes, macular degeneration, heart disease, cancer, Alzheimer’s and ALS.
Dr. Bratt-Leal said that the Summit4StemCell is a unique project because people have donated their own time, skills and money to support this research. “I think it has really become a community project that San Diego can be proud of and excited about,” he said. “This is a San Diego research project that really has a chance to change how we do medicine.”
More information about the Summit4StemCell project, upcoming fundraising events and how to make a donation can be found Summit4StemCell website: https://www.summit4stemcell.org/.