Scripps Clinic and The Scripps Research Institute (TSRI) have begun the research phase of a proposed clinical trial using a unique type of stem cell to halt or reverse the effects of Parkinson's disease.
These stem cells, known as pluripotent cells, can regenerate and have the ability to change into any other type of cell.
Skin cells taken from Parkinson's disease patients who meet select criteria are being cultivated in vitro and turned into pluripotent stem cells. These stem cells will be developed into dopamine-producing brain cells.
The plans, which will require Food and Drug Administration (FDA) approval, are to implant these cells back into the donor patients' brains; the goal is for the cells to integrate inside the brain and produce enough dopamine to alleviate the worst symptoms of Parkinson's disease.
There are currently several clinical trials using pluripotent stem cells to replace cells lost to injury or neurodegenerative disease, as well as for the development of pharmaceuticals, but reimplantation of cells into the same patient for function restoration as a treatment for Parkinson's disease has never been attempted.
Dr. Melissa Houser of Scripps Clinic's Parkinson's Disease and Movement Disorders Center, and Jeanne Loring, co-investigator, professor and director of the Center for Regenerative Medicine at TSRI, are leading the efforts.
"What sets our study apart from many others is that it's patient-specific," said Dr. Houser. "That means our patients who initially donated their skin cells will eventually receive their own cells again, with their own DNA, just in a different form. Our hope is that this will slow or stop the progression of Parkinson's disease while minimizing the potential for rejection."
Preliminary research will likely be completed in this year. That stage will be followed by preparation of the cells for clinical use when the group plans to join other qualified organizations to help design a national, multicenter trial.
The clinical trial is slated to launch two or three years later, depending on the timeframe required for obtaining FDA and other regulatory approvals.