TSRI and Scripps Clinic Lay Groundwork for Parkinson’s Disease Clinical Trials

The Scripps Research Institute (TSRI) and Scripps Clinic have collaborated to begin the research phase of a proposed clinical trial using induced patient-derived stem cells to halt or reverse the effects of Parkinson’s disease.

“What sets our study apart from others is that it’s patient-specific,” said Melissa Houser, neurologist and medical director of Scripps Clinic’s Parkinson’s Disease and Movement Disorders Center, who is leading the initial phase of the study with Jeanne F. Loring, professor of developmental neurobiology at TSRI. “That means our patients who initially donated their skin cells will eventually receive their own cells again, 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.”

While there are several clinical trials using a stem cell approach to treating injury and neurodegenerative disease, re-implantation of cells made from a patient back into the same patient has never been attempted.

In Parkinson’s disease, nerve cells degenerate, resulting in a loss of the neurotransmitter dopamine—a chemical that helps control muscle movement. Symptoms of Parkinson’s disease include muscle tremor, muscle stiffness, trouble walking, imbalance, and speech problems.

While Parkinson’s disease itself is not technically fatal, the Center for Disease Control rated complications from the disease as the 14th leading cause of death in the United States; it is estimated to affect one in 100 people over age 60. While medications and surgery can be helpful in lessening symptoms, there is currently no cure.

‘A Remarkable Journey’

In the current phase of the project, the TSRI and Scripps Clinic team is taking skin cells from Parkinson’s disease patients who meet select criteria, then cultivating these cells in vitro and turning them into pluripotent stem cells.

“The word pluripotent means that the cells can turn into any other kind of cell,” said Loring. “In this case, we’re converting adult cells taken from skin samples into stem cells. These simple skin cells are now in the middle of a remarkable journey toward becoming nerve cells, and we’re excited to explore their use as a viable treatment for Parkinson’s disease.”

The longer-term plan, which will require US Food and Drug Administration (FDA) approval, is to implant these cells into the patients’ brains; the goal is for the cells to produce enough dopamine to alleviate the worst symptoms of Parkinson’s disease.

This first phase of the research will likely be completed in 2013, followed by the second phase of the project, which will be preparation of the cells for clinical use. In the second phase, the group plans to join other qualified organizations to help design a national, multi-center 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.

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