TSRI scientists receive funding to advance stem cell-based Parkinson’s therapy

Researchers hope that this ‘patient-specific’ form of therapy will herald ‘a new era of long-term treatment for Parkinson’s disease’

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LA JOLLA, Calif.—Scientists at The Scripps Research Institute (TSRI) and Scripps Clinic have received a grant of nearly $2.4 million from the California Institute for Regenerative Medicine (CIRM) to support safety and quality tests of a potential stem cell therapy for Parkinson’s disease.
The new two-year project will be led by Jeanne Loring, professor of developmental neurobiology at TSRI. Loring will be partnering with Dr. Melissa Houser, neurologist and medical director of the Parkinson’s Disease and Movement Disorders Center at Scripps Clinic.
“The goal is to restore the quality of life for Parkinson’s patients,” said Loring. “The methods we’re using will raise the bar for quality considerably—for all kinds of cell therapy.”
 “What sets our study apart from many others is that it’s patient-specific,” said Houser. “Our hope is that this grant will help to begin a new era of long-term treatment for Parkinson’s disease.”
Parkinson’s disease strikes when specialized neurons in the brain begin dying. These neurons produce dopamine, a chemical messenger that maintains normal nerve-firing patterns. Without dopamine, patients suffer from tremors, a lack of balance and even speech difficulties.
For the study, the Loring lab will investigate induced pluripotent stem cells (iPSCs), which are derived from adult subjects and can differentiate into any kind of cell in the body. In this case, iPSCs derived from cells donated by 10 Scripps Clinic Parkinson’s patients were developed into dopamine-producing neurons—the same kind that die during Parkinson’s.
The new grant will allow the researchers to advance these cells through U.S. Food and Drug Administration preclinical testing requirements, with the hope of moving closer to clinical trials.
Loring’s work focuses in part on improving the quality and safety of stem cell therapies. She and her colleagues recently published the first comprehensive analysis of genomic sequence of iPSCs, and her lab’s advances include the development of the PluriTest quality control assay for pluripotency (the ability of stem cells to differentiate), which was recently licensed to The Coriell Institute for Medical Research.

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