TSRI Scientists Receive $2.4 Million Funding to Advance Parkinson's Therapy

Parkinson's disease strikes when specialized neurons in the brain begin dying. Without dopamine, patients suffer from tremors, and even speech difficulties.

To support safety and quality tests of a potential stem cell therapy for Parkinson's disease, 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).

The new two-year project will be led by Jeanne Loring, professor of developmental neurobiology at TSRI. Loring will be partnering with Melissa Houser, M.D., 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."

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‘Parkinson's disease strikes when specialized neurons in the brain begin dying. Without dopamine, patients suffer from tremors, a lack of balance and even speech difficulties.’

"What sets our study apart from many others is that it's patient-specific," said Dr. 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. The grant number is DISC2-09073.

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About The Scripps Research Institute

The Scripps Research Institute (TSRI) is one of the world's largest independent, not-for-profit organizations focusing on research in the biomedical sciences. TSRI is internationally recognized for its contributions to science and health, including its role in laying the foundation for new treatments for cancer, rheumatoid arthritis, hemophilia, and other diseases. An institution that evolved from the Scripps Metabolic Clinic founded by philanthropist Ellen Browning Scripps in 1924, the institute now employs more than 2,500 people on its campuses in La Jolla, CA, and Jupiter, FL, where its renowned scientists-including two Nobel laureates and 20 members of the National Academy of Science, Engineering or Medicine-work toward their next discoveries. The institute's graduate program, which awards PhD degrees in biology and chemistry, ranks among the top ten of its kind in the nation.

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About Scripps Health

Founded in 1924 by philanthropist Ellen Browning Scripps, Scripps Health is a nonprofit integrated health system based in San Diego, Calif. Scripps Health treats more than 600,000 patients annually through the dedication of 3,000 affiliated physicians and more than 15,000 employees among its five acute-care hospital campuses, hospice and home health care services, 28 outpatient centers and clinics, and hundreds of physician offices throughout the region.

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Recognized as a leader in prevention, diagnosis and treatment, Scripps Health is also at the forefront of clinical research, genomic medicine and wireless health care. With three highly respected graduate medical education programs, Scripps Health is a longstanding member of the Association of American Medical Colleges. Scripps Health has been ranked four times as one of the nation's best health care systems by Truven Health Analytics. Its hospitals are consistently ranked by U.S. News & World Report among the nation's best and Scripps Health is regularly recognized by Fortune, Working Mother magazine and AARP as one of the best places in the nation to work.

Source-Newswise