Stem cells for Parkinson’s getting ready for clinic

A groundbreaking attempt to heal eight Parkinson's patients with their own cells could move from research to the clinic next year.

For eight Parkinson's patients seeking treatment with a new form of stem cell therapy, 2014 promises to be a milestone. If all goes well, next year the FDA will give approval to begin clinical trials. And if the patients can raise enough money, the scientists and doctors working with them will have the money to proceed.

Jeanne Loring, a stem cell scientist at The Scripps Research Institute, discusses the status of a project to treat Parkinson's patients with their own cells, turned into the kind of brain cells destroyed in Parkinson's. The project is a collaboration with Scripps Health and the Parkinson's Association of San Diego.

Scientists at The Scripps Research Institute led by Jeanne Loring have taken skin cells from all patients and grown them into artificial embryonic stem cells, called induced pluripotent stem cells. They then converted the cells into dopamine-making neurons, the kind destroyed in Parkinson's disease.

Loring discussed the project's progress on Friday morning at the 2013 World Stem Cell Summit in San Diego.

If animal studies now under way and other requirements are met, doctors at Scripps Health will perform a clinical trial. They will grow neurons until they are just short of maturity, then transplant them into the brains of the respective patients. The cells are expected to complete maturation in the brain, forming appropriate connections with their new neighbors, and begin making dopamine.

Earlier attempts to treat Parkinson's with a stem cell-like therapy mostly failed because of difficulties in quality control of the source, neural cells from aborted fetuses, Loring said. But some patients gained lasting improvement, a tantalizing hint that the trials were on the right track.

In January, a "pre-pre-IND meeting" is planned with the FDA, Loring said.

Also speaking were Ed Fitzpatrick, one of the eight patients, and Kyoto University researcher Jun Takahashi, who is independently trying the same approach in Japan.

Ed Fitzpatrick, one of eight Parkinson's patients in a program to be treated with his own cells, grown into the kind of brain cells destroyed in Parkinson's.

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Stem cells for Parkinson's getting ready for clinic