Unique Clinical Trial Will Use Reprogrammed Adult Stem Cells to Treat Parkinson's Disease | Science

Japanese researchers announced today the launch of a clinical trial to treat Parkinson's disease with a material neurological derived from induced pluripotent stem cells (iPS), chemically manipulated mature cells to return to an early stage of the disease. development from which they can theoretically differentiate themselves in one of the specialized cells of the body

The study team will inject dopaminergic progenitors, a cell type that develops into neurons producing dopamine directly in a region of the brain known to play a key role. in the neural degeneration associated with Parkinson's disease. This effort is led by Jun Takahashi, a neurosurgeon at the Kyoto University's iPS Cell Research and Application Center (CiRA), in cooperation with the Kyoto University Hospital.

Parkinson's disease results from the death of specialized cells in the brain. Dopamine neurotransmitter A lack of dopamine causes a decrease in motor skills, which leads to difficulties in walking and involuntary tremors. As the disease progresses, it can lead to dementia. The test strategy is to derive dopaminergic progenitors from iPS cells and inject them into the putamen, a round structure at the base of the forebrain. Surgeons drill two small holes in the skull of a patient and use a specialized device to inject approximately 5 million cells.

Studies in animals have shown that progenitors differentiate into dopaminergic neurons in the body and graft into the brain. The Takahashi group reported last year that monkey models of Parkinson's disease showed significant improvement for 2 years after injection of neurons prepared from human iPS cells. cells from healthy donors with specific cell types that are less likely to cause immune rejection. "By using cell stocks, we can proceed much more quickly and cost-effectively," said Shinya Yamanaka, director of CiRA, who won the Nobel Prize in Physiology or Medicine in 2012 for discovering how to create iPS cells [19659008] in 2017. As an extra precaution, patients will receive a common immunosuppressant in tandem with the progenitors.

The recruitment of patients began today at 17 hours. local time, when the Kyoto University Hospital issued the notice of recruitment of patients on its website. The team plans to recruit seven patients and follow them for two years after the injection.

This is the third human trial using iPS cells approved in Japan. The first, using retinal cells derived from iPS cells to replace eye tissue damaged by age-related macular degeneration (AMD), was launched in 2014 and led by Masayo Takahashi-Jun's wife. Takahashi from the RIKEN Center for Developmental Biology in Kobe. . The treatment of AMD was initially reported as safe, although there was one reported adverse event. Earlier this year, a team from the University of Osaka, Japan, obtained conditional approval for an ischemic stem cell study for ischemic heart disease.