First-of-its-kind clinical trial will use reprogrammed adult stem cells to treat Parkinson's

By Dennis NormileJul. 30, 2018 , 3:35 PM

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

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

Parkinson’s disease results from the death of specialized cells in the brain that produce the neurotransmitter dopamine. A lack of dopamine leads to a decline in motor skills, resulting in difficulty walking and involuntary trembling. As the disease progresses it can lead to dementia. The trial strategy is to derive dopaminergic progenitors from iPS cells and inject them into the putamen, a round structure located at the base of the forebrain. Surgeons will drill two small holes through a patient’s skull and use a specialized device to inject roughly 5 million cells.

Studies in animals have shown that the progenitors differentiate into dopaminergic neurons inside the body and engraft into the brain. Takahashi’s group reported last year that monkey models of Parkinson’s disease showed significant improvement lasting 2 years after getting injections of neurons prepared from human iPS cells.

More:
http://www.sciencemag.org/news/2018/07/first-its-kind-clinical-trial-will-use-reprogrammed-adult-stem-cells-treat-parkinson-s