Cellular therapy of parkinsonism
Parkinson's disease will be treated with induced stem cells
Daria Spasskaya, N+1
On Wednesday, the first clinical trials using induced pluripotent stem cells (iPSCs) for the treatment of Parkinson's disease in humans will start in Japan. The announcement of the upcoming tests was posted by the Center for Research and Application of iPSC of Kyoto University (CiRA), also reported by the Kyodo news agency.
The pathogenesis of Parkinson's disease, an age–related neurodegenerative disease that ranks second in prevalence after Alzheimer's disease, is caused by the loss of dopamine neurons in a part of the brain called substantia nigra. Until now, there were no means to resume the growth or activity of neurons, so treatment for this disease existed only symptomatic.
Last year, Japanese researchers from Kyoto University led by Jun Takahashi An article has been published on the successful implantation into the brain of rhesus monkeys with a model of Parkinson's disease of precursors of dopaminergic neurons derived from induced pluripotent human stem cells (iPSCs). As the study showed, the transplanted cells developed into mature neurons that formed a network of interactions with their surroundings. Implantation led to the relief of the symptoms of the disease in animals, and the injected stem cells did not lead to the formation of tumors. In parallel, Professor of the Center for Research and Application of iPSC (CiRA – Center for iPS Cell Research and Application) Kyoto Takahashi University has announced its intention to apply for human clinical trials.
The application was approved by the Ministry of Health of Japan and the start of the tests was scheduled for the first of August 2018. This study will be a Phase I/II trial involving seven patients from Kyoto University Hospital to evaluate the safety and efficacy of therapy. Five million precursors of dopaminergic neurons derived from iPSCs and provided by CiRA will be injected into the brain of patients.
Induced pluripotent stem cells are obtained from "adult" specialized cells, for example, skin cells, by reprogramming back to a non-specialized state. Different types of cells can then be obtained from them. Developments involving iPSC are especially popular in Japan, as the method of reprogramming cells was discovered by Japanese scientist Shinya Yamanaka, for which he was awarded in 2012 Nobel Prize in Physiology or Medicine.
Recently in At Osaka University, researchers began a trial to treat the consequences of a heart attack using cardiomyocytes grown from iPSCs, and earlier a new retinal pigment epithelium was grown from iPSCs to Japanese patients. However, in addition to iPSC, other sources of stem cells can be used for these purposes – we told, for example, that American scientists have grown retinal pigment epithelium from embryonic stem cells in patients.
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