IPS (induced pluripotent stem): a second human clinical trial on the cards between now and 2016

Publié le : 17 March 2014

 On 6 March 2014, a team of Japanese scientists at CiRA (Center for IPS cells Research and Application) – University of Kyoto – published an article in Stem Cell Reports outlining "its plan to start clinical trials by 2016" focusing on treatment for Parkinson’s disease using IPS cells.

In their article, the scientists discussed a "new cell separation technique that considerably improves the efficacy of cell transplants". This project would be the world’s second human clinical trial involving IPS cells. The first trial, which is currently underway in Japan, is focusing on the treatment of ARMD (age-related macular degeneration) (Gènéthique press review on July 22nd, 2013).

One of the main characteristics of Parkinson’s disease is the "deregulation of the dopaminergic system", dopamine being an "essential neurotransmitter for motor and mental functions". The treatment devised by Dr. Jun Takahashi and his team seeks to "replace dopaminergic cells destroyed by the disease with new cells to eradicate or simply slow down disease advance". To this end, they reprogram the patient’s somatic cells into IPS cells, which they then differentiate "into neural cells in order to inject them into the patient’s brain".

To ensure the safety of the transplant carried out and to "prevent the risks of developing a tumour", the scientists have "developed a separation technique to isolate or at least increase the proportion of the progenitor cells". This method uses laminine, a protein artificially created by Professor Kiyotoshi Sekiguchi, at the Protein Research Institute, University of Osaka. Using this protein, the number of redifferentiated cells increased 20-fold and scientists were able to "purify the cocktail of various neuronal cells in order to obtain up to 80% of dopaminergic progenitor cells" using specific enzyme markers.

Based on research previously carried out in rats with Parkinson’s disease, "the transplants generated using this method have a better survival rate and are less likely to trigger cancer". Furthermore, "the quantity of functional dopaminergic cells obtained following a separation phase is greater than that obtained during this stage".

Clinical research preparations will begin in 2015. After an initial month of screening patients suffering from Parkinson’s disease and with no predisposed hereditary factors, scientists will reprogram "somatic cells collected from patients into IPS cells" over a period of 6 months, and prepare them for transplant. Finally, it will take 3 months to "ensure the safety of the cells before transplanting them into patients", with the first transplants scheduled for 2016. Patients will then undergo medical follow-up for one year to check that no tumour develops. Scientists will focus primarily on patient autonomy and lifestyle/living conditions.

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