September 26, 2020

The Niche

Knoepfler lab stem cell blog

Japan IPS cell trial for vision to start in new incarnation

Masayo Takahashi, IPS cell trialIt’s been a long road, but the first ever IPS cell clinical study in humans is starting up again in a new incarnation. You might say it has been regenerated in a novel form.

Masayo Takahashi (高橋 政代) first started the pioneering IPS cell study a few years back in 2014, but it ended up getting put on hold in the summer of 2015 in the midst of changing regs in Japan and the discovery of some mutations in the IPS cell derivatives.

About a year after the hold was put in place, we got news that the study would be restarted in a new incarnation, and now more definitively the study is on track to start up again with 5 patients in Japan with the wet type of macular degeneration.  You can read the RIKEN PR here.

The PR provides more info including a co-leader:

“The project will be led by Yasuo Kurimoto and Masayo Takahashi of Kobe City Medical Center General Hospital, and Osaka University’s Graduate School of Medicine/ Faculty of Medicine, and will be conducted in collaboration with the RIKEN Center for Developmental Biology (CDB) and Kyoto University’s Center for iPS Cell Research and Application (CiRA).”

This is great news.

Yasuo Kurimoto is the surgeon who did the initial surgery in the study before it stopped.

One of the most striking things about the new form of this IPSC study is that it will reportedly focus on allogeneic use of IPSC rather than autologous. The foundation for this switch is that allogeneic cells derived from the IPSC can be used in matched recipients and in a relatively genetically homogenous population such allogeneic cells can be used in a number of patients with a good possibility that there won’t be rejection of the transplant.

Embryonic stem cell-based therapies can be used in the same allogeneic way and those are being studied for macular degeneration as well. I still feel like in the long run that the most power and unique positive contribution from IPSC clinically speaking may come from autologous use, especially in diverse populations such as the U.S. Their use in disease modeling is quite impactful as well.

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