Cynata Therapeutics Limited has received approval from UK regulators to start a first-of-its-kind allogeneic IPSC-based trial of MSCs for graft versus host disease (GVHD). Cynata also had some big news a couple weeks back with a deal with Fujifilm.

The company is aiming to recruit 16 patients to test whether the MSCs (a type of adult stem cell) made from pluripotent stem cells created in the lab is safe and eventually whether it can aid patients facing GVHD, a potentially life threatening consequence of bone marrow/hematopoietic stem cell transplantation. There are 4 key bullet points the company released on the study:

  • “UK regulatory authority MHRA approves Phase 1 trial with Cymerus(TM) MSCs
  • World first clinical trial with allogeneic iPSC-derived product
  • Major milestone for stem cell therapeutics and regenerative medicine
  • Cements Cynata’s global leadership in second generation MSC therapeutics”

Another allogeneic IPSC study, this one in Japan and led by Masayo Takahashi, appears to be on the cusp of beginning using IPSC-derived retinal pigmented epithelial cells (RPEs) to treat macular degeneration. An earlier related autologous clinical study began with one patient receiving autologous IPSC-derived RPEs, but was halted due to regulatory changes in Japan. Also, IPSC-derived RPEs from a different patient were found to have a few mutations, which I’m still unclear as to whether had any significance.

Takahashi’s team just published a couple important papers on the allogeneic therapy reporting encouraging pre-clinical data in non-human primates where there wasn’t rejection. My sense is that their human clinical study is likely to start early in 2017.

I expect between these trials and other coming new ones we could see a half-dozen or so IPSC-based trials in the works by the end of 2017. Exciting times in the pluripotent stem cell-based clinical translation arena.

One question here with Cynata’s approach that first comes to mind relates to the question or autologous versus allogeneic therapies.

Why pursue IPSC-derived MSCs, particularly in an allogeneic manner, if one could in theory simply use autologous unmodified MSCs? The answer to this may be the greater practicality of an off-the-shelf universal allogeneic product, but that could also be made without using IPSCs. I do wonder if there are data as to whether IPSC-derived MSCs somehow might be demonstrably better in terms of efficacy or safety, which had been suggested in the past by some work. For more information on the use of innovative stem cell products like MSCs for GVHD see item #1 in this post by Alexey (HT to him for his expertise in this area) on Mesoblast’s TemCell product, its approvals, and data so far.

Let’s see how this Cynata trial goes.

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