Cynata in the Japanese Media (#2)” Yomiuri Shimbun March 31, 2017: Clinical trial with donated iPS cells

Clinical Trial with donated iPS cells

Immune suppressor cells: Potential for practical application from 2019

Interview with CEO of Australian venture firm

A medical treatment project using donated induced pluripotent stem cells (iPS cells) has been promoted by a Japanese team led by Riken, and an Australian regenerative medicine bioventure firm in which Fujifilm has invested. The Australian bioventure firm – Cynata Therapeutics – plans to conduct clinical trials in the U.K. and elsewhere on graft-versus- host disease (GVHD) patients, which is manifested in an excessive response by immune cells. Their Chief Executive Officer (CEO), Dr Ross Macdonald, was exclusively interviewed by the Yomiuri Shimbun in Japan at the beginning of this month, and revealed, “We hope to make this practically available, possibly as soon as 2019.” (Yusuke Toyama)

Interviewer (I): How do you plan to proceed with the clinical trial?

Macdonald (M): GVHD is a condition in which the patient’s body is attacked due to an excessive response by immune cells contained in bone marrow that has been transplanted into patients suffering from leukaemia and suchlike. The most severe cases often prove fatal. The intention is to administer donated iPS cell-derived mesenchymal stem cells , which appear to restrain excessive immune function, to GVHD patients. A total of 16 patients will participate in the trial in the U.K. and Australia.

I: What is the advantage of using donated iPS cells?

M: GVHD treatment using mesenchymal stem cells derived from donated bone marrow has a proven track record, and in Japan, JCR Pharma (based in Ashiya City, Hyogo prefecture) also brought it to market in 2015. However, it is difficult to cultivate mesenchymal stem cells, so many donors need to be found. There is no limit to cultivating iPS cells though.

I: What is the basis for your tie-up with the Fujifilm Group?

M: We have been provided with iPS cells from Fujifilm’s subsidiary in America. They were the first company in the world to cultivate iPS cells of a quality that can be used for clinical applications, so the benefits are substantial.

I: There are reportedly over a thousand GVHD patients a year in Japan that develop severe conditions, of whom several hundred are considered to be the most severe cases and could be considered as subjects for this clinical trial.

M: If beneficial results can be obtained through the clinical trials conducted in the U.K., we are considering seeking authorization (for this treatment) by conducting further clinical trials in Japan in the future.

I: What are the future prospects?

M: Phase 1 clinical trials are currently underway to verify safety. If beneficial results can be obtained within 100 days of administration, we would like to proceed to the next step of confirming the treatment’s efficacy. We are working hard to ensure that we can make this treatment available as quickly as possible for patients.
Illustration of GVHD therapy based on donated iPS cells


Mesenchymal stem cells: Inherent in bone marrow and body fat, these cells can be transformed into a variety of tissues, such as bone, muscle, and fat. Numerous projects are being conducted to find ways to be able to use them for regenerative medicine.



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