A brief presentation on recent activities at Cynata

Please listen to an interview with Dr Adrian Bloor, UK Chief Investigator in Cynata’s Phase 1 clinical trial in acute steroid resistant graft-versus-host disease (GvHD).

Please listen to a webcast recording with Cynata Managing Director and CEO Dr Ross Macdonald and FUJFILM Corporate Vice President and General Manager, Regenerative Medicine Business Division, Mr Toshi Ban.

Dr Paul Wotton talks about what the importance of Cynata’s Cymerus technology, what drew him to the company and what he’s seen change since the early days of stem cell therapy. 

Having spent many years working in the stem cell therapy industry, how do you view where it currently stands?

My belief is that cell therapies, or cell-derived therapies, are coming of age. They’ve been around a long time and there’s been a lot of disappointments, but that’s because the technology hasn’t been well-defined or well-developed.

More recently that has changed. We’ve seen some companies like Fujifilm getting into the space, working on technology like Cynata’s Cymerus, and with these sorts of resources it’s my personal belief that it’s just a matter of time until someone shows an outstanding success and the sheep start to follow the leader.

We can see what’s happening with Cynata’s graft-versus-host disease trial for example - it’s a new form of treatment. The world has been waiting for companies like Cynata. 

What attracted you to Cynata Therapeutics?

I think the work they are doing is revolutionary. And the reason it is important is that cell therapy has had a chequered history, part of the reason for that being that it has been an unregulated industry to some extent.

Cynata is one of the companies that’s going about it in the right way. If they get the GvHD working properly when the clinical trial starts, the first patient in the world to be dosed with an induced pluripotent stem cell (iPSC) will come from a Cynata trial. It’s a world-first.

Cynata is taking a population of cells that are quality-controlled, reproducible. They’ve got a consistent product and that’s really important when you’re trying to treat a population of people as a whole.

This being an ‘off the shelf’ product means quality control issues are much easier to monitor, and means it’s more cost-effective for Cynata to provide therapy to patients and it’s ultimately a win-win all around.

These clinical trials that have been approved by the pertinent bodies are going to make a meaningful difference to people’s lives – it’s a sign the company has grown up and is moving in the right direction. 

What differentiates Cynata from other companies involved in the therapeutic stem cell industry?

I think a lot of people had a lot of technology that was very promising but cell therapy was unregulated in the sense that those people were able to make outrageous claims – and often tried to test them – without an approved product from a regulatory body. These bodies are there for a purpose, to make sure that patients receive safe treatment that is shown to work under controlled clinical trials.

The difference for Cynata is the fact that they’ve got a product in the clinic. That means they’ve worked with the regulatory bodies who are satisfied that there will be benefit of using that technology in humans, and that that outweighs any risks.

And I have also been very, very impressed with Dr Ross Macdonald as a CEO. He is a very responsible man who knows exactly what he is doing, he’s very professional and would never make claims or work on something that he could not substantiate.

To be honest the most important thing is that patients are going to get the benefit of the therapy we are developing. That’s the fact. 

What is your vision for Cynata’s work?

This goes back a long way for me. I was at the Westminster Hospital in the early 80s when they were doing bone marrow transplants to kids with orphan diseases. I was on the periphery of this type of technology back in its early days.

Now I come forward to this period in time, and I can see that a lot of these kids benefitted from that early research. That’s what can happen with Cynata.

I am now in a situation where I am looking at cell therapy having come full circle. It’s no longer just being practised by some visionary professor at a top teacher hospital in London, it’s becoming reality and it’s on the verge of curing a number of serious diseases.

Our technology is now at the point where we can understand what cells can do and what they can’t do. How they communicate and interact. We can harness that now. It has taken 30 years for the science to catch up with the vision and I’m very excited. 

What is the significance of Cynata gaining these regulatory approvals for its graft-versus-host disease clinical trial?

You cannot underestimate the power of the technology moving out of the lab and into the clinic. This is a huge step for a company. It means Cynata’s product has been validated by independent experts who are interested in seeing this used in humans to get a cure.

They have to be very careful about what they do in approving these go-aheads. Believe me, many, many biotechs don’t get this far. I think Dr Macdonald and his team have done – and will continue to do – a magnificent job.

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