Cynata approval for 1st ever allo trial of IPSC-derived MSCs for GVHD

cynataCynata 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.

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Stem cell news bites: Mesoblast, BioTime, MS, FDA Warning, & More

Mesoblast stockStem cell studies continue to make big news with a mixed bag of trends, and some good stem cell news and others not so much.

Mesoblast ($MESO) had its stock suspended for quite so time recently in anticipation of a big announcement. When that news came today it was discouraging in the form of its partner Teva choosing to end their relationship with Mesoblast on a stem cell heart disease trial. Mesoblast spun this as something good that they regained the rights from Teva, but investors saw it very differently and the stock has plunged today. I’m not sure I get the point of the two-week hold on the stock leading up to this.

On a more upbeat front, BioTime‘s subsidiary Cell Cure Neurosciences is making progress on their trial work for a treatment for Dry AMD. The company received safety board approval to start a second cohort for the OpRegen product that will include a more clinically meaningful dose.

The use of stem cells for multiple sclerosis (MS) continues to make headlines. It is important to note that the recent upbeat media stories on a stem cell treatment for MS, while based on encouraging data, are focused on an approach that is extremely high risk and thus is appropriate only for the most severe MS cases. The treatment itself can be fatal. Other stem cell offerings for MS, such as those sold by stem cell clinics, have little if any data behind them. Interestingly, Health News Review did an analysis of that “this isn’t hype” Vox story on the stem cells for MS development and they gave the story a very strong rating (9/10). They only dinged it for “disease mongering”.

The FDA sent a warning letter to another stem cell cosmetics company, Hollywood Skincare International. The FDA says the product “DermaSet Stem Cell 3D Renewal Treatment” is an unapproved drug. The FDA continues to be active on stem cell cosmetics, but not so much on stem cell clinics.

Next week is the big ISSCR annual meeting in San Francisco. If you haven’t registered yet, you can do so here. It’s a great opportunity to learn about stem cells and talk about everything to do with stem cells with other stem-o-philes.

Did I miss any recent stem cell news that you think is important? Weigh in.

Mesoblast stock on hold: Poll if you expect good or bad news

Adult stem cell biotech Mesoblast ($MESO) is in the unusual situation currently of having its stock on hold. This is a relatively rare event and usually portends some kind of major news. The hold is expected to run from June 1 to June 10.Mesoblast_Ltd

Mesoblast will announce ““material corporate developments with respect to certain assets of the company”.

This could involve good or bad news related to one of several partnerships or some other development. In late 2015 Mesoblast had upbeat news of a first stem cell drug approval in Japan under that country’s new more permissive regenerative medicine oversight rules.

Do you expect this current stock hold signals good or bad news for Mesoblast? Take our poll.

Disclosure: I have no investment in this or any other stem cell stock currently.

First impressions of the US stem cell environment from an Aussie

Heather_MainBy Heather Main

I moved to the San Diego from Australia in August 2015, and Paul asked me if I could write something on my first impressions of doing science in the US, as opposed to other countries I have worked/studied in (Australia, Scotland, Sweden and Singapore).

If you look at the land size of Australia and consider that we have only 7% of the US population you will understand directly that funding and collaborative opportunities are not as easy to find. There’s probably little collaborative advantage in the US compared to Europe, which has over double the number of people as the US and only 3% larger land mass. In defense of Australia we punch above our weight, with the first stem cell company (Stem Cell Sciences) and currently the world’s largest (Mesoblast) originating there…even Viacyte has Australian origins.

It seems like it is hard not to be political here in the US….I guess I have arrived at a special time, but after working in Sweden for 5 years and living through gun control regulation following the 1996 Port Arthur massacre in Australia, I don’t understand the opposition to publicly funded healthcare, how a regarded education is ‘purchased’ and the 17th century resistance to gun control regulation. …I guess you need to be born into it. I think the political minorities have almost as much backing in Australia but are not as extreme and have less volume, so the international effects are minimised.

Relevant to stem cell research, the Colorado Springs Planned Parenthood shooting in November 2015, made me wonder how extreme the fight to protect the rights of a fertilised egg still is. My only explanation for these people is a 16th century belief in a ‘homunculus’ (fully formed miniature human present in every sperm)…Someone please get them a microscope.


Above. The : A 16th century theory for human development. Obviously not real though but an image that seems to exist in the mind of the anti-choice movement

All of this seems contradictory to a country with arguably the most advanced thinking and technologies but I was pleased to see the numbers don’t support it with;

– 2008 Bush allocation of $88 million in NIH funds to human embryonic stem cell research (compared to $381 million to adult stem cell research)

– 2014 Obama allocation of $166 million, while non-embryonic only slightly increased ($443 million), with a large allocation to iPSC projects.

This even indicates the recognised importance of pluripotent technologies over adult somatic technologies.

I wonder though if this balance is also recognised in the public and adult somatic cell spaces. In an effort to sell an alternative to hESC, the potential of adult somatic stem cells was drastically inflated. Recently I have attended a number of adult somatic stem cell talks who describe the expression of Oct4/nanog etc. in their population of choice, even call them pluripotent….though I am yet to see the teratoma/PluriTest, or clinical trial for functional long term grafting behaviour. Literally hundreds of clinical trials on MSCs have been done over the last 15 years so I guess if the data were there, we would have seen it.

MSC clinical trials

Number of registered clinical trials of mesenchymal stem cells-based therapy on

My fears were allayed when reading Don Reed’s book, in which he interviews fantastic adult stem cell researcher and patient advocate, Dr Jan Nolta. When asked if injection of mesenchymal stem cells was a possible cure for Huntington’s Disease, she answered straight out “No,…..But it buys us time”. Herself and Dr Vickie Wheelock agreed that hESC or hiPSC would be needed for the actual cure. It’s refreshing to see this honesty, a recognition that we need to work together, promote both sides, but realise the timelines of expected contribution from each. Saying it is not a cure does not mean it is not important.

So far I have enjoyed a range of meetings, including both Stem Cells on the Mesa and the World Stem Cell Summit on US soil and look forward to ISSCR in June. Unfortunately from Australia it is wildly expensive and time-consuming to travel to all of these meetings….you can fly LAX to Sydney in 15 hours as opposed to the 12 hours it took my husband to get from San Diego to Florida….

Australia US map

Good or bad I think the US is a place where anyone with a decent backing can make changes with great relevance on the International scale. If you can legalise hESC research under a Republican government, you can do anything!

Stem cell treatment cost 2.0: legit therapy

stem cells costWe hear so much about exciting potential stem cell therapies. Some of these are rigorously evaluated ones in the FDA clinical trial pipeline and others are available right now mainly through predatory stem cell clinics. Earlier this year I posted about the cost of the offerings of dubious stem cell clinics.

In this post, I address the cost of a future, legitimate, FDA-approved stem cell therapy. How high will that be?

This is a critical question because if many patients cannot afford a stem cell therapy then the impact of that therapy is reduced. Cost is inversely related to access. On the other hand, stem cell biotechs must make some profit or they will go out of business. Investors, who are often enthusiastic boosters of the stem cell field, will lose large sums of money and confidence in the field too in that scenario if stem cell treatments are priced “too low”. What is the “right” price?

There is likely to be increasing pressure on biologics drug prices as well from the federal government. Witness Hillary Clinton’s recent tweet on this topic below that sent people into a tizzy.

At the state level, such as here in California, the question of stem cell treatment cost is also becoming more pressing including for our state stem cell agency, CIRM. As CIRM-funded clinical trials advance, which is a wonderful thing, at the same time we get closer to where someone will have to decide on stem cell price tags.

We can look at what other cellular drugs have cost as guidance for the price tag range for stem cell treatments. For instance, Prochymal (its old name under Osiris) that is now rebranded as TEMCELL from Mesoblast/JCR likely will cost about $200K for a full treatment for GVHD (HT to Alexey). Provenge, the cellular prostate cancer drug from the controversial biotech Dendreon had (has?) a price tag of $93K. The most expensive drug in history, the gene therapy med Glybera will cost around $1.5 million per patient.

Realistically, a typical legit stem cell therapy could easily be $100K per patient. A personalized cellular medicine such as an autologous stem cell-based therapy could easily run into the hundreds of thousands per patient. Some therapies could go as high as $500,000 (see this helpful piece by David Jensen) or even into the millions.

Irv Arons

Irv Arons (@iarons) has come up with a great table of cost estimates focused in the area of vision therapy (free registration required). Thanks to Irv for permission to use it here (above).

How will patients afford such expensive therapies?

Will such therapies be covered by governmental agencies or insurance companies? They should.

We should also be keeping in mind the current costs of treating today’s patients with major and sometimes chronic diseases. These costs run into the hundreds of billions or above a trillion dollars each year in the US alone. That’s important context and rightly indicates that the costs of stem cell therapies to society may be appropriate even if at an individual level they seem high.

How does this compare to stem cell treatments at predatory clinics?

Such “treatments” range from $5,000-$20,000 each and most patients with whom I have talked either received or were pitched at least two such treatments, amplifying the total cost. The cost to the clinic of the treatment itself can be as low as $500-$1,000. Some clinics claim to have treated thousands of patients suggesting they are making millions in profits.

Why are stem cell clinic offerings typically relatively cheaper than legit treatments? Frankly, it is because they don’t follow the rules or do the necessary studies to prove safety and efficacy. Ten thousand dollars is still a lot to pay for something that doesn’t work and could even be harmful.

Even so some consumers may perceive dubious stem cell treatments as the way to go because of the lower cost, particularly if the legitimate stem cell field fails to do a good job at educational outreach and the FDA continues to effectively do nothing about the stem cell clinic problem.

The bottom line remains a question. Where’s the stem cell price sweet spot where we can help the most patients, but also generate a needed profit for the biotechs?

We need to find an answer to this question soon.