Top 20 Stem Cell Predictions for 2017

stem cell crystal ball

Stem cell crystal ball

Each year I make a list of predictions for the stem cell and regenerative medicine field for the coming new year. Later in this post I list my top 20 stem cell predictions for 2017. In looking at my past predictions I realized this will now be my 7th year doing stem cell/regenerative medicine yearly predictions.

You can see below links to these predictions for past years, which sometimes seems rather far removed from today and in other cases strike me as strangely apropos of our times.

What will 2017 bring? Below are my top 20 predictions in no particular order except starting with a few hopeful visions for the coming year.

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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.

homunculus

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 ClinicalTrials.gov.

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!