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Getting to know Mesenchymal Stem Cells (MSCs)

img3A “gold rush” for the next wave of regenerative medicine therapies.  But what are MSCs? And what do they do? Let us break it down for you.

What are they?

img1Mesenchymal stem cells (MSCs) are multipotent stem cells that naturally occur in adult tissues such as bone marrow and fat. They are also found in placental tissues.  MSCs are able to produce a wide range of biologically active chemicals that stimulate the body’s own healing systems. Around 650[1] clinical trials around the world are investigating MSCs as a treatment for a diverse range of illnesses, including immune disorders, heart disease, stroke, arthritis, fractures, degenerative disc disease, diabetes, lung disorders and eye disease[2].  To date, all the MSC products tested have been isolated from human tissue, mostly bone marrow.

MSCs can be derived from Induced Pluripotent stem cells (iPSCs) which are a type of pluripotent stem cell that can be generated directly from adult cells.  It all started in 2006, when Japanese researchers identified conditions that allowed specialised adult cells to be genetically ‘reprogrammed’ to an embryonic stem cell-like state[3]. This was a profound discovery as it enabled a solution to the controversy over using pluripotent stem cells from human embryos. There has been enormous interest globally in the development of iPSC-derived therapies for a number of years.

What are the limitations with MSCs?

MSCs are free from ethical concerns and can come from numerous sources, so why aren’t MSC’s more widely used?  Well they are the most commonly used stem cells in current clinical trials and applications, however a major hurdle that scientists face has been the ability to produce a consistent commercial supply of clinical quality MSCs for use in clinical trials and eventually for commercial therapeutic use.  Current methods of producing MSCs reply on traditional means of extraction from the donor’s bone marrow or other tissue, this is costly, not efficient or sustainable for commercial development of MSC-based therapeutics.

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Where does Cynata come in?

Here at Cynata Therapeutics, we have developed and patented a therapeutic stem cell platform technology – Cymerus™. The technology is based on discoveries made at the University of Wisconsin-Madison, a world leader in stem cell research. We are utilising iPSCs as the starting material for generating mesenchymoagioblasts (MCAs) and in turn for manufacturing the MSC therapeutic product. With this technology, we can ensure that MSCs for therapeutic use can be produced consistently, economically and in virtually limitless quantities, which means we won’t need to constantly seek out fresh stem cell donors to fuel manufacturing demands.   Because we can reproduce the iPSCs virtually limitlessly we also do not need to massively expand the MSCs themselves which should result in a more effective MSC product.

What is Cynata working on?

We have proved our that our Cymerus™ technology is effective in various animal models for specific disease targets. In a world-first, we have also been given the green light to commence a clinical trial of CYP-001, our patented Cymerus product for graft-versus-host disease (GvHD).  If we demonstrate that our technology is effective in these clinical trials, this will fast track our ability to commercialise our platform technology for further diseases that represent major unmet medical needs.

Here’s a snapshot of what we are working on:

Heart attacks

  • When a blood vessel supplying blood to the heart becomes blocked, it damages the heart muscle and disrupts the functioning of the heart, causing what is recognized as a heart attack. In Australia, heart attacks claimed over 8,000 lives in 2014, equating to 24 deaths per day.
  • Our preliminary results from our pre-clinical trials suggests that Cymerus™ iPSC-generated MSCs may have the potential to restore cardiac function and reduce scar size after a heart attack[4]. Read more here.

Graft-vs-host disease (GvHD)

  • A complication that can occur after a bone marrow transplant. The newly transplanted cells from the donor (the “graft”) attack the transplant recipient’s cells (the “host”).
  • We have successfully completed pre-clinical research to show that Cymerus™ MSCs can be an effective treatment for GvHD. Our results showed that CYP-001 treatment substantially prolonged survival in an animal model.
  • The upcoming clinical trial in GvHD is believed to be the first clinical trial worldwide involving an allogeneic therapeutic product derived from induced pluripotent stem cells (iPSCs)4. Read more here.

 Asthma

  • Asthma is a chronic, long term lung condition recognised by the World Health Organisation (WHO) as a disease of major public health importance due to its global prevalence.
  • The medication used to manage chronic asthma can have significant side effects and impact the patient’s quality of life. Due to the prevalence of asthma, the global market for these medicines is expected to reach US$25.6 billion by 2024.
  • Our preclinical study confirms Cymerus™ MSCs have significant beneficial effects on three key components of asthma: airway hyper-responsiveness, inflammation and airway remodeling4. Read more here.

Scientists and clinical physicians are seeing the benefits of using MSCs as therapies, and we believe our technology is leading the way to enable commercial production of these therapies for patients in need. We have already reached a major milestone in the field of regenerative medicine, being the first to commence clinical trials with an MSC product derived from iPSCs. Our Cymerus™ technology has the potential to produce life-saving therapies, so we are working to make the future, the present and its exciting times ahead!

 

[1] Clinical trials, Homepage. Available from: www.clinicaltrials.gov. [7 March 2017].

[2] APS 2017, percentages_of_common_diseases, image. Available from: http://www.nature.com/aps/journal/v34/n6/fig_tab/aps201350f3.html#figure-title. [7 March 2017].

[3] National Institutes of Health 2016, The Promise of Induced Pluripotent Stem Cells (iPSCs). Available from: https://stemcells.nih.gov/info/Regenerative_Medicine/2006Chapter10.htm. [2 March 2017].

[4] ASX 2017, CYP: Prices and research. Available from: http://www.asx.com.au/asx/share-price-research/company/CYP. [7 March 2017].