Cynata – stem cell industry
Investors looking for a sector that has the potential to deliver robust returns might like to take a look at the stem cell industry. Stem cells for many still brings to mind human embryo research, and if they have a vivid imagination well, a science of which Dr. Frankenstein would unquestionably have approved. But stem cell technology, aka ‘regenerative medicine’ offers very real hope to those suffering Parkinson’s disease, stroke, spinal cord injuries, retinal diseases, Alzheimer’s, Type 1 diabetes and a host of auto immune diseases such as Crohn’s. It’s not far off either.
Scientists will always be viewed with some degree of skepticism of course. After all they do sometimes get it wrong. However looking at the stem cell industry now, one can’t help drawing comparisons with the monoclonal antibodies sector in the 80s and 90s. Many then – scientists and sophisticated investors alike, viewed the science as a pipe dream. But after the FDA approved the first product in 1997 the industry has boomed, and by 2013 had achieved combined product sales of USD 60 Billion! There are now 14 monoclonal antibody ‘blockbusters’, with six of them in the world’s 20 biggest selling drugs. Needless to say there are now hundreds of antibodies in development across the planet. The science among big pharma has now become routine.
Stem cell technology is already out of the blocks with a rapidly developing clinical trial record along with many clinical successes. Add to this that the regulators have made a decision to move faster in this sector, the FDA now requiring a single Phase 2 and a single Phase 3 for some regenerative medicines. This is a global phenomenon too. In 2013 Japan passed amendments to its ‘Pharmaceutical Affairs Law” defining new medical products, ie those containing regenerative medicine products. This will allow the country’s Ministry of Health to give conditional approval (and for reimbursements to be obtained) to such products if their safety and efficacy is confirmed after just Phase 2.
Other boxes that needed ticking were the earlier ethical issues posed by the use of cells derived from embryos, along with production limitations implicit in the use of such material.
Recently though, there has been some very exciting advances, largely due to the development of a different type of stem cell – mesenchymal stem cells (MSCs). MSCs are very stable multipotent stem cells. MSCs possess properties that are ideal for the treatment of inflammatory and degenerative diseases. They possess natural abilities to detect and respond to pathological changes in their environment, such as inflammation. They can then induce the release of bioactive agents and can facilitate the formation of progenitor cells in reponse to these changes. MSCs have also been shown to actively migrate to sites of inflammation far from the injection site; and they’ve now been used to treat a wide variety of disorders including cardiovascular diseases, spinal cord injury, multiple sclerosis and complications associated with diabetes. There are currently around 300 ongoing human clinical trials using MSCs.
So the next stage? Well the final hurdle is the ability to produce stem cells on an industrial scale, in effect to make an ‘off the shelf’ product. Otherwise it’s a marginalized field. Stem cells companies have been isolating their MSCs from bone marrow or other donor tissues such as fat, and then attempting to expand these to make enough for a few thousand doses. Then they have to start the whole process again with a new donation. Cottage industry production. But a company called Cynata Therapeutics Ltd., only this year proved it can make MSCs using a scalable, reproducible and consistent process under GMP – that’s ‘Good Manufacturing Practice’, the set of standards laid down by the regulators for the production of clinical grade therapeutic products. They’ve really achieved this by a clever combination of science and engineering.
Ethical issues? Cynata are able to generate their stem cells using just one single donation from one adult human donor. They do not have to constantly seek out fresh donors with all the issues that entails, and can get on with producing virtually limitless quantities of a consistent product.
The global stem cell technology sector is now one of the fastest developing areas of the healthcare market, projected to grow to nearly USD12.2 billion in 2018. Research Council funding and government grants have recently come into the mix with private equity and venture capital money all seeing the massive potential in what will be a revolution in healthcare. Investors are already making waves. Last year stem cell giant Mesoblast raised USD 170 million via a private placement. Following this, in July, Mesoblast announced that its partner, JCR Pharma, planned to launch the world’s first allogeneic MSC cell therapy product in a developed world market – Japan. This could be followed by a US launch only next year. And of course the pharmaceutical giants are now beginning to get on board. One example – Swiss company Novartis, has invested USD 35m in the Israeli stem cell specialist Gamida Cell. In exchange Novartis will receive a 15% equity and an option to fully acquire the company. These companies will require consistent and scalable manufacture for their stem cell medicines, something Cynata can provide.
Yes the word revolution is no hyperbole in this instance. Stem cells, as they’ve always done will change human life forever! Did you see what I did there? Regenerative medicine IS the future, and a closer look at the many companies in the field may be worth your time and effort. It could also give you the very real opportunity of becoming part of the revolution and the future of medicine.
Author : Lawrence Barrett
More about Cynata : http://cynata.com/