What is regenerative medicine? Cynata’s CEO explains
Health & Biotech
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Special Report: Stem cells still have somewhat of a bad rep, as news headlines either repeat the embryo origin story or recount shills promising them as a cure-all for everything from ADHD to Alzheimer’s.
But beneath those headlines is a flourishing industry, emerging as a more mainstream therapeutic approach under the name ‘regenerative medicine’.
“Regenerative medicine covers a broad range of topics but essentially the trend is to extend our health span,” says Cynata Therapeutics (ASX:CYP) CEO Ross Macdonald.
“Obviously we have a lifespan, but broadly what we’re looking to do with regenerative medicine is to extend the healthspan and therefore be healthier within our life span.”
That’ll keep people out of hospitals, in the workforce, and living their best life for longer.
The rejuvenated industry sources stem cells primarily from non-embryonic origins and is focused on creating therapeutic products that can be used “off-the-shelf” by the masses — rather than bespoke, extremely expensive treatments.
Furthermore, the idea is that as these cells have the capacity to profoundly influence their environment within the body, they can be used to help the body fix itself after a catastrophic health crisis, like a heart attack.
So far, so, science fiction.
But we’re not quite there yet: the race is on to turn stem cell treatments into a ‘one-stop-shop’ drug, rather than the bespoke, very expensive individual treatments currently on offer.
There are a handful of regenerative medicine companies on the ASX.
Macdonald says Cynata is the only stem cell play in the world that can produce therapeutic mesenchymal stem cells (MSCs) on a commercial scale without requiring multiple donors.
MSCs are adult stem cells which can be isolated from donor human tissues such as bone marrow which requires a painful bone marrow extraction.
Cynata’s manufacturing solution is its patented Cymerus process, which uses induced pluripotent stem cells (iPSCs) to make MSCs: ‘pluripotent’ means the iPSCs can develop into any type of adult cell and be generated from cells anywhere in the body – typically skin and blood cells – and grown in limitless quantities in the lab. This does not require the use of embryos or the aforementioned bone marrow extraction.
“We have taken an approach that manufacturing is the major challenge to industrialising stem cell therapeutics. Our approach is to instead of relying on lots of donors and massive expansion, we use IPSCs so we only need one human donor, one time, and then have the capacity to manufacture a consistent and potent product, essentially without limitation,” Macdonald says.
Using this process Cynata has developed a treatment which, following a very successful Phase 1 trial, is currently being prepped for phase 2 clinical trials for Graft versus Host Disease (GvHD), an immunological disease where donor cells attack the recipient, osteoarthritis, and critical limb ischemia (a severe arterial blockage that prevents blood flow to the extremities).
Others in the space include Regeneus (ASX:RGS), which is looking at how stem cells’ anti-inflammatory effect can repair knee cartilage affected by osteoarthritis.
Mesoblast (ASX:MSB) is also looking hard at GvHD and other indications like heart disease.
And ExoPharm (ASX:EX1) is looking at using exosomes, a derivative of stem cells that act as a kind of cellular courier that shuttles genetic information and proteins between cells, as the key to curing age-related illnesses.
All of these companies say they have a working method with which to make a stem cell-based medicine for mass production.
This is called an ‘allogeneic’ treatment — drugs can be made from stem cells that don’t belong to the patient — and is one of the holy grails of stem cells treatments.
On the other side are the ‘autologous’ treatments — drugs made from your own stem cells, an expensive and time consuming process.
Where ASX companies like Cynata are looking is at harnessing the body’s own cells to repair itself.
Cynata’s Macdonald says the trick is taking lessons from different biological breakthroughs to make one really effective treatment.
“What biology is revealing to us now are the processes by which the body repairs itself. That process breaks down as we get older, so it’s down to a balance of repair to regeneration, and as we get older the balance moves towards degeneration,” he says.
“CAR-T cell immunotherapy, for example, turbo-charges the body’s own immune system to attack cancer cells. The results have been very good and it provides a useful example of a cellular therapy involving the harnessing of the body’s own tissue to treat its own problems.”
Macdonald is taking the lessons learned from the body’s repair and regenerative processes, particularly where MSCs are involved and applying it to GvHD.
“We know GvHD is caused by an overreaction by the donor immune cells against the host so we’re using MSC [adult stem cells] to temper that rogue response without destroying or over-inhibiting the whole immune system,” he said.
“We’re unfolding many of the mysteries around how we regenerate damaged tissues, and that includes how the immune system works in fighting off disease.”