Cynata Therapeutics Limited (ASX:CYP) Managing Director and CEO Ross Macdonald details plans to commercialise its therapeutic technology.
IN a deal that plumbs the potential of one of medicine’s most hyped branches, the University of Western Australia has secured a supply of one of the human body’s most formative cells.
The Perth sandstone has partnered with one of Australia’s few commercial-scale stem cell manufacturers to find a cure for an incurable lung disease. Idiopathic pulmonary fibrosis has no known cause and limited treatment options, and a diagnosis usually means the sufferer will be dead within five years.
For the university, the alliance offers a consistent supply of identical stem cells for a trial study in animals. For the company, it offers “intellectual horsepower” to validate the products.
“There are some (deals) where the company just throws money at academics and hopes for the best,” Cynata Therapeutics chief executive Ross Macdonald said.
“In this case it’s a genuine two-way relationship. The more areas we expose our cells to, the better the commercial opportunity. If it was found that our stem cells were only useful for ingrowing toenails, they wouldn’t be much good commercially, but lung fibrosis – which is a particularly devastating condition – has an annual therapeutics market of a billion dollars.
“It’s the sort of thing which gets doctors and ultimately investors interested.”
The Centre for Cell Therapy and Regenerative Medicine, based at UWA, represents a collaboration of WA universities, hospitals and medical research institutes.
It’s looking to stem cells for solutions to scourges from heart and lung disease to Alzheimer’s, diabetes and cancer.
“(Stem cell therapy) has the capacity to transform the way we do medicine, but one of the big challenges is getting the right cells,” director Geoff Laurent said.
“Having a really tight procedure for preparing these cells, which means you get consistency from one batch to the next, is really important. You do your tests with a batch of cells, and you want to be using the same cells all the time.”
Cynata says it has found a way of mass-producing mesenchymal stem cells, which are the focus of about 300 studies into treatments for various diseases.
The approach avoids the ethical minefield of harvesting embryonic stem cells and the need for painful bone marrow extractions, with an unlimited supply of cells generated from a single blood donation.
Dr Macdonald said some Australian companies were able to produce “bespoke” stem cells, where cells from the patients themselves were reinjected.
“(That’s) fine for relatively rare diseases, but if you want to treat diseases of economic importance – like heart attack, stroke or lung fibrosis – (you need) an off-the-shelf product, where (someone’s) stem cells can be used in any patient,” he said.
Professor Laurent, who also directs the Lung Institute of WA, said the hope was that injected cells would “go straight to the tissues we want to treat (and) regenerate viable functional tissue”.
He said there had been significant breakthroughs in treating blood cancer and macular degeneration using stem cells.
While the field was still to live up to its more general promise, “at some stage people will be treated with stem cells off the shelf. I don’t know when it will be, but it will come, and I don’t want Australia to be behind the eight ball.
“We need to be leading in this area. It will be good for people’s health and good for Australia’s economy.”
Cynata featured on leading independent financial analysis and news website, FNArena
Stem cells have a potential to develop into multiple cell types and divide without limit to replenish cells in human body throughout the life of individual.
There are two major types of stem cells embryonic and adult stem cells (also called somatic stem cells). Adult stem cells reside within different organs and usually regenerate tissue in locations where they live in. Adult stem cells can be differentiated into multiple cell types and therefore called multipotent stem cells.
In contrast, embryonic stem cells are capable to produce any type of cell found in a human body. That is why embryonic stem cells are called pluripotent stem cells.
Human embryonic stem cells were first isolated in 1998 by James Thomson at University of Wisconsin, Madison. This discovery significantly elevated the interest in stem cell biology because many viewed embryonic stem cells as unlimited source of cells for regenerative medicine.
Advances in understanding embryonic stem cells soon led to another discovery by Shinya Yamanaka at Kyoto University who demonstrated in 2006 that adult somatic cells can be turned into embryonic stem-like cells, so called induced pluripotent stem cells.
In my laboratory we study differentiation of pluripotent cells toward blood and vascular cells. In 2010 we identified a novel multipotent progenitor mesenchymoangiobalst (MCA) which formed during differentiation of human pluripotent stem cells in a culture dish.
The MCA has a capacity to produce variety cell types, including major components of blood vessels (endothelial cells which form blood vessel lining, pericytes which stabilize small vessels, and smooth muscles a major component of large vessel wall) and mesenchymal stem cells (MSCs) with a potential to produce bone, cartilage, and adipose tissue.
Because pluripotent stem cells can expand prodigiously, our technology makes it possible the scalable manufacturing of different types of vasculogenic cells and MSCs.
Melbourne-based company Cynata acquired intellectual property related to MCA technology from WARF with the goal to advance this technology into the clinic.Currently we are at pre-clinical stage.
This technology makes it possible to produce many different types of cells for cellular therapies, including mesenchymal stem cells (MSCs). MSCs generated a great deal of interest because their potential to encourage the regeneration and repair of the damaged tissue and reduce inflammation.
Multiple clinical trials were initiated to test the use of MSCs for treatment of variety of medical conditions. However, commercial-scale manufacture of current MSC products is a major practical and regulatory challenge, because limited expansion potential of adult stem cells and significant donor-to-donor variations.
Our technology enables the unlimited production of uniform, pharmaceutical grade MSCs from a single donor. This means easier regulatory route, easier manufacturing, and greater clinical predictability.
During the last decade significant progress has been made in understanding stem cell biology and their differentiation toward particular cell type. Multiple clinical trials have been initiated to explore potential of MSCs and other types of adult multipotent stem cells to heal tissue damage and reduce inflammation.
The next significant challenge is to use pluripotent stem cells to generate tissues for transplantation. To be used for transplantation, we need to find the way to produce cells that integrate into the recipient organs and function properly throughout the duration of life recipient’s life.
Only few clinical trials have been initiated with cells derived from pluripotent stem cells. It will be critical to demonstrate the safety of pluripotent stem cell-based therapies.
Aging population and the rise in chronic degenerative diseases dictate the need for novel therapies to regenerate the damaged tissue. Stem cell technologies have a potential significantly improve management of medical conditions associated with tissue damage.
Stem cell therapies are rapidly developing. Recently significant effort has been made to translate pluripotent stem cell technologies into the clinic. I am sure that within several years we will see more clinical trials with stem cell therapies. Recently, Japanese government approved the law that provide rapid approval process for human stem cell-based therapies. This law will facilitate swift translation of novel stem cell technologies into the clinic.