The lead investigator of the world’s first human clinical trial using reprogrammed stem cells to treat an immune disorder is describing it as a breakthrough that could boost the industry.
Royal Prince Alfred Hospital Professor John Rasko AO published a paper in Nature Medicine describing how the team used pluripotent stem cells produced by Cynata Therapeutics (ASX:CYP) to treat 15 patients with steroid-resistant acute graft-versus-host disease, a complication that affects people who have received bone marrow transplants.
While the results of the study were first disclosed by Cynata back in December 2018, this is the first time the results have been published in a peer-reviewed journal.
“You don’t get published in Nature Medicine without extremely rigorous peer review,” Professor Rasko told Stockhead.
“This is being hailed as a world first.”
Specifically, it’s the first time a human trial using induced pluripotent stem cells has been completed for any disease worldwide, he said.
A pair of scientists, Sir John B. Gurdon of the University of Cambridge in England and Shinya Yamanaka of Kyoto University in Japan, won the Nobel Prize in Medicine in 2012 for discovering that mature cells could be reprogrammed in such a way.
While Professor Rasko said he was optimistic the team’s discovery could contribute to the management of graft-versus-host disease, he saw the real promise of the study as a proof-of-concept of the potential of pluripotent stem cells.
Graft-versus-host disease occurs when a donor’s white blood cells treat a host’s healthy cells as foreign and attacks them.
While graft-versus-host disease is a rare disorder, it will be familiar to those following the ASX biotech space, with fellow Melbourne-based Mesoblast (ASX:MSB) having developed a treatment for it that’s before the US Food and Drug Administration for approval this month.
Professor Rasko said the genius of Cynata’s Cymerus technology was that it could be used to create 30 million doses of stem cells from a single donation of whole blood.
“No one else can claim anything like that,” he said.
Because so many stem cells can be manufactured from just a single donation of blood, that solves issues of donor-to-donor variability, Professor Rasko said.
The hope is that now that it has been proven that induced pluripotent stem cells can be manufactured at scale, they can be used to treat more common disorders.
“The whole idea of stem cells is that they can create any cell in the body in a dish,” Professor Rasko said.
Dr Kilian Kelly, Cynata’s chief operating officer, said the company planned to commence clinical trials of Cymerus mesenchymal stem cells to treat osteoarthritis and COVID-19 in the near future.