For the first time stem cells used to treat immune diseases in humans.

In Australia, for the first time in history a clinical trial using induced pluripotent stem cells (iPSCs) was conducted to treat 15 patients with an immune disorder known as graft versus host disease (GvHD).

The disease is one of the most frequent and clinically significant complications after allogeneic bone marrow transplantation. Chronic GvHD usually occurs more than 3 months from the moment of transplantation and is a complex disease involving many organs and systems. Often it becomes the cause of the disability of patients and even leads to death.

Royal Prince Alfred Hospital head of Cell and Molecular Therapies Professor John Rasko said that in patients with graft versus host disease, the main target organs are the skin, intestines, liver and lungs.

“It’s an immune attack and the mesenchymal stem cells, derived from these reprogrammed cells, are capable of suppressing that kind of an immune response”, – he said.

Professor Rasko said that the early phase of the clinical trial with a small number of patients showed promising results, which were published in Nature Medicine.

“None of these patients had any complications that were of any significance”, – he said.

Professor Rasko says that the use of embryonic stem cells is associated with ethical issues, and adult allogeneic stem cells often come from different donors, which can lead to discrepancies in the results obtained or different problems. However, you can get “unlimited supplies” of reprogrammed cells.

“We were able to grow these cells indefinitely in an unlimited amount, and produce them safely and then check them and make sure they’re safe”, – he said.

Rebecca Rudy was diagnosed with leukemia in 2018, and as part of treatment at St. Vincent’s Hospital with hematologist Professor Sam Milliken, she received a donor (allogeneic) stem cell transplant previous year. Two months later, she developed GvHD.

“I felt really good. And then all of a sudden, I started to get really, really bad cramps and contractions in the stomach like I was giving birth, that bad,”– she said. “I basically had to come into hospital, get morphine injections, and then be hospitalised for a week or two weeks while they upped my steroids and other medication till I was well enough to go home again”.

Rebecca Rudy said she had four more “excruciating” seizures since the first incident in April. Although her symptoms are “minor” at present, Ms. Ruby said she would have to stay on steroids indefinitely until her condition returned to normal.

Feeling great today, Ms. Ruby said that Professor Milliken’s treatment saved her life, but she was not involved in Professor Rasko’s clinical research. However, she said that it would be “wonderful” if Professor Rasko’s treatment prevented the graft versus host reaction she had to endure in other patients.

Professor Sam Milliken, head of the Department of Hematology at St. Vincent’s Hospital, said the study looks promising, especially the massive production of mesenchymal stem cells from iPSCs. However, the small number of patients means more work needs to be done, he said.

“If you’ve only got 15 patients in this study, chance can play a much bigger role than if you have 500 people in the study”, – he said.

Professor Milliken said it would be important to see how these results are replicated in larger randomized trials to ensure that induced pluripotent stem cells play a therapeutic role.

“I don’t want to downplay it, it’s a very good result, but it needs to be confirmed with some bigger studies”, – he said.

Progress in treatment with induced pluripotent stem cells has been limited so far, mainly due to technical difficulties. Professor Rasko said that the current study, conducted in conjunction with the medical company Cynata, has shown that these obstacles can be overcome.

“Now we’ve set the scene for using these cells to give rise, for example, to bone cells where we can treat people who have fractures which aren’t repairing, or people who need a new kidney, potentially”, – Professor Rasko said. “Ultimately, we dream of the day where we will be able to regenerate human tissues, and this is the first little step in that direction”.