Australian stem cell and regenerative medicine company, Cynata Therapeutics (ASX:CYP), has announced the first patient has been treated with CYP-001, its first mesenchymal stem cell (MSC) product, in its Phase 1 clinical trial in patients with steroid-resistant acute graft versus host disease (GvHD).
The company described it as a milestone and the first time in the world a patient has been treated with an allogeneic, induced pluripotent stem cell (iPSC)-derived therapeutic MSC product.
CYP-001 is manufactured in a scalable process using Cynata’s Cymerus platform with iPSCs as the starting material. Cynata said it has sourced its iPSCs from Cellular Dynamics International, a Fujifilm subsidiary company. The company recently announced a strategic alliance with Fujifilm. As part of the alliance Fujifilm has taken an approximately 9 per cent equity stake in Cynata, making it the company’s largest shareholder.
The clinical-grade iPSCs were derived from a single blood donation using a non-viral, non-integrating episomal reprogramming method, without genome modification. The Cymerus process overcomes both the need to source multiple donors and the inherent variability in products derived from multiple donations.
According to the company, a total of 16 patients are expected to participate in the phase 1 trial. The trial has been opened for recruitment at several major transplant centres in the UK and Australia. The first patient was treated at a UK centre.
“Treatment of steroid-resistant GvHD is one of the major challenges in management of patients undergoing bone marrow transplantation,” said Dr Adrian Bloor, consultant haematologist at The Christie Hospital, based in Manchester, and the UK chief investigator for this trial.
“A number of previous studies have demonstrated that MSCs can be an effective treatment, but producing consistent MSCs in sufficient numbers for clinical use has been a major challenge until now. The Cymerus process offers a solution to this problem, by enabling the large-scale manufacture of a uniform MSC product derived from a single, one-time donor. I am delighted that patient dosing has now commenced, and I am optimistic that this technology will be of benefit to patients,” said Dr Bloor.
Cynata managing director and CEO Dr Ross Macdonald says the trial was another major milestone and value catalyst for the Company.
“The treatment of the first patient in this trial marks the beginning of an exciting new chapter in stem cell therapeutics and the future of regenerative medicine,” said Dr Macdonald.
“Our Cymerus technology eliminates the reliance upon multiple donors and the need to excessively expand MSCs derived from them. Cynata is truly at the forefront of the industry with a sustainable and robust manufacturing process for therapeutic MCS products.”