The first stem cell therapy developed at UW-Madison to enter a clinical trial was found to be safe and beneficial to patients, according to the company overseeing the experimental treatment.
In a phase 1 trial, concluded this year, 15 patients received the therapy in England for graft versus host disease, or GVHD, a complication of bone marrow transplants in which donor cells attack the recipient’s body.
No safety concerns were reported. Fourteen patients showed improvement in their GVHD, including eight for whom all signs and symptoms of the condition went away, Australia-based Cynata reported in August.
The therapy is based on research by Dr. Igor Slukvin, a UW-Madison pathologist. A co-founder of Cynata, he figured out how to direct induced pluripotent stem cells, or iPS cells, to grow into a steady supply of mesenchymal stem cells, blood cells found in bone marrow.
Through the Wisconsin Alumni Research Foundation, Slukvin patented the technology, which was licensed by Cynata. The cells used in the clinical trial were produced at Waisman Biomanufacturing — part of the university’s Waisman Center — a clean-room facility that develops clinical-grade cells for human trials.
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Patients in the study had steroid-resistant GVHD, meaning they didn’t respond to steroids, the main treatment for the life-threatening condition. Mesenchymal stem cells are thought to help by modulating the immune system, but doctors have faced challenges in obtaining a reliable supply of such cells from donors.
By using iPS cells, “we can produce an unlimited number of mesenchymal stem cells from a single donor,” Slukvin said. “They suppress abnormal activation of the immune system and alleviate GVHD.”
Fujifilm has a license option to pursue development of the therapy. The Japanese company owns Madison-based Cellular Dynamics International, co-founded by Slukvin and UW-Madison stem cell pioneer James Thomson, among others.
Cynata plans to study the therapy in patients with other conditions, including inadequate blood flow to the limbs.
Slukvin is also working on growing iPS cells into an immunotherapy for cancer.