The trial is being launched by the University of Florida Center for Regenerative Medicine in collaboration with RESTEM.
The University of Florida (UF) Center for Regenerative Medicine has launched a phase 1 trial (NCT04723303) of umbilical cord lining stem cells (ULSCs) for the potential treatment of 2 autoimmune disorders, dermatomyositis and polymyositis, in collaboration with RESTEM.
“We’re excited to embark on this innovative and important research as we examine potential therapies for these intractable diseases,” David R. Nelson, MD, senior vice president for health affairs, UF and president, UF Health, said in a statement. “We are enthusiastic about this collaboration with RESTEM on this novel approach and grateful for the generous support of the donor.”
The trial will enroll participants to be assigned to 1 of 3 dose groups of intravenous infusions of 50 million, 100 million, or 200 million stem cells, with 3 patients in each dose group. An independent review group will evaluate data from each dose group before moving to the next. The trial is primarily evaluating the number of participants with dose-limiting toxicities within 24 hours of infusion.
“In an autoimmune condition, the immune system attacks the body’s own tissue,” Keith March, MD, PhD, director, UF Center for Regenerative Medicine, added to the statement. “Our hope is that this novel stem cell approach will have an anti-inflammatory effect through a decreased immune response, thereby protecting the body. If the therapy works, this stem cell therapy might help patients avoid having to take immunosuppressive drugs with potentially significant adverse side effects. Right now, those drugs are the only known treatment for these disorders.”
The trial is supported by private funding and approved by the FDA and the Institutional Review Board at UF Health. The trial is believed to be the first of its kind in the US evaluating possible therapies for dermatomyositis and polymyositis. A phase 2 trial will be conducted to evaluate efficacy of ULSCs in a larger number of patients with these disorders if data from the phase 1 trial are favorable.
“It is a pleasure to work with the amazing regenerative medicine center at the University of Florida,” Rafael Gonzalez, PhD, senior vice president of research and development, RESTEM, added to the statement. “We are excited to launch this study and explore the potential effectiveness of our novel and proprietary umbilical cord lining stem cell therapy.”