A University of Miami Miller School of Medicine research team has launched a Phase 3 multicenter clinical trial to evaluate the effectiveness of mesenchymal stem cells in treating COVID-19 patients with acute respiratory distress syndrome.
“There is an urgent need for novel therapies that can dampen the excessive inflammatory response in the lungs from COVID-19, especially in unvaccinated individuals,” said Camillo Ricordi, M.D., the Stacy Joy Goodman Professor of Surgery, Distinguished Professor of Medicine, professor of biomedical engineering, and microbiology and immunology and director of the Diabetes Research Institute (DRI) and Cell Transplant Center.
“We hope that the use of mesenchymal stem cells (MSCs) will also accelerate the regeneration of healthy lung tissue,” said Dr. Ricordi. “We are also working on preventive strategies to inhibit viral replication, regardless of vaccination status or COVID-19 variant.”
Phase 2b/3 Trial FDA Approved
Giacomo Lanzoni, Ph.D., research assistant professor at the Diabetes Research Institute, is the principal investigator of the Phase 2b/3 trial, “Umbilical Cord-Mesenchymal Stem Cells for Patients with COVID-19,” recently approved by the U.S. Food and Drug Administration. The double-blind, randomized, controlled, multi-center trial aims to enroll 128 patients at sites in Florida, Arizona, Texas and other states. Patients hospitalized with severe COVID-19 infections will be asked to take part in the trial if there is a sudden drop in blood oxygen levels.
Dr. Ricordi said the new clinical trial builds on the successful completion of a Phase 1/2a trial, launched in April 2020 by an international team of scientists to explore the safety and effectiveness of MSCs in blocking severe lung inflammation in COVID-19 patients.
The FDA had previously authorized the testing of umbilical cord-derived mesenchymal stem cell products in patients with type 1 diabetes and Alzheimer’s disease at the University of Miami as part of other clinical trials.
That Phase 1/2a trial was completed last December, with results published the following month in STEM CELLS Translational Medicine. In that study, researchers observed that UC-MSC treatment was associated with substantial improvements in survival – 91 percent versus 42 percent in the one month after treatment, and 100 percent in subjects less than 85 years old. The UC-MSC infusions were also associated with significant improvements in time to recovery.
A Challenging Time
The UC-MSC investigational product will be manufactured at the NABTU (North America’s Building Trades Unions) Rapid Response Stem Cell Repository, a key component of the cGMP Advanced Human Cell and Biologic Manufacturing Facility of the DRI Cell Transplant Center.
“This has been an incredibly challenging effort, during unprecedented times, and we will continue to work to deliver this cell therapy to patients in need,” said Dr. Ricordi. “The FDA authorization of this Phase 3 trial will also facilitate opportunities for other countries worldwide, to duplicate our protocol for their own emergency situations.”
In addition to treating severe COVID-19, UC-MSC treatment may be beneficial for other patients with respiratory distress from other viral or bacterial causes, added Dr. Ricordi.
Confirmation of the efficacy of stem cell treatments could also expand therapies for other disease conditions characterized by hyper-inflammatory reactions, such as autoimmune diseases like Type 1 diabetes, as well as age-related chronic diseases like Alzheimer’s and Parkinson’s disease. As Dr. Ricordi said, “This could be life-changing for millions of patients in the U.S., and for hundreds of millions worldwide.”