Newswise — (New York, NY – April 9, 2020) Mount Sinai Health System is the first in the country to use an innovative allogeneic stem cell therapy in COVID-19 patients and will play a central role in developing and conducting a rigorous clinical trial for patients with severe acute respiratory distress syndrome, the breathing illness that afflicts people who have severe cases of COVID-19.
The therapy, known as remestemcel-L, has previously been tested in bone marrow transplant patients, who can experience an overactive immune response similar to that seen in severe cases of COVID-19.
Mount Sinai began administering the therapy, known as remestemcel-L, to patients in late March under the Food and Drug Administration’s compassionate use program, which allows patients with an immediately life-threatening condition to gain access to an investigational therapy. Ten patients with moderate to severe cases of COVID-19-related acute respiratory distress syndrome (ARDS), most of whom were on ventilators, were given the therapy and doctors saw encouraging results.
“We are encouraged by what we have seen so far and look forward to participating in the randomized controlled trial starting soon that would better indicate whether this is an effective therapy for patients in severe respiratory distress from COVID-19,” said Keren Osman, MD, Medical Director of the Cellular Therapy Service in the Bone Marrow and Stem Cell Transplantation Program at The Tisch Cancer Institute at Mount Sinai and Associate Professor of Medicine (Hematology and Medical Oncology) at the Icahn School of Medicine at Mount Sinai. Dr. Osman oversaw the treatment of the first Mount Sinai patients with this innovative therapy.
Under the leadership of Annetine Gelijns, PhD, Alan Moskowitz, MD, and Emilia Bagiella, PhD, of Mount Sinai’s Institute of Transformative Clinical Trials, Mount Sinai will serve as the clinical and data coordinating center for a randomized clinical trial evaluating the therapeutic benefit and safety of this stem cell therapy in 240 patients with COVID-related ARDS in the United States and Canada. The trial will be conducted as a public-private partnership between the Cardiothoracic Surgical Trials Network, which was established as a flexible clinical trials platform by the National Heart, Lung, and Blood Institute, and Mesoblast, the manufacturer of the cells.
“The coronavirus pandemic has caused exponential increases of people suffering with acute respiratory distress syndrome, requiring intubation and mechanical ventilation with many dying,” said Dr. Gelijns, who is also the Edmond A. Guggenheim Professor of Health Policy at the Icahn School of Medicine at Mount Sinai. “We have designed a clinical trial that will expeditiously determine whether the stem cell therapy will offer a life-saving therapy for a group of patients with a dismal prognosis.”
“We are interested to study the potential of this anti-inflammatory cell therapy to make an impact on the high mortality of lung complications in COVID-19 patients,” said CSTN Chairman A. Marc Gillinov, MD. “This randomized controlled trial is in line with our mandate to rigorously evaluate novel therapies for public health imperatives.”
The therapy consists of mesenchymal stem cells. These cells are found in bone marrow and serve many functions including aiding tissue repair and suppressing inflammation. The therapy was previously tested in a phase 3 trial in children who had an often-fatal inflammatory condition called graft-versus-host disease (GVHD) that can occur after bone marrow transplants.
The inflammation that occurs in GVHD is the result of a cytokine storm, which activates immune cells that attack healthy tissue. A similar cytokine storm that causes damage to the lungs and other organs appears to be taking place in COVID-19 patients who develop acute respiratory distress syndrome, said John Levine, MD, Professor of Medicine (Hematology and Medical Oncology), and Pediatrics, at the Icahn School of Medicine at Mount Sinai, who helped implement the compassionate use of the drug at Mount Sinai.
“These stem cells have shown excellent response rates in severe graft-versus-host disease in children,” said Dr. Levine, who is also the co-director of the Mount Sinai Acute GVHD International Consortium (MAGIC). “Mesenchymal stem cells have a natural property that dampens excessive immune responses.”
Several people were instrumental in quickly and efficiently working through the complex application process for each patient to gain compassionate use of the therapy. Three key players involved were Stacey-Ann Brown, MD, MPH, Assistant Professor of Medicine (Pulmonary, Critical Care and Sleep Medicine) at the Icahn School of Medicine at Mount Sinai; Tiffany Drummond, Assistant Director of Regulatory Affairs at The Tisch Cancer Institute at Mount Sinai; and Camelia Iancu-Rubin, PhD, Director of the Cellular Therapy Laboratory at the Icahn School of Medicine at Mount Sinai.
About the Mount Sinai Health System
The Mount Sinai Health System is New York City's largest academic medical system, encompassing eight hospitals, a leading medical school, and a vast network of ambulatory practices throughout the greater New York region. Mount Sinai is a national and international source of unrivaled education, translational research and discovery, and collaborative clinical leadership ensuring that we deliver the highest quality care—from prevention to treatment of the most serious and complex human diseases. The Health System includes more than 7,200 physicians and features a robust and continually expanding network of multispecialty services, including more than 400 ambulatory practice locations throughout the five boroughs of New York City, Westchester, and Long Island. The Mount Sinai Hospital is ranked No. 14 on U.S. News & World Report's "Honor Roll" of the Top 20 Best Hospitals in the country and the Icahn School of Medicine as one of the Top 20 Best Medical Schools in country. Mount Sinai Health System hospitals are consistently ranked regionally by specialty and our physicians in the top 1% of all physicians nationally by U.S. News & World Report.