A unique method of increasing the number of cord blood stem cells used to treat patients with blood cancers and blood disorders, such as sickle cell anemia, is being readied for clinical trials at the Icahn School of Medicine at Mount Sinai, with an $8.8 million grant from the New York State Stem Cell Science Program (NYSTEM).
The stem cells—also known as hematopoietic stem cells—are derived from the vein of the umbilical cord and help renew and replenish blood cells. They represent the only potential therapy for blood cancer patients who do not respond to chemotherapy. The new method is necessary to compensate for the limited number of stem cells that are typically found in blood cord collections and the fact that using stem cells from two or more blood cord collections is generally not a viable option because the blood cells are not identical.
A research team led by Ronald Hoffman, MD, Albert A. and Vera G. List Professor of Medicine (Hematology and Medical Oncology), and Director of the Myeloproliferative Disorders Research Program, Icahn School of Medicine at Mount Sinai, has pioneered a method that promises to significantly increase the number of stem cells in blood cord collections.
“I was ecstatic when we received the grant earlier this year,” Dr. Hoffman says. “It provides a unique opportunity to take something that we’ve developed in the lab and move it into the clinic.” A leader in stem cell research, Dr. Hoffman’s earlier studies of human hematopoietic stem cells and progenitor cells in myeloproliferative neoplasms have led to therapeutic advances.
Research and clinical trials funded by the NYSTEM grant will be carried out over four years in partnership with AllCells, LLC, and NeoStem Inc., two companies that specialize in cellular therapy. The clinical trials will establish whether Dr. Hoffman’s novel cell expansion method can be safely replicated with human cord blood cells.
NYSTEM aims to foster New York State’s role as a leader in health care by funding promising programs that accelerate scientific knowledge about stem cell biology and the development of therapies and diagnostics that improve human health.
“We’re going to take everything we’ve done at lab scale and see if we can make this into a process that will meet the standards of the U.S. Food and Drug Administration (FDA),” says Dr. Hoffman.
In the final step of research for the grant, Dr. Hoffman says his team expects to collaborate with a stem cell transplant group at The Mount Sinai Hospital to write clinical protocols to submit to the FDA that would allow the potentially lifesaving technique to be used in humans.
“We are very excited to have this opportunity to work with Dr. Hoffman to move this technology into large-scale production and clinical trials soon,” says Jay Tong, MD, President and CEO, AllCells, LLC.