Researcher awarded $12 million for a stem cell trial to improve outcomes of young blood cancer patients | News Center

The California Institute for Regenerative Medicine has awarded nearly $12 million to support a clinical trial of a new cell-based treatment to improve outcomes and survival rates in children and young adults with blood cancers who receive a stem cell transplant.

The treatment, called T-allo10, aims to improve the immune response to pathogens and cancer without increasing the likelihood of graft-versus-host disease in patients who need to receive a transplant from an unmatched donor.

The trial will be led by Maria Grazia Roncarolo, MD, professor of pediatrics and medicine. Roncarolo is the George D. Smith Professor of Stem Cell and Regenerative Medicine, director of the Stanford Center for Definitive and Curative Medicine, and co-director of the Institute for Stem Cell Biology and Regenerative Medicine.

“Each year, about 500 children receive stem cell transplants in California, and while many children are doing well, too many children experience transplant rejection or cancer relapse,” said Maria Millan, MD, president and CEO of the institute. press release. “Finding an improved therapy for these children means a shorter hospital stay, less risk of needing a second transplant and a higher quality of life for the child and the entire family.”

The current standard of care for many blood cancers is a two-part chemotherapy regimen to destroy a patient’s cancer cells, followed by a transplant of blood and immune stem cells from an immunologically matched donor. However, sometimes patients only have the option of receiving a transplant that is a partial immunological match, increasing the risk of graft-versus-host disease, where the donor’s immune cells attack the recipient’s tissues. To reduce this risk, some of the donated cells are removed prior to transplantation, which in turn increases the chance of a cancer relapse or a dangerous infection.

Roncarolo and her team will test T-allo10, in which mature immune cells are co-administered with cells called type 1 regulatory T cells, or Tr1 cells, from the donor after a stem cell transplant. The Tr1 cells, originally discovered by Roncarolo’s team, reduce the chance that the donor’s immune cells will perceive the recipient’s tissue as foreign.

T-allo10 is intended to improve transplant outcomes by reducing cancer and infection rates, as well as the risk of graft-versus-host disease.

“My team and I are excited to receive CIRM’s support for our clinical trial of immunotherapy, which can help leukemia patients who receive blood stem and progenitor cell transplants from non-perfectly matched donors — a population that continues to experience poor outcomes and which has a high unmet need,” said Roncarolo. “T-allo10 is unique in that it contains both Tr1 cells, which prevent graft-versus-host disease and rejection, as well as cells that can fight infection and remove residual cancer cells in a single-cell product.The development of a bench-to-bedside Tr1-cell-based product to improve stem cell transplant outcomes and induce tolerance is a shining example of the pioneering translational work performed at Stanford Center for Definitive and Curative Medicine.”