For highly aggressive types of blood cancer, stem cell transplantation is often the only potentially curative therapy, yet even after a transplant, these cancers often return. 

According to the study’s corresponding author, John F. DiPersio, MD, Ph.D., the Virginia E. & Sam J. Golman Professor of Medicine at WashU Medicine, this gene-editing technology could help address a longstanding frustration in the field: CAR-T cell therapy—an immunotherapy that effectively treats some aggressive blood cancers—has not worked against all blood cancers, including acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).

According to DiPersio, who treats patients at Siteman and is a research member there, myeloid cancers like AML and MDS are tricky to treat with CAR-T cells because the same proteins on cancer cells that the immunotherapy homes in on for destruction are also present on healthy myeloid cells, including therapeutic donor stem cells.