A new study presented at the American Society of Hematology 2023 Annual Meeting explores the use of CRISPR technology as a potential treatment for high-risk acute myeloid leukemia (AML). The phase 1/2 trial, led by Guenther Koehne, MD, PhD, Deputy Director and Chief of Blood and Marrow Transplant at Baptist Health Medical Group, focuses on the safety and efficacy of trem-cel, a CRISPR/Cas9 gene-edited allograft lacking CD33.
AML patients at high risk of relapse, particularly those with FLT3-positive AML and TD53-mutated AML, face challenges even after receiving allogeneic transplants. Post-transplantation maintenance treatments have been difficult due to the presence of markers expressed on both leukemia cells and healthy stem cells. However, the use of CRISPR technology allows for the downregulation of CD33 expression on healthy stem cells prior to transplantation, while maintaining its overexpression on leukemia cells.
With a CD33-negative stem cell product from the donor, patients can experience normal reconstitution and blood production, while the cells are all CD33-negative. This opens opportunities for targeted treatments that specifically address CD33-positive leukemia cell populations. The study represents a significant step forward in personalized medicine for AML patients, offering new potential avenues for treatment and improved outcomes.