Dr. Damon on Drug Development in AML

Lloyd Damon, MD, director of the Adult Blood and Marrow Transplant and Hematologic Malignancies Program, and chief of the University of California, San Francisco (UCSF) Hematology Clinic, UCSF Helen Diller Family Comprehensive Cancer Center, discusses drug development in acute myeloid leukemia (AML).

Venetoclax (Venclexta) has shown activity in almost every hematologic malignancy in which it is tested, explains Damon, including AML. The oral drug is fairly well tolerated and has tremendous activity in older patients in combination with a hypomethylating agent, such as 5-azacytidine (Vidaza) or decitabine. For patients who are ineligible to receive intense inpatient chemotherapy, this may change the standard of care, adds Damon.

In terms of immunotherapy, it has been difficult to identify targets that are differentially expressed. Nonetheless, researchers are looking at CD123 as a potential target because it is differentially expressed on what is considered the leukemic stem cell and underexpressed on the normal hematopoietic stem cell. A number of trials using bispecific T-cell engagers or dual-affinity retargeting as immunotherapy directed against CD123 are now ongoing.