Jacob Appelbaum, MD, PhD, on Controlling DARIC CAR Therapy With Rapamycin for AML

“One of the key challenges in AML is that the antigens that are most commonly targeted are expressed on stem cell progenitors. And so, if those cells are eliminated, the blood cell counts in those patients will decrease, and that presents a very serious infectious risk. So, 1 question is, can we eliminate that risk through regulation, i.e., turning the CAR T -cells on and off? One of the things that we're focused on is a platform where we can do that with a small molecule drug, in this case, rapamycin. So, when rapamycin is present, the CAR T-cells are on and when rapamycin is not present, the CAR T-cells are off.”

REFERENCE

Appelbaum J, Cooper T, Annesley C, et al. first in human studies show activation of SC-DARIC33, a rapamycin-regulated anti-CD33 CAR t cell therapy, in patients with AML. Presented at: ASGCT 2023 Annual Meeting; May 16-20; Los Angeles, California.