Brief background of the proposal and its relevance to the CDI’s objectives
Pediatric acute myeloid leukemia that has relapsed or is refractory to standard chemotherapy or hematopoietic cell transplantation (HCT) has a poor prognosis. This project applies a new form of immunotherapy discovered at Washington University as a treatment for these patients. The treatment includes harvesting a donor’s immune cells, isolating natural killer (NK) cells, and activating them to potently respond to the patient’s leukemia blasts. This overarching goal of this project is to advance novel NK cell immune therapies for patients with pediatric blood cancers.
Proposed specific aims·
- To complete a pilot clinical trial of donor memory-like NK cells infused with a DLI, or donor memory-like NK cells followed by rhIL-2, for pediatric/YA patients that have relapsed AML.
- To elucidate the expansion, activation, persistence, and functionality of transferred memory-like NK cells within pediatric and YA leukemia patients treated on these clinical trials.
Potential impact on child health
Two clinical trials are now open and available for pediatric or young adult patients that have relapsed or
refractory AML prior to hematopoietic cell transplant (HCT), or AML patients that have relapsed after
undergoing an allogeneic hematopoietic cell transplant. These patients have very poor long term clinical
outcomes, and this therapy provides a new avenue to utilize anti-tumor immunity from various immune
effectors to fight the leukemia. Thus, this new personalized NK cell immunotherapy approach has the
potential to cure relapsed pediatric and YA patients in a situation where effective treatments have yet to be