Treat high risk blood cancers using novel immunotherapy and biomarkers


Year of Grant: 2017

Location: United States


Leukemia is the most common cancer in children, afflicting more than

4,000 children per year in the U.S. While advances in science have improved survival for most children, patients with Acute Myeloid Leukemia (AML)

can still expect 50% survival. If the leukemia is advanced, survival rates

drop to 20%. Currently, these patients receive high doses of chemotherapy

or radiation followed by a bone marrow transplant using marrow and immune cells from a healthy patient.


Despite this aggressive therapy, relapse occurs in more than 40% of patients, and 90% of relapsing patients will die of their disease. In part, this is due to patients being identified as leukemia-free when low levels of leukemia are still lurking in their system (minimal residual disease).


Dr. Williams’ team have developed a novel immunotherapy that expands

T cells from the donors, enabling more effective targeting and killing of leukemia using leukemia-specific proteins that leaves healthy tissues untouched. They hypothesize that this leukemia-specific T cell immunotherapy can eradicate residual leukemia, with few to no side effects, preventing relapse in even high risk patients. This phase I trial proposes

the enrollment of 18 patients in a timeframe of two years.