Year of Grant: 2016

Location: United States

Acute Myeloid Leukemia (AML) is a frequently diagnosed blood cancer that is in need of new therapies as patients suffering from this high-risk disease have little chance of cure.

Approximately 20,000 new AML cases are diagnosed in the United States annually with estimated 10,000 individuals projected to die of the disease in 2015. The general standard of care to treat AML is using chemotherapy to destroy the leukemia cells, followed by stem cell transplant (SCT). Unfortunately, some patients develop graft vs. host disease following SCT with leukemia relapse still occurring in the majority of patients.

In this phase I/II clinical study, the team is seeking to utilize a specific type of immune cell called T-cell, which has a natural ability to seek out and kill malignant cells while leaving normal cells unharmed. They will generate T
cells that recognize multiple different TAAs (tumor associated antigens), allowing them to effectively kill a diverse array of malignant cells when administered to patients.

Importantly, this strategy should also minimize tumor immune escape due to TAA loss – a problem that has emerged in other T-cell clinical trials where a single TAA was targeted. This approach is expected to be both safe (nontoxic) and highly effective.

Correlative studies will also be performed to further understand the 
expansion, persistence, anti-tumor and immunomodulatory effects in vivo. Dependent on the toxicity outcomes, a minimum of 2 and a maximum of 14 patients will be accrued in each disease group, with a total number of
patients ranging between 4 and 28.

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NOTE: This is one of three winning proposals for the 2016 RTFCCR/LLS International Research Grant to advance immunotherapy research for blood cancer.