Year of Grant: 2015

Location: United States

Acute myeloid leukemia (AML) is a devastating hematologic malignancy, with a five-year survival rate of only 25 percent. Only modest advances have been made in the effectiveness of therapies over the last three decades. Patients who have high-risk or relapsed AML may be cured with an allogeneic stem cell transplant; however, co-morbidities limit the use of this option. Therefore, drugs with different cellular targets and mechanisms of action than current chemotherapies are urgently needed.

This study focuses on the clinical implementation of one such agent. The study drug (8-Cl-Ado) interferes with the intra-cellular process, which are critical for survival of these cancer cells and are preferentially toxic to these cells. In pre-clinical studies, the study drug was extremely potent against a variety of solid and hematologic cancers, particularly in leukemia and human AML.

This project intends to replicate these very promising findings in a phase I/II clinical trial in patients with relapsed/refractory AML. The phase I trial will treat up to 24 patients to determine the maximum tolerated dose, and the phase II trial will treat up to an additional 19 patients to evaluate antitumor activity and response rate of the study drug as a single agent. In parallel, correlative studies will be conducted to identify biomarkers that are predictive of response.

Successful completion of these studies could substantially benefit enrolled patients and advance this novel therapeutic drug as a precision medicine for the treatment and cure of patients with AML.