Marie Huong Nguyen, MD, assistant professor, Hematology and Oncology, University of Michigan Health System, discusses the safety and efficacy of ABL001 as a potential treatment for patients with chronic myeloid leukemia (CML).

ABL001 is a potent, allosteric BCR-ABL inhibitor designed to treat patients with CML and Philadelphia chromosome-positive acute lymphoblastic leukemia. In a phase I study examining ABL001 in patients with CML, three dose-limiting toxicities were reported, due to reports of arthralagia (n = 1), lipase elevation (n = 1) and acute coronary syndrome (n = 1). The majority of adverse events reported were grade 1/2, Nguyen adds. There were 5 discontinuations due to disease progression.

With regard to efficacy, 11 TKI-resistant patients who received ABL001 achieved a > 1-log reduction. Five patients who had hematologic relapse also achieved complete hematologic response within 3 months. Nine out of 11 patients achieved complete cytogenetic response, Nguyen adds.

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Marie Huong Nguyen, MD, assistant professor, Hematology and Oncology, University of Michigan Health System, discusses the safety and efficacy of ABL001 as a potential treatment for patients with chronic myeloid leukemia. 


Marie Huong Nguyen, MD

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Dr. Nguyen on ABL001 in Patients With CML