Journal article

Venetoclax in Patients with Previously Treated Chronic Lymphocytic Leukemia

Andrew W Roberts, Stephan Stilgenbauer, John F Seymour, David CS Huang

Clinical Cancer Research | AMER ASSOC CANCER RESEARCH | Published : 2017


Venetoclax is the first BCL2 inhibitor to enter routine clinical practice. It is an orally bioavailable small molecule that binds BCL2 very specifically. Acting as a pharmacologic mimic of the proteins that initiate apoptosis (a so-called BH3 mimetic), venetoclax rapidly induces apoptosis in chronic lymphocytic leukemia (CLL) cells, which express high levels of BCL2 and rely on it to maintain their survival. As a single agent, daily venetoclax treatment induced durable responses in 79% of patients with relapsed or refractory CLL or small lymphocytic lymphoma in a phase I study, including complete remissions in 20% of patients. Its use was approved by the FDA in April 2016 for patients with p..

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Awarded by National Health and Medical Research Council of Australia

Awarded by Leukemia & Lymphoma Society

Awarded by Victorian Cancer Agency

Awarded by DFG

Funding Acknowledgements

Research by A.W. Roberts and D.C.S. Huang is supported by fellowships and grants from the National Health and Medical Research Council of Australia (1079560, 1043149), the Leukemia & Lymphoma Society (7001-13), the Victorian Cancer Agency (TRP13041), and the Australian Cancer Research Foundation. S. Stilgenbauer received support from EC TransCan program (FIRE CLL), DFG (SFB1074 subproject B1), and BMBF (PReCISE).