The drug venetoclax (Venclexta) recently received FDA approval to treat chronic lymphocytic leukemia (CLL).ASCO 2016 presented a phase 1 trial in patients with advanced, relapsed or refractory multiple myeloma. Overall results showed that venetoclax significantly reduced the tumor size in 21% of patients, but that patients with a specific genomic change, specifically the translocation of chromosomes 11 and 14, t(11;14) responded best. 40% of these patients had a reduction in tumor size. The most common side effects were GI-related; the most serious were hematologic, primarily thrombocytopenia (low platelet count) and anemia. Trials are now being planned that will specifically target t(11;14) patients, underscoring the potential for precision medicine approaches. However, a phase III trial had been launched with no results yet available, adn this drug cannot be said to be supported by the medical literature on the basis of what is known now.
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