Cancer Metabolism Drug AG-221 Shows Clinical Activity in Advanced Blood Cancers

“AG-221, a novel inhibitor of isocitrate dehydrogenase (IDH) 2-mutant metabolic enzyme, was well tolerated and showed early promise in patients with advanced and refractory blood cancers harboring IDH2 mutations, according to the initial results of a phase I study presented here at the AACR Annual Meeting 2014, April 5-9.

” ‘Mutations in the genes for the metabolic enzymes IDH1 and IDH2 are thought to be the drivers of distinct subsets of acute myeloid leukemias (AML),’ said Eytan M. Stein, M.D., assistant attending physician in the Leukemia Service at Memorial Sloan Kettering Cancer Center in New York. ‘They lead to the production of increased levels of an oncometabolite called 2-hydroxyglutarate (2-HG), which is hypothesized to prevent normal healthy bone marrow cells from maturing, leading to cancer.’ ”

Editor’s note: AG-221 is a targeted therapy drug. The goal of this phase I clinical trial was to test the safety of AG-221 for patients. The trial found that AG-221 is safe and shows real promise as a cancer-fighting treatment for patients whose blood cancers have mutations in the IDH2 gene, as detected by molecular testing.