Cambridge, MA November 23, 2009 Agios Pharmaceuticals today announced that its scientists have established, for the first time, that the mutated IDH1 gene has a novel enzyme activity consistent with a cancer-causing gene, or oncogene. This breakthrough discovery shows that the mutated form of IDH1 produces a metabolite, 2-hydroxyglutarate (2HG), which may contribute to the formation and malignant progression of gliomas, the most common type of brain cancers. This discovery appears to reverse the previously held belief that IDH1 was non functional for cancer-causing activity. It is also one of the first reported instances where a metabolic enzyme such as IDH1 is shown to play a role in cancer formation, in this case through altered metabolic activity.
This finding creates opportunities for therapeutic intervention in brain cancer and other cancers where IDH1 mutations are present using new drugs that can target the IDH1 metabolic pathway.. The Agios research also identified an exciting new biomarker, 2HG, that could be used to develop an important diagnostic. The research was published on November 22 by the journal Nature, in a paper entitled "Cancer-associated IDH1 mutations produce 2-hydroxyglutarate (2HG)".
"This groundbreaking work is profound for the field," said Professor Lew Cantley, Ph.D., Director of the Cancer Center at the Beth Israel Deaconess Medical Center, a founder of Agios and a supporting author. "The team at Agios has demonstrated that what was previously considered an inactive enzyme is in reality an active oncogene and a potential therapeutic target. This has fundamentally changed our understanding of the field. Additionally, there is an easily measured metabolic biomarker, 2HG, that will help in the diagnosis and treatment of any related therapeutics that arise from this work."
Agios scientists uncovered the function of the IDH1 mutation by employing novel techniques in a new area of cancer biolog
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