Researchers at the University of North Carolina at Chapel Hill School of Medicine have identified a compound that could be modified to treat one of the most deadly types of cancer, and discovered how a particular gene mutation contributes to tumor growth.
The findings and potential treatment apply to a type of brain tumor called secondary glioblastoma multiforme (GBM). GBMs are part of a larger group of brain tumors called malignant gliomas, which is the type of cancer Senator Edward Kennedy suffers from.
A report of the research will appear in the April 10, 2009 issue of the journal Science .
In experiments with tumor cells, the researchers reversed the effects of a mutation in a gene called isocitrate dehydrogenase-1 (IDH1) by replenishing a compound called á-ketoglutarate (á-KG).
"When the IDH1 gene is mutated, the level of á-KG is reduced, which in turn contributes to tumor growth by helping to increase the supply of nutrients and oxygen to tumor cells. When we added the á-KG to tumor cells, the effects caused by the IDH1 mutation were reversed," said Yue Xiong, Ph.D., William R. Kenan Jr., Distinguished Professor of Biochemistry and Biophysics and a member of the UNC Lineberger Comprehensive Cancer Center.
"If scientists can develop á-KG into a clinical drug, it could potentially be used for treating brain tumor patients who have this specific gene mutation. The á-KG compound is already there; it only needs to be modified to be used clinically, so that may save a lot of time," Xiong said.
Xiong is a corresponding author of the study along with Kun-Liang Guan, Ph.D., professor of pharmacology at the University of California, San Diego.