A new drug for treatment of a type of brain cancer, called IDH-mutant low-grade glioma, was approved Aug. 6 by the U.S.
Food and Drug Administration (FDA). The promising new drug stems from a 2008 genetic discovery made at the Johns Hopkins Kimmel Cancer Center. The drug, called vorasidenib, is a targeted cancer therapy that works by inhibiting the activity of a mutated gene called IDH, slowing the growth of the cancer.
The gene was identified by Bert Vogelstein, M.D., and team at the Johns Hopkins Kimmel Cancer Center's Ludwig Center in 2008 when they became the first to map the genetic blueprint for brain cancer.
The blueprint was considered the most comprehensive genetic analysis for any tumor type, evaluating all known protein-encoding genes in brain cancer. The researchers found that the IDH gene-;which had never been suspected to be involved in any tumor type-;was frequently mutated in a subset of brain cancers. IDH is the poster child for cancer genome sequencing, and it illustrates the importance of basic research.
The history of medicine shows that when a disease is understood, it eventually becomes manageable. It may not be immediately evident, but in time, as in this case, such discoveries result in better treatment for patients." Bert Vogelstein, the Clayton Professor of Oncology, Howard Hughes Medical Institute investigator, and co-director of the Ludwig Center Vogelstein and team's genetic discoveries ushered in what is known as precision, or individualized, can.