A promising new drug for brain cancer, approved in August by the U.S. Food and Drug Administration, 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, called IDH-mutant low-grade glioma. 

The gene was identified by oncologist Bert Vogelstein 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,” says Vogelstein, co-director of the Ludwig Center. “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.”