A new drug was approved by the U.S. Food and Drug Administration this month for a type of brain cancer called IDH-mutant low-grade glioma, a promising treatment that grew out of a genetic discovery 16 years ago at the Johns Hopkins Cancer Center.
The drug, called vorasidenib, is a targeted cancer treatment that slows cancer growth by blocking the activity of a mutated gene called IDH.
The gene was identified in 2008 by Dr. Bert Vogelstein, whose team at Hopkins created the first genetic blueprint of brain tumors, which evaluated all known protein-coding genes in brain tumors and is believed to be the most comprehensive genetic analysis of any tumor type.
The researchers found that the IDH gene, which had not previously been suspected to be involved in any tumor type, is frequently mutated in a subset of brain tumors.
Treatment typically involves surgery to remove as much of the tumor as possible, followed by radiation therapy and chemotherapy to attack any remaining cancer cells, but in some patients, the addition of an IDH inhibitor can delay the need for radiation therapy and chemotherapy.
“The possibility of delaying radiation and chemotherapy with this drug may be beneficial for certain patients with slowly progressing IDH-mutated gliomas,” said Matthias Holdhoff, MD, PhD, co-director of the Brain Tumor Program at the Johns Hopkins Kimmel Cancer Center and a collaborator on the 2023 clinical trial.
“We believe we are looking at a new standard of care option for these types of tumors.”
Vogelstein also invented: A new anti-cancer drug is so effective against tumors that the FDA quickly approved it.
Survey results Published last year Results of a phase 3 clinical trial of vorasidenib published in the New England Journal of Medicine concluded that 331 patients with grade 2 IDH-mutated glioma who received the drug experienced a significant improvement in progression-free survival and that the treatment delayed the time to next intervention (compared to patients who received a placebo).
Precision medicine for cancer
Vogelstein and his team’s genetic discoveries pioneered so-called precision cancer medicine, treatments that target the specific genetic makeup of each patient’s cancer.
Not only has this research led to the development of new FDA-approved drugs, but the discovery of the IDH gene has led to a new classification of gliomas, allowing us to distinguish cancers that have IDH mutations but have better overall outcomes and responses to treatment from highly aggressive gliomas that do not have IDH mutations, such as glioblastoma, the most common primary brain tumor in adults.
According to the National Cancer Institute, approximately 80% of low-grade gliomas contain IDH mutations. These include IDH-mutated astrocytomas and oligodendrogliomas, which occur most commonly in young adults. Low-grade gliomas tend to progress more slowly and have a longer survival time than aggressive high-grade gliomas.
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“The IDH is a prime example of cancer genome sequencing and demonstrates the importance of basic research.” Vogelstein says:Creighton Professor of Oncology, Howard Hughes Medical Institute Investigator and Co-Director of the Ludwig Center.
“The history of medicine shows that once diseases are understood, they ultimately become treatable. Though it may not be immediately obvious, over time, as in this case, such discoveries lead to better treatments for patients.”
It also paves the way for additional research into other types of brain tumors.
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Johns Hopkins University holds patents related to the discovery of IDH, which have been licensed by Servier Laboratories, which also funded the Phase 3 trial. As a result of this license agreement, the university and its inventors, including Dr. Bert Vogelstein, are entitled to receive royalties related to the discovery of IDH.
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