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Drug breakthrough for brain tumour that strikes young people



A new drug could transform how doctors treat a brain tumour that typically strikes younger people, a leading US cancer experts has said.

David Schiff, MD, co-director of the University of Virginia Cancer Center’s Neuro-Oncology Center, has authored an editorial in the New England Journal of Medicine describing the potential significance of the drug vorasidenib for patients with tumours known as “grade 2 IDH-mutant gliomas.”

The drug, when tested in the INDIGO clinical trial, was found to slow tumour growth significantly and extended the average time until the tumour started growing from 11.1 months to more than 27 months.

If the drug receives approval from the FDA, it would become the first targeted therapy for low-grade gliomas.

However, Schiff notes that there are also other recent advances that are improving our understanding of such gliomas.

Schiff said: “It used to be that we thought of all gliomas as being on a spectrum.

“We now understand that those with the IDH gene mutation have a markedly different biology, outcome and, as this study shows, vulnerabilities that new therapies can exploit.”

Approximately 2,500 Americans with a median age of just 40 are diagnosed with grade 2 IDH-mutant gliomas each year.

The tumours often affect the patients’ ability to think and hold a job, as well as interfere with other aspects of daily life.

Eventually the tumours become resistant to treatment options and typically prove fatal.

Because of the limited treatment options available, doctors usually take a “watch and wait” approach to managing the gliomas, holding off on treatment until after the tumour progresses.

But vorasidenib could change that, Schiff said, and the drug could offer the first early treatment for the cancer, giving patients an important new option that could extend their lives.

In the INDIGO trial, more than 300 patients were randomised to receive vorasidenib or a harmless placebo.

Neither the patients nor their doctors knew which the participants were receiving.

Schiff, in a New England Journal of Medicine editorial, described the results as “striking.”

Not only did the patients receiving vorasidenib live longer, but they did not need more toxic treatments, such as radiation and chemotherapy, as quickly as the patients who received placebos.

Schiff was so impressed by the success of the drug that he wrote that vorasidenib could “put a nail in the coffin” of the watch-and-wait approach.

Schiff said:

“There are still many unanswered questions about how we can best utilize this new medication if and when it receives FDA approval.

“Nonetheless, considering that existing standard therapies for these tumours [radiation and chemotherapy] are tough on patients, with short- and long-term side effects, it will be wonderful to have a useful and very well-tolerated treatment option.”

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