An 83-year-old Duke professor's breakthrough research leads to new brain cancer drug

Brain cancer research by a Duke scientist who's spent more than six decades at the university helped a drug receive FDA approval this week.

Why it matters: The approval is the first major advancement in low-grade brain cancer treatment in more than two decades, according to the news outlet Fierce Pharma .

Driving the news: The drug, called Voranigo, is the result of collaborative research by 83-year-old Duke physician-scientist Darell Bigner and Bert Vogelstein of Johns Hopkins.

• Voranigo is the seventh drug now on the market that has roots in Duke intellectual property, according to Robin Rasor, the school's associate vice president of translation and commercialization.
• The pharma company Servier sponsored the clinical trials for the drug and will bring it to market.

What it does: The treatment is taken orally and targets a type of brain cancer referred to as a low-grade IDH-mutant glioma.

• The treatment doesn't cure the cancer but works by halting the activity of the mutated gene and, in turn, slowing the growth of the tumor.
• In a phase 3 trial of more than 300 participants, the drug was shown to significantly improve how long a patient could live without the tumor growing, according to the New England Journal of Medicine .
• It also significantly delayed time before the tumor required additional treatment, like surgery or chemotherapy.

Between the lines: Katherine Peters, a neuro-oncologist who helped run the clinical trial for the drug at Duke, said they are still studying these patients over the long term.

• The clinical trial began in 2020 and many participants are continuing to see the drug prevent tumor growth, she said, adding it raises interesting questions about whether patients will stay on Voranigo for 10 to 15 years.
• Those still on the drug "have just been doing quite well compared to patients that were on the placebo," she said.

What they're saying: "For this group of patients, we've never really had anything very good," Bigner, who has been at Duke since 1963, told Axios, "and this is going to be a real game changer."

What's next: Research from Bigner's lab continues to be used in trials for other cancers with the IDH mutation, and he is hopeful the efforts will lead to breakthroughs similar to Voranigo.

• "I hope I can be around another five years to complete those," he said.

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