New brain tumor drug hits market 16 years after Duke neurosurgeon's discovery: ' Excited'

DURHAM, N.C. (WTVD) -- It's a discovery Dr. Darrell Bigner has never forgotten.

"I'm more excited about this than virtually anything in my entire 50-to-60-year career working on brain tumors," said Bigner, who amongst several titles serves as the Chief of the Division of Experimental Pathology at The Preston Robert Tisch Brain Tumor Center at Duke.

In 2008, Dr. Bigner was working alongside a team of doctors from Johns Hopkins University began sequencing genes in brain tumor tissues that had been collected over decades.

"What we found was what's called a point mutation in this one gene that was never suspected to be involved in cancer before," said Bigner, referring to isocitrate dehydrogenase.

Based on the discovery, Duke and Johns Hopkins provided a license allowing a company to develop the treatment. Ultimately, it led to the creation of vorasidenib, marketed as Voranigo, made by Servier Pharmaceuticals.

"About one-third of the primary brain tumor types can be treated with this new drug. By having a target like this, only the patients that are likely to respond are treated. It enables us to know exactly who needs the drug and who will respond to it instead of treating all patients and just hoping that some of them will respond," Bigner explained.

In 2020, Duke hosted clinical trials for the drug, which Rebecca Richmond participated in.

"Sixteen years ago, it didn't exist. It wasn't even a thought. So now that we have this opportunity and this treatment for myself and other patients that are in a similar situation, I mean, it's nothing but positive," said Richmond, who was diagnosed in 2016.

While she was feeling well when the trial started, IDH-mutant gliomas are incurable, and Richmond was aware of possible future progression.

"With anything, it's a trial. It's an experiment. So happily and pleasantly surprised," Richmond shared.

Over the past four years, her MRIs have remained stable.

"Those patients (in the clinical trial) didn't have any evidence of any tumor progression for an extended period of time. The next (point) we looked at a measure called Time to Next Intervention, and that was the time when they would need radiation, chemo or more surgery. What's wonderful for the patients that received the drug is that hasn't even been met yet, and the trial started during the pandemic in 2020. We're still following all those patients and they're doing great," said Dr. Katy Peters, a Professor of Neurology and Neurosurgery at Duke who served as a principal investigator during the clinical trials.

Voranigo, which was approved by the FDA earlier this month, is an oral pill taken once daily.

"Not (being) tied to an infusion center where you're sick for days and days afterward, that's definitely a benefit of this pill. I've been fortunate not to have any side effects," said Richmond.

"What this drug does is it goes in and blocks that mutant protein. It specifically targets the cancer cell and then actually makes the cancer get smaller and also prevents them from having to use other types of interventions like radiation and chemotherapy," added Peters.

While it's currently for patients with a Grade 2 IDH-mutant glioma, there's ongoing research on whether it can be used on more patients.

"We're doing a series of clinical trials looking at patients that are having maybe more aggressive tumors or patients that have seen other types of therapies. We now have four additional clinical trials at Duke," Peters shared.

Bigner noted clinical trials also took place in other countries, clearing the pathway for approval elsewhere.

According to the FDA, it is the first approval of "a systemic therapy for patients with Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation."

In a statement to ABC 11, Servier Pharmaceuticals CEO David Lee wrote:

"Glioma is a unique cancer. Many of the patients I've met are in their 30s and 40s and in the prime of their lives. They have small children and are at the height of their careers. We know these patients are in a constant state of anxiety not knowing what the next scan will show. We've created a therapy that we hope allows these patients to live their life, pursue their careers, make memories with friends and family, instead of just waiting for their disease to progress."

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