Medical breakthrough NC | Discovery by a neurosurgeon at Duke University in North Carolina leads to the new brain tumor drug Voranigo

DURHAM, N.C. (WTVD) – It is a discovery that Dr. Darrell Bigner has never forgotten.

“This excites me more than almost anything else in my entire 50- to 60-year career working on brain tumors,” said Bigner, whose roles include chair of the Division of Experimental Pathology at Duke University's Preston Robert Tisch Brain Tumor Center.

In 2008, Dr. Bigner and a team of doctors at Johns Hopkins University began sequencing genes in brain tumor tissue that had been collected over decades.

“We found a so-called point mutation in this one gene that had never been previously suspected of being involved in cancer,” Bigner said of isocitrate dehydrogenase.

Based on this discovery, Duke and Johns Hopkins licensed a company to develop the treatment. This ultimately led to the development of vorasidenib, sold under the name Voranigo and manufactured by Servier Pharmaceuticals.

“About one-third of all primary brain tumor types can be treated with this new drug. This goal allows us to treat only those patients who are likely to respond. This way we know exactly who needs the drug and who will respond, rather than treating all patients and only hoping that some of them will respond,” explained Bigner.

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

“16 years ago, this didn't exist. It wasn't even thought of. Now we have this option and this treatment for me and other patients who are in a similar situation. That's just positive,” said Richmond, who was diagnosed in 2016.

Although she was doing well at the start of the study, IDH-mutated gliomas are incurable and Richmond was aware of the possibility of future disease progression.

“Like everything, it's a trial. It's an experiment. I'm so happy and pleasantly surprised,” Richmond said.

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

“These patients (in the clinical trial) had no signs of tumor progression over a long period of time. Next, we looked at a metric called 'time to next intervention,' and that was when they would need radiation, chemotherapy or more surgery. The wonderful thing for the patients who received the drug is that that time hasn't even been reached yet, and the trial started during the pandemic in 2020. We're still caring for all of these patients, and they're doing well,” said Dr. Katy Peters, a professor of neurology and neurosurgery at Duke who served as principal investigator during the clinical trials.

Voranigo, 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 afterward is definitely a benefit of this pill. I've been fortunate not to have any side effects,” Richmond said.

“This drug goes in and blocks the mutated protein. It specifically targets the cancer cell and actually makes the cancer shrink. It also avoids the need for other interventions such as radiation and chemotherapy,” Peters added.

While the therapy is currently used in patients with IDH-mutated grade 2 glioma, research is ongoing to determine whether it can be used in more patients.

“We are conducting a number of clinical trials in patients who may have more aggressive tumors or who have already tried other forms of therapy. At Duke, we are currently conducting four other clinical trials,” Peters said.

Bigner noted that clinical trials were also taking place in other countries, paving the way for approval in other countries.

According to the FDA, this 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, David Lee, CEO of Servier Pharmaceuticals, wrote:

“Gliomas are a unique type of cancer. Many of the patients I've met are in their 30s and 40s and in the prime of their lives. They have young children and are at the peak of their careers. We know these patients are in a constant state of anxiety because they don't know what the next scan will show. We've developed a therapy that we hope will allow these patients to live their lives, continue their careers and create memories with friends and family, rather than just waiting for their disease to progress.”

SEE ALSO | Advances in the treatment of metastatic cancer offer patients new hope

SEE ALSO | Alzheimer's research: Duke and UNC collaborate to achieve medical breakthroughs in women's health