NC Fast Facts: FDA approves breakthrough brain cancer drug linked to Duke trial

DURHAM — The Preston Robert Tisch Brain Tumor Center at Duke recently announced patients with low-grade glioma brain tumors, a deadly form of brain cancer, now have a new treatment option for the first time in decades. The FDA approved vorasidenib earlier this month for adult and pediatric patients 12 and older with Grade 2 astrocytoma or oligodendroglioma.

“I have been working on malignant brain tumors for 50 to 60 years, and it is extremely gratifying to finally have a breakthrough we know is going to help a significant number of patients,” Dr. Darell Bigner with Duke’s Preston Robert Tisch Brain Tumor Center told WRAL.

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In 2008, Bigner and a team from Johns Hopkins discovered that the mutated gene IDH was frequently found in brain cancers, such as gliomas.

“This IDH mutant protein, it produces essentially a chemical that causes tumor cells to grow. What the drug does is it inhibits that mutant protein,” said Dr. Katy Peters.

Peters, also with the Preston Robert Tisch Brain Tumor Center, led the largest clinical trial for the drug through Duke.

“All the way to discovery at Duke, to translation at Duke, and finally now treatment at Duke, it’s very exciting,” Peters said. “This is a rare scenario, and I’m excited to be a part of it.

“To have all of these wonderful discoveries at Duke is pretty exciting, and I think it’s wonderful that we have so many patients and caregivers who can benefit from it,” said Peters.

Three hundred thirty-one patients enrolled in the study. One of those patients was Rebecca Richmond.

According to an interview with WRAL News, the Durham mother said her glioma diagnosis came at age 37.

“It’s terrifying. I had a young family so a lot of things go through your mind of what does our future hold?” she said.

Richmond said she feared missing out on her 11-year-old daughter’s future.

“When I was diagnosed, she was 3 and a lot of it goes through your mind of the things you could potentially miss and the things you want to prepare her for that you may not be around to do so,” the mom shared.

Richmond is now starting her 49th cycle of drug therapy.

“It seems to be working great. I’m able to function, carry out work as normal and activities of daily living completely normal,” she said.

Peters explained that the drug was shown in trials to slow the growth of low-grade tumors, buying patients like Richmond time.

It also provided patients and physicians with another treatment option besides chemotherapy and radiation.