FDA gives patients hope after approving brain cancer drug with Duke ties

Patients with low-grade gliomas 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 Grade2 astrocytoma or oligodendroglioma.

It’s the culmination of 16 years of research and clinical trials involving Duke researchers.

"I have been working on malignant brain tumors for 50-60 years and it is extremely gratifying to finally have a breakthrough we know is going to help a significant number of patients," said Dr. Darell Bigner with The Preston Robert Tisch Brain Tumor Center at Duke.

Back in 2008, Bigner, in collaboration with a team at Johns Hopkins, discovered a mutated gene IDH was frequently found in brain cancers, like gliomas.

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

Peters, also with The Preston Robert Tisch Brain Tumor Center at Duke, 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 just really excited to be a part of it."

Peters said she believes this kind of research targeting specific kinds of tumors is becoming more common among cancer researchers.

"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 that can benefit from it," Peters said.

331 patients enrolled in the study. One of those patients was Rebecca Richmond.

The Durham mother told WRAL News her glioma diagnosis came at age 37.

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

Richmond said she was most scared of missing out on her now 11-year-old daughter’s future.

"When I was diagnosed, she was three 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 the drug therapy.

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

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

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

"I’m grateful for every day and blessed with every day and don’t take anything for granted," Richmond shared.