Hollings neuro-oncologists celebrate new drug approval

Neuro-oncologists at MUSC Hollings Cancer Center are thrilled with the news that the U.S. Food and Drug Administration has approved a new drug to treat certain types of gliomas.

“This kind of breakthrough hasn’t happened in decades,” said Alicia Zukas, M.D. “This is a targeted therapy and a first of its kind for brain tumors.”

Further, Hollings contributed to the research that led to this approval. Hollings was the only trial site in South Carolina during the INDIGO trial, an international phase 3 clinical trial that showed that the drug vorasidenib significantly lengthened the amount of time that people lived without the cancer progressing.

The FDA approval, announced on Aug. 6, shows how critically important clinical trials are, said Scott Lindhorst, M.D., the MUSC site investigator for the INDIGO trial.

 

“The pursuit of new treatments through clinical trials is the lifeblood of cancer research at National Cancer Institute-designated cancer centers like Hollings Cancer Center. Patients who participate in clinical trials are true heroes, helping to move the field forward in tangible ways, benefiting multitudes of patients beyond themselves,” he said.

“At the same time, trial participation is a two-way street, offering patients the opportunity to gain access to new, innovative treatments they otherwise might not be able to receive. We are extremely grateful to all our patients who elect to pursue treatment in trials within our institution. The INDIGO trial is a prime example of bench-to-bedside research yielding substantial benefits for patients.”

Another benefit of clinical trial participation, Zukas noted, is that the care teams at Hollings gain experience with the ins and outs of a specific medication before it hits the market. They’re ready to work with the medication as soon as it’s approved rather than needing to learn the drug and its protocols.

In fact, Zukas has already written her first prescription.

Coastal South Carolina was hunkered down for Tropical Storm Debby, but this drug approval was bigger news to one of her patients, who asked about it during a telehealth appointment during the storm.

“I love that technology has evolved to let me do a doctor's appointment from my house during a hurricane and that social media can inform doctors and patients at virtually the same time,” Zukas said.

New treatment for gliomas

Gliomas are the most common type of malignant brain tumor diagnosed in adults under age 50. Yet not all gliomas are the same. “Glioma” describes tumors that begin with the glial cells, a catchall term for all the cells in the central nervous system that aren’t neurons. Glial cells support, clean up and insulate neurons.

Glial cells include astrocytes and oligodendrocytes, and gliomas include astrocytoma, oligodendroglioma and glioblastoma.

Vorasidenib, the newly approved drug, targets Grade 2 astrocytomas and oligodendrogliomas that have an IDH1 or IDH2 mutation, which is common for these two types of gliomas. A press release from Servier, the company that developed vorasidenib, explained that a family of genes code for enzymes called isocitrate dehydrogenases (IDH) that break down nutrients and generate energy for cells. But mutations in IDH1 and IDH2 genes prevent cells from properly maturing into specialized roles; instead, they just start growing out of control.

The existing treatment regimen would start with surgery to remove the tumor. If the patient had no high-risk factors for their tumor, surgery would be followed by a “watch and wait” period to monitor for any potential recurrence of the tumor. If the cancer reappears, then it would be treated with radiation and chemotherapy. Those are harsh treatments, though, Lindhorst said, which is why doctors may delay utilizing them.

“In the past few years, neuro-oncology research has started producing exciting new advancements for patients, and the team at Hollings is ready to bring those advancements to the people of South Carolina.”

Scott Lindhorst, M.D.

Targeted therapies are developed to home in on particular proteins or structures that exist only on the cancer cells, unlike older therapies that are less discriminating and end up hurting healthy cells as well as cancerous cells. Because of this, targeted therapies usually have fewer side effects, and this was true for vorasidenib as well.

Vorasidenib was given to patients in that “watch and wait” period to see if it would prolong the amount of time before the tumor reappeared. It did, giving patients who got the drug twice as long without cancer progression as the patients who got a placebo.

The results were so promising that the study was “unblinded” so that the participants who were getting the placebo would have the opportunity to switch to the actual drug.

“We try to balance quality of life with extension of life,” Lindhorst explained. “The hope is that by having a targeted intervention like vorasidenib, that's much more tolerable, we can avoid other things like radiation and traditional chemotherapy for many, many years and help people live normal lives not thinking about their brain tumor every day.”

“It’s gratifying to play a role in research like this that makes a substantial difference in people’s lives,” he continued. “In the past few years, neuro-oncology research has started producing exciting new advancements for patients, and the team at Hollings is ready to bring those advancements to the people of South Carolina.”

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