Promising Sylvester Glioma Study Highlighted at National Neuro-Oncology Conference

There could be new hope for people diagnosed with glioma. An enzyme inhibitor in development can control the cancer and reduce the tumor growth rate by as much as 50 percent, according to a Phase I clinical trial conducted by researchers at Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine and other institutions. The results are particularly striking because the 66 patients in the study failed to completely respond to previous treatments for this form of brain cancer.

The findings look so promising that the scientific planning committee for the Society of Neuro-Oncology Annual Meeting selected the study for presentation during the plenary session on the first morning of their annual meeting in November in San Francisco. The society also gave the study its Adult Clinical Research Award.

The inhibitor blocks the action of isocitrate dehydrogenase type 1 (IDH1), an enzyme present in a mutated form in gliomas and many other cancers. Left unchecked, IDH1 is believed to play a role in driving cancer growth.

“Since 2014, Sylvester has been part of many clinical trials with this drug, AG-120, a first-in-class mutant IDH1 inhibitor, and many other compounds for multiple tumor types including brain tumors, hematologic malignancies and cholangiocarcinomas,” said Macarena de la Fuente, M.D., study co-author from Sylvester and assistant professor of neurology at the University of Miami Miller School of Medicine.

“The drug is well tolerated and resulted in durable disease control and reduced the volumetric tumor growth rate in gliomas,” De La Fuente said.

The investigators reported about a 50 percent reduction on the volumetric tumor growth rate following treatment with AG-120 (Ivosidenib, Agios Pharmaceuticals). The agent was well tolerated and associated with a durable disease control response. In addition, progression free survival was approximately 13 months in the population of previously treated, primarily mostly grade 2 or grade 3 non-enhancing IDH1m glioma patients.

De La Fuente worked with researchers from Memorial Sloan-Kettering Cancer Center, Dana Farber Cancer Center, MD Anderson Cancer Center and other leading institutions on the Phase 1 safety and efficacy study.

Based on a previous trial of AG-120 at Sylvester, the FDA recently approved enasidenib (IDHIFA, Celgene Corp.) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia.

Going forward, De La Fuente and colleagues plan to further explore the role of the IDH1 inhibitor to help people living with gliomas.  

Tags: De La Fuente, glioma, neuro-oncology