John Theurer Cancer Center at Hackensack University Medical Center in Hackensack has treated the first patient in the country with an investigational new drug for relapsed glioblastoma and other anaplastic gliomas, according to a Monday announcement from the hospital.
Glioblastoma is one of the most aggressive forms of brain cancer, which often recurs despite standard treatments such as chemotherapy and radiation. Survival beyond five years is rare, and new treatments are gravely needed.
The new treatment, DSP-0390, has not been approved by the U.S. Food and Drug Administration, nor has its safety and efficacy been established. It is an investigational new agent developed by Cambridge, Massachusetts-based Sumitomo Dainippon Pharma Oncology that acts as an inhibitor of emopamil-binding protein, a molecule in cholesterol biosynthesis.
“Our bodies need cholesterol to build healthy cells and for those cells to signal one another,” said Dr. Samuel Goldlust, medical director of neuro-oncology. “But GBM cells can force the production of cholesterol into overdrive, leading to tumor growth and blocking EPB may inhibit this growth.”
This multicenter, global Phase 1/2 study is planned to enroll 70 patients at five brain tumor centers in the U.S. and Japan.
“Clinical trials in GBM have taught us that traditional treatments are not enough to defeat this challenging tumor,” said Dr. George Kaptain, director of neurosurgical oncology. “It is a privilege to be able to offer our patients streamlined access to novel, investigational molecularly targeted therapies like DSP-0390 in our ongoing pursuit of treatments to help maximize survival and minimize side effects.”