Clinical Trial Hopes to Shed New Light on Glioblastoma

An international group has joined together to advance an innovative clinical trial that hopes to improve the survival odds for glioblastoma, an aggressive type of adult brain cancer. Tumors grow quickly, causing difficult symptoms like headaches, nausea, and seizures. Care for glioblastoma is complex, typically involving a team that includes experts across neurosurgery, neuro-oncology, neuroradiology, neuropathology and radiation oncology—a level of care that is usually only available in major medical centers. Unfortunately, the average survival for this type of cancer is only 12–18 months.

The study, called GBM AGILE, or Glioblastoma Adaptive Global Innovative Learning Environment (NCT03970447), sponsored by the Global Coalition for Adaptive Research, a nonprofit organization, is using an approach that offers study participants multiple new glioblastoma drugs from different pharmaceutical companies. New treatments are rapidly assessed in real time against a standard-of-care control group using sophisticated statistical analyses. As new data become available on the efficacy of the drugs from the worldwide patient group, the randomization of the patients on the trial is adjusted to increase assignment of patients to drugs that are more effective. 

Huntsman Cancer Institute at the University of Utah (U of U) is one of the select sites in the United States to offer the GBM AGILE. A recent press release announced an extension of this study to include two new drugs and enrollment in Europe and China. Howard Colman, MD, PhD, a physician-scientist at Huntsman Cancer Institute, professor of neurosurgery at the U of U, and U.S. regional principal investigator of GBM AGILE, will lead one of the new arms of the study. This arm will evaluate a drug called VT1021, manufactured by Vigeo Therapeutics, in both newly diagnosed and recurrent GBM. Currently, GBM AGILE is evaluating several treatment combinations and constantly assessing data to determine which combination is best for each unique patient.

“This study is unique in that it allows for more rapid evaluation of drugs being studied to see if they are effective compared to more traditional clinical trial designs,” says Colman. “The hope is that by having multiple drugs evaluated simultaneously worldwide in a large international trial, we will be able to rapidly advance the cutting-edge drugs that are performing best in this study to patients who need them.”

At any given time, Huntsman Cancer Institute offers more than 250 clinical trials to cancer patients. Clinical trials work to improve the standard of care for treatment and quality of life among cancer patients. All clinical trials require rigorous monitoring to gather data needed to validate the study. Trial ideas are studied extensively before being offered to people to ensure they are safe. In addition to GBM AGILE, Huntsman Cancer Institute’s neuro-oncology disease center offers an array of trials for people with brain tumors and people whose cancers have spread to the brain.

“For glioblastoma, we have not seen the same type of treatment advances as quickly as we have for other types of cancer,” says Colman. “The GBM AGILE trial is one example of a study designed to be really efficient in showing if a drug is effective or not. This type of approach is critically important for people with glioblastoma.”

This research is supported at Huntsman Cancer Institute by the National Institutes of Health/National Cancer Institute, including P30 CA042014 and Huntsman Cancer Foundation. GBM AGILE is funded through grants by the National Brain Tumor Society, National Foundation for Cancer Research, Asian Fund for Cancer Research, and direct funding from pharma/biotech companies involved in the trial. Colman co-leads the evaluation of VT1021 in GBM AGILE with Tom Mikkelsen, MD, medical director at Henry Ford Precision Medicine Program and Clinical Trials.

Find more information about GBM AGILE.