The dismal survival statistics surrounding glioblastoma multiforme have gone unchanged for years, with 50% of patients surviving for 1 year or less. Now, a global coalition of health care providers, clinical investigators, and advocates are coming together to design and plan a new type of clinical trial to help identify successful therapies.
The project, called GBM AGILE (Adaptive Global Innovative Learning Environment), is designed to be adaptive in order to streamline the drug and biomarker discovery process.
“This will be a Silicon Valley approach to medical research,” said Mustafa Khasraw, MD, a senior research fellow at the University of Sydney’s Clinical Trials Centre. “We want to fail early and fail often so we can rapidly discriminate between effective and ineffective therapies.”
The trial, which is backed by Arizona State University’s National Biomarker Development Alliance (NBDA), will be driven by Bayesian statistics and molecular markers in order to allow individual patient outcomes to inform the ongoing conduct of the trial. The trial will also utilize a “master protocol” designed by the U.S. FDA in order to simplify the trial process.
The ultimate goal is to increase the number of single agents, drug combinations, and biologics tested in an ethnically-diverse group of patients in order to help successful agents progress more rapidly to phase 3 trials. The trial design will also allow researchers to qualify and validate biomarkers observed in individual patients.
The coalition currently includes over 130 clinicians, researchers, and representatives from the U.S., Australia, Europe, and China, and plans to begin enrolling patients in 2016.
“The adaptive design will allow us to modify the trial as it proceeds based on the data collected — and to test many drugs and combinations versus single agents — and to do it faster,” said Timothy Cloughesy, MD, GBM AGILE Principal Investigator and Director, UCLA Neuro-Oncology Program. “It’s an opportunity for patients to benefit from precision medicine, and a real source of hope for patients and their families.”