Alliance formed to cure glioblastoma with personalized medicine

A new-generation clinical trial for glioblastoma multiforme (GBM) — the deadliest form of brain cancer – will begin enrolling patients by mid-year 2016. The trial is designed to identify effective treatments faster for this aggressive form of brain cancer which kills half of all patients diagnosed within one year. Five years after diagnosis, the survival rate is less than 2 percent.

The international trial is adaptive, meaning researchers will modify the trial as it proceeds based on how each patient responds to the many single drugs and drug combinations that will be tested.

GBM AGILE (Adaptive Global Innovative Learning Environment), as the trial is named, “will employ advanced statistical tools to ensure than better treatments can be assigned to more patients and ineffective treatments can be eliminated from the trial,” said Donald Berry, Ph.D., professor of biostatistics at MD Anderson and the trial’s co-principal investigator.

Although literally hundreds of clinical trials have tested numerous therapies for GBM, treatment options and patient outcomes have not changed for several decades. The most notable advance occurred more than a decade ago when a drug called temozolomide was tested with radiation therapy, and reported to extend life by about two months.

In addition to this sparse record of clinical trials for GBM, there are virtually no biomarkers — telltale proteins revealing the genetic makeup of each person’s tumor — that can be used to tailor drugs for each individual patient. As a result, GBM patients have not benefited from personalized medicine.

GBM AGILE will use molecular biomarkers to assign specific patients to arms of the trial that are most likely to work for them.

“Thanks to the deep molecular characterization of GBM, we are beginning to get a better picture of the genes and pathways that are altered in GBM, so there is finally an opportunity to identify real biomarkers and conduct a “smart” trial like GBM AGILE,” said Alfred Yung, M.D., chairman of Neuro-Oncology and executive committee member of the trial, which will take place across the United States, Europe, China and Australia.

This international collaboration will enable recruiting sufficient numbers of patients to learn which therapies do or do not work more quickly than in the past.

GBM AGILE is being developed through the National Biomarker Development Alliance, a nonprofit organization created as part of the Research Collaboratory at Arizona State University. The alliance’s mission is to move biomarkers through all phases of discovery, development and validation through clinical trials to ensure personalized medicine will be available to all patients.