Scientists to launch multinational precision medicine trial for deadliest brain tumor

Researchers from the U.S., China, Australia and Europe on Thursday announced plans to launch a multinational precision medicine trial for glioblastoma multiforme (GBM), the most common and deadliest form of brain tumors in adults.

The trial, called GBM Agile, will test multiple treatments based on the "molecular profile" of a patient's tumor and treatments that do not work will be dropped from the trial to allow patients to start on other agents or combinations rapidly.

Scientists believed the molecularly targeted approach known as "precision medicine" can help speed up discovery of effective therapies for specific GBM patients.

"Patients are treated on the basis of their molecular biomarkers (today), but GBM patients are not benefited from this," Anna Barker, project director and executive committee (EC) chair of GBM AGILE and professor at Arizona State University's School of Life Sciences, said at a news conference in Washington, D.C. "We need a big idea."

Each year, over 12,000 people in the U.S. and tens of thousands more around the globe, including 35,000 in China alone, were diagnosed with GBM.

A devastating reality is that 50 percent of GBM patients will survive for a year or less and five-year survival for GBM is less than two percent.

Although hundreds of clinical trials have tested numerous therapies for GBM, treatment options and patient outcomes have not changed for several decades.

Notably, the single advance in the treatment of GBM occurred over a decade ago when a drug called temozolomide was tested with radiation therapy in a phase three trial and reported to extend life by about two months.

Calling the situation "bloody unacceptable," Barker said: "It is in that spirit, that this broad 'coalition of the willing' today announce the design and plan for a new-generation clinical trial for GBM."

The trial will begin enrolling patients by mid-2016. It involved over 130 neurosurgeons, neuro-oncologists, pathologists, imagers and neuroscientists from the U.S., China, Australia and Europe.

"The Chinese neurosurgery communities look forward to implementing GBM AGILE in China and to working with the GBM Global team," Tao Jiang, GBM AGILE EC China Liaison, Vice Director of Beijing Neurosurgical Institute and Director and Founder of Chinese Glioma Genome Atlas (CGGA), said in a statement.

"We finally have the opportunity to work together across borders to learn from every patient and identify better therapies for GBM patients no matter where they are on the Globe." Enditem