China joins fight against brain cancer

It's the deadliest and most aggressive form of brain tumors and affects 35,000 people in China and 12,000 in the United States, and one with no cure. Now scientists and doctors from China, the US, Australia, and Europe are teaming up for global clinical trials in search of therapies to help patients diagnosed with glioblastoma multiforme (GBM).

Fifty percent of people diagnosed with GBM, which is most common among adults, are expected to die within a year; the five-year survival rate is less than 2 percent.

GBM AGILE is the name of the global alliance of 150 neurosurgeons, neuro-oncologists, clinical investigators from 40 leading cancer institutions that will embark on new adaptive trials to find an effective treatment for GBM patients. Adaptive trials, compared to standard clinical trials, can be moderated to incorporate the newest information available, and allows researchers and doctors to test multiple treatments and combinations of treatments in parallel.

"We really don't have an affective drug at all for GBM," said Dr Anna Barker, GBM AGILE project director and former director of the National Cancer Institute in Rockville, Maryland.

"We're looking for effective agents. It could be a biologic, it could be a drug like we think normally of drugs, or it could be some combination. The beauty of the trial also is we can test combinations of drugs and biologics, biologics by themselves, and drugs by themselves," she told China Daily. Biologics are man-made proteins that are derived from human genes.

There are no biomarkers for the disease - a measurable unit whose presence indicates disease or infection - which makes it hard to anticipate when a person could potentially be affected, Barker said.

The trials, expected to begin by the spring, will take place concurrently at 100 sites around the world, Barker said.

The number of affected patients in China is about three times the size of those in the US, which is why China is involved in the trial and is "critical" to its success, said Dr. Sujuan Ba, president of the National Foundation for Cancer Research and executive committee member of GBM AGILE.

"The percentage (of patients) is relatively the same, but it's the sheer number of GBM patients in China that's an incredible resource and critical factor for the GBM AGILE success," she said.

A network of experts will make up 10 committees that donate their time and pay for travel costs to design and plan the trials, and include members from the National Foundation for Cancer Research, the National Brain Tumor Society, Accelerate Brain Cancer Cure, and Cure Brain Cancer Foundation.

"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 Dr. Alfred Yung, professor in the department of neuro-oncology at the University of Texas M.D. Anderson Cancer Center, and GBM AGILE executive committee member.

There will be more than 70 doctors and experts involved in the US, more than 30 from China, and the remaining will be from Europe and Australia.

"Not only is the disease expensive, there is simply very little hope. The negative impact on patients is actually worse than other cancers, because it hits the central nerve system. Patients' condition is not only worsened by the cancer, but it's also degradation of their brain capabilities, so it's more deadly and more debilitating," she said.

amyhe@chinadailyusa.com