New brain tumor trials are on the fast track

Published: Nov. 8, 2018 at 4:30 PM EST
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In the last 30 years, a brain tumor expert says median survival has only increased by a few months. Now, a new brain tumor center program is fast-tracking patients into new therapies.

U.S. Sen. John McCain from Arizona recently died after fighting glioblastoma, the most aggressive and deadliest kind of brain tumor. Now, other patients whose brain tumors come back have a revolutionary new option for treatment.

Leslie Casillas recently found out her glioblastoma is back. She doesn’t have symptoms and feels good.

“Sometimes, you know, I’m in the shower, I’ll think, 'Oh, I have a brain tumor. I have cancer.' And it’ll kind of hit me for a second. And then I just kind of go about my day,” Leslie said.

She’s waiting to see if she gets in to a Phase 0 trial at the new Ivy Brain Tumor Center in Phoenix.

Dr. Nader Sanai says Phase 0 trials shorten testing of new drug therapies from years to months, since the drugs are already approved for other conditions. Doctors give patients small amounts of drug combinations they believe are a good genetic match for the tumor, then surgeons remove new growth.

“In doing so, we can take the tumor that we remove in the operation, test it, and answer two basic questions: did the drug get through and did the drug do what it’s supposed to do?” Sanai said

Doctors know if there’s a drug response within seven to 10 days.

“If they’re on the study and they actually graduate to the therapeutic dosing after surgery, they’re really doing so knowing that there’s some hard evidence connecting their tumor’s response to the drug,” Sanai said.

“That was exciting to me, to have the chance to take something that I know might actually work and have proof that it’s working,” Leslie said.

Sanai says three trials for about 50 patients are complete. In two of them, doctors identified drugs that had an effect and will be studied further. The third didn’t work, but he says researchers still got good information by understanding how it failed.

New trials are open, and you can get more information at




REPORT: MB #4497

BACKGROUND: Glioblastomas (also called GBM) are malignant Grade IV tumors, where a large portion of tumor cells are reproducing and dividing at any given time. They are nourished by an ample and abnormal tumor vessel blood supply. The tumor is predominantly made up of abnormal astrocytic cells, but also contain a mix of different cell types, including blood vessels, and areas of dead cells. Glioblastomas invade into nearby regions of the brain. They can also sometimes spread to the opposite side of the brain through connection fibers. It is rare for glioblastomas to spread outside of the brain.


PHASE 0 TRIAL: In a first-of-its-kind clinical trial, a drug called AZD1775 was shown to penetrate deadly glioblastoma brain cancer cells, according to a study conducted at Barrow Neurological Institute and initiated by the Translational Genomics Research Institute (TGen). The study of 20 patients at Barrow was published in the scientific journal Clinical Cancer Research, a journal of the American Association for Cancer Research (AACR). This so-called “Phase 0” clinical trial showed that AZD1775 could penetrate the blood-brain-barrier that protects the brain from toxins but also blocks most anti-cancer drugs. “This Phase 0 approach for malignant brain tumors is a game changer in cancer research and our program is the first of its kind in the world,” said Nader Sanai, M.D., the study’s lead author, a brain tumor surgeon at Barrow Neurological Institute who also directs Barrow’s Brain Tumor Research Center, and co-Director of the Brain Tumor Early-Phase Clinical Trials Unit at TGen. “This study confirms the utility of Phase 0 trials as part of an accelerated paradigm for drug development in glioma patients.”


NEW RESEARCH: Each of the study’s 20 patients had previously received the current FDA-approved standard of care, including surgery to remove their brain tumors, followed by radiation treatment and TMZ. Each patient’s cancer had returned. Each was then given varying doses of AZD1775 at various times just prior to operations to remove tumors that had grown back. “The hope is that these drugs — AZD1775 and TMZ — could work in tandem to exploit a particular genetic vulnerability discovered by TGen in certain glioblastoma tumors,” said Michael Berens, Ph.D., a TGen Deputy Director, a co-Director of TGen’s Early-Phase Clinical Trials Unit, and one of the study’s authors.


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