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Potential Glioblastoma Treatments Entering The Pivotal Phase of Evaluation

Jump Ahead to List of Trials

Glioblastoma (GBM) is the most common and aggressive malignant brain tumor and, regrettably, very limited treatment options exist for these patients. The current standard-of-care for newly diagnosed patients is surgery, followed by radiation and chemotherapy (with temozolomide), and, in some cases, use of the tumor-treating-field device, Optune. If and when tumors relapse – which, unfortunately, almost all GBMs do – even fewer options exist. Furthermore, the effectiveness of these treatments is marginal, and average survival for GBM patients remains little-changed in decades, ranging from 12-18 months, with only about 5% of patients living five-years or more. Glioblastoma is therefore estimated to account for at least 50% of all brain tumor-related deaths, annually.

SONY DSCConsequently, GBM represents a major unmet challenge for the neuro-oncology field and brain tumor community, and, indeed, across all of medicine. Thus, this tumor type is accordingly a substantial focus of many research organizations’ brain tumor efforts, including ours at the National Brain Tumor Society, where our current flagship research program, the Defeat GBM Research Collaborative, is aiming to double the percentage of patients who survive five years or more beyond diagnosis.

In addition to posing an urgent medical need given their devastating nature, glioblastoma also offers the biomedical research field, ultimately, with a potential glide-path for reciprocal discovery. That is to say, because of their extreme complexity, as well as ability to adapt and resist virtually all treatments that have been deployed to try and treat them, the theory goes that if researchers can unlock the mystery of GBM treatment, the field may just find the key to treating a number of other less-aggressive and complex brain and additional solid tumors.

To wit, the extensive amount of glioblastoma research funded by NBTS (both before, and now with, the Defeat GBM program), the National Institutes of Health, and a number of other organizations has led many leading scientists to suggest that GBM is actually, ‘the most genomically characterized tumor’ in all of cancer. In fact, many studies have been successful in determining much of the underlying machinery of these tumors – how and why they grow so fast, their strengths and weaknesses, and potential targets for treatment. But, again, historically there has been maddeningly little success in translating this really exceptional scientific research into actual treatments that can reach the cancerous cells these tumors seed throughout the brain with enough punch to halt their spread and prevent them from employing their dirty tricks to circumvent the attack.

STK_AnalyzingBrainScanAs such, for years clinical trials in GBM were marked by many heartbreaking disappointments as potential new therapies dropped-off; determined to be ineffective before ever even reaching the pivotal last stage of the clinical research and evaluation process – large Phase III confirmatory studies.

But times have changed.

The dedication, hard work, and commitment of the brain tumor community is beginning to offer real hope and promises of new glioblastoma treatment options available in the near-future.

“Just the fact that we have some phase 3 trials in glioblastoma, where for years we had a hard time getting past phase II trials, is an encouraging sign,” said Michael Lim, MD, director of the brain tumor immunotherapy program at Johns Hopkins Kimmel Comprehensive Cancer Center, in a blog post on the National Cancer Institute’s website. “For the first time in a long time, there’s some real excitement [in the field].”

We anticipate that we’ll see more advanced data, and possibly even results, from a number of these phase III trials for GBM patients later in 2017 and the first half of 2018.

So what are the scope and nature of these trials? NBTS’ Research Department conducted a review to identify all of the phase III clinical trials for GBM patients that have been registered with regulators, including some active; some still recruiting and enrolling patients; while others are waiting to begin enrolling patients. Excitingly, as Dr. Lim alluded to, many of these are trials of a “novel investigational agent” – meaning it is the first time a particular treatment is being tried for GBM patients. There are also a number of registered phase III trials with therapies already in use for some brain tumor patients – meaning researchers are evaluating these already-in-use treatments in different combinations, different dosing regimens, or subpopulation of patients.

What follows below is a breakdown/overview of the current GBM phase III trial landscape, followed by the full-list of trials with hyperlinks.

Phase III GBM Clinical Trials Overview

  • There are ten (10) trials with “novel” treatments currently in phase III
    • Three (4) are already active;
    • Three (3) are still recruiting; and
    • Three (3) haven’t started recruiting yet
  • Nine (9) of the ten trials with “novel” treatments have an endpoint (or goal/measure) of increasing “overall survival”
    • Five (5) are immunotherapies, or treatments designed to harness the body’s own immune system to fight the tumor
    • One (1) is a “Gene Therapy
    • Thee (3) are “targeted therapies,” or treatments designed to attack specific abnormality in the tumor (this is the concept of “precision” or “personalized” medicine).
    • One (1) is for a drug that would sensitize tumor better to chemotherapy and radiation
  • One (1) of ten trials of a “novel” treatment has an endpoint of improving quality of life for GBM patients

* * *

  • Eleven (11) phase III GBM trials are registered with known/already approved brain tumor therapies/treatment types
    • Five (5) are already active
    • Six (6) are still recruiting
  • These trials include different variations of:
    • Different combinations of chemotherapies and radiation
    • Different combinations of chemotherapy with radiation and Avastin
    • Different types of radiation
    • Different types of surgical techniques

* * *

Phase III Clinical Trials in GBM with Novel Investigational Agents



Not Yet Recruiting

Phase 3 Clinical Trials in GBM with Prior/Known Agents



*To support GBM research and help us get to even more potential new glioblastoma treatments faster, please visit here.

*To checkout more clinical trial options for GBM, and other types of brain tumors, please visit the brand new NBTS Clinical Trial Finder at

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