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Report: 2018 Defeat GBM Retreat – Priorities for the Future

Glioblastoma (GBM) is perhaps the most complex and treatment-resistant cancer. As such, this tumor type is an acute focus for the National Brain Tumor Society (NBTS).

The Defeat GBM Research Collaborative (Defeat GBM) is NBTS’ flagship adult brain tumor research program.

Defeat GBM-funded researchers, Drs. Ingo Mellinghoff (left) and Frank Furnari (right) at the 2017 NBTS Scientific Summit

Launched in 2013, the program is now in its fourth year of funding toward a five-year, $10 million commitment. The disease trajectory goal of Defeat GBM is to conduct “team science” that ultimately leads to doubling the percentage of glioblastoma patients living five years or longer. Defeat GBM also seeks to demonstrate a new path forward for how glioblastoma research can be funded and conducted in a more effective, efficient, and collaborative manner, while increasing our knowledge of how to fight these devastating tumors. The program was designed to bring together multiple research labs from world-renown institutions to conduct science as a team, to share data, and advance discoveries from the lab to be ready for clinical trials.

To date, Defeat GBM has unquestionably been a major success. The Defeat GBM team of researchers has made discoveries that have changed the course of glioblastoma and, indeed, all brain tumor research as well as opened up whole new strategies and leads for developing more effective treatments. And over the last year, the researchers have progressed their work to now be ready to explore new drug approaches for sub-groups of GBM patients where particular drugs are more likely to be effective. They are doing the research that leads to what is commonly known as “precision medicine” or finding the right drug for the right patient at the right time.

At the 2018 Defeat GBM Research Annual Retreat the major emphasis was on:

  1. Prioritizing these discoveries, in terms of which held the most promise to move rapidly into clinical trials that could begin helping patients, and creating action plans to move forward,
  2. Aligning Defeat GBM progress and priorities with NBTS’ desire to become a stronger catalyst for new treatment development, and
  3. Identifying other areas of opportunity that could be acted upon to speed the accomplishment of Defeat GBM goals and projects.

At the end of the first day of the two-day Retreat, a list of some Defeat GBM priorities for 2018/2019 was developed:

  1. A project which utilizes the Defeat GBM discovery that when glioblastoma cells are effectively targeted by a type of drug called an “EGFR inhibitor” they reduce their uptake of the molecule glucose.
    • The strategy for using EGFR inhibitors to treat GBM is highly attractive because EGFR mutations are very common in GBM tumors.
    • PET imaging – a type of medical imaging – is able to detect changes in glucose levels in tumors, therefore – knowing that effective EGFR inhibitors will reduce glucose in GBM cells – the Defeat GBM team believes it can use a PET imaging approach in pilot clinical studies to rapidly (and non-invasively) determine which of the many EGFR inhibitors currently available for testing has the best chance of ultimately becoming an effective treatment for GBM patients, by monitoring the glucose levels in patients after giving the drug.
  2. A project which leverages the Defeat GBM-funded discovery that GBM cells become dependent on cholesterol to fuel their aggressive growth. The team has already identified a drug that gets into the brain and limits the amount of cholesterol the GBM cells can take in, potentially starving the tumor. They will continue efforts to acquire the drug and corresponding safety data and begin clinical trials in glioblastoma patients with this potential new medicine.
  3. A project which builds on a novel finding from the Defeat GBM team that using a type of drug called an “FGFR inhibitor” can make GBM cells more vulnerable to the effects of radiation treatment. Virtually all GBM patient will receive radiation as part of their treatment, but it, unfortunately, has only limited effectiveness. This new approach could help make it more effective. The team has four possible FGFR inhibitors they that want to complete final pre-clinical testing on, and then move into early phase clinical trials.

Also discussed were the potential importance of an Early Phase Clinical Trial Program and funding opportunities from outside of NBTS that may support the research.

These priorities will serve as a framework and plan for the remainder of the initial Defeat GBM program.

“It has been incredibly gratifying to be part of team Defeat GBM and see our results mature towards clinic application.” – Dr. Frank Furnari, Ludwig Cancer Research

To support the work of Defeat GBM, please consider making a gift, here.


  • MsMills MsSmith says:

    How does this relate to the use of a ketogenic diet? It certainly seems related.

  • MsMills MsSmith says:

    After my original diagnosis, my oncologist suggested using the ketogenic diet. However, I consumed a lot of food-based cholesterol during that time (while my serum cholesterol stayed low.) Is cholesterol consumption an issue or is it cholesterol levels that are more dependent on a person’s metabolism? Can anyone help me figure out how to find out?

    • TomHalkinNBTS says:

      Sorry to hear about your diagnosis. We’re wishing you the very best. To answer your question, at this point do not point to a particular supplement and/or diet that impacts (either way )the process involved in the dependency these tumors develop to cholesterol. That is to say, it’s not necessarily about the cholesterol we intake as part of our diets, which mainly gets metabolized outside the brain. It’s a corruption of the process the regulates the mechanisms of cholesterol intake within the brain…thus, at this point at least, the thought is that we need a drug called an “LXR Angonist,” which turns the correct process back on. We’re working getting an LXR angonist and trying it in clinical trials. A longer read on this discovery is here:

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