Key Research and Emerging Protocols

The EndBrainCancer Initiative / Chris Elliott Fund is committed to identifying and promoting key research and protocols. The nature of the science and technologies is fluid and dynamic. Therefore, the medical protocols likewise need to be dynamic, agile, and aggressive.

As part of the EndBrainCancer Initiative we are actively encouraging patients, caregivers, and the medical community to collaborate and adopt the best and most promising treatment approaches. Below we explain our approach and highlight some of those areas of research that show the greatest promise.

translational2Translational Medicine

Translational medicine is an emerging field within biomedicine dedicated to quickly “translating” new scientific discoveries into clinical practice. This represents a paradigm shift in that laboratory research is conducted, not just for expanding our knowledge of biomedicine, but instead with the intent of predicting, preventing, diagnosing, and treating diseases. This approach also brings together the key players required to “translate” or introduce new discoveries into clinical practice – patients, clinicians, research scientists and industry partners – thereby making new therapies available to patients much sooner than would otherwise be possible.

Translational Medicine approaches serve as the foundation of the EndBrainCancer Initiative. It’s all about timing and getting patients the best care / advanced treatments and into clinical studies. It’s about staying constantly abreast of new developments and then “translating” those developments into fresh protocols. It is a dynamic process of effecting change. We at the EBC Initiative are focused on getting more patients into clinical trials and research. Currently only 6% of patients enter into clinical trials. This slows down the research and diminishes the possible benefits to the patients. At EBC we seek to be Change Agents spurring things on by advocating for public policy, advanced treatments and by an active “translation” of findings in the field.

Medical Devices

Medical devices are an emerging and exciting technology in the treatment of glioblastoma. Currently, the only one which the FDA has approved is Optune. Optune delivers TTF fields, frequency specific, low intensity fields which interfere with cell division to fight and stop tumor growth. In the United States, Optune is now available for both newly-diagnosed and recurrent GBM tumors. More on Optune can be found at

Testing Approaches

We recommend that two tests be made available for all GBM patients. This is critical for determining the most effective course of treatment and potentially increasing survivorship for this disease:

  • The MGMT promoter methylation test to identify those patients most likely to benefit from the alkylating agent chemotherapy temozolomide (Temodar) currently the Standard of Care.
  • Epidermal growth factor receptor (EGFR) test to identify those patients most likely to benefit from EGFR kinase inhibitors

What these tests do and why they are important:

MGMT promoter methylation test

Patients with glioblastoma and other malignant gliomas may have the MGMT gene inactivated due to methylation of its promotor region. When that happens, the gene is unable to repair DNA damage caused by chemotherapy and the patient is much more likely to respond to an alkylating agent chemotherapy such as temozolomide (Temodar) because it interferes with tumor growth. The MGMT promoter methylation test identifies those patients most likely to benefit from this type of treatment.

Epidermal growth factor receptor (EGFR) test

Mutations that lead to EGFR overexpression have been associated with a number of cancers, including lung cancer, anal cancers and glioblastoma multiforme. These mutations involving EGFR lead to its constant activation, which produces uncontrolled cell division resulting in tumor growth.

Now it is possible to identify those patients most likely to respond EGFR kinase inhibitors which can potentially “turn off” EGFR, disrupting this pathway for tumor growth. Testing for EGFR mutations identifies those patients most likely to benefit from this type of treatment.

Personalized or Precision Medicine

Increasing treatment options for GBM and other cancer patients

Personalized or precision medicine is a whole new way of looking at cancer treatment driven by recent advances in genomics and its practical application in tailoring treatment to the unique genetic makeup of each individual. This has profound implications for how cancer is treated and ultimately on survivorship because it opens up additional and better-targeted treatment options.
Up until very recently, most cancer treatment was based on the physical characteristics of the tumor cells, or their histological properties, as determined by looking at tumor cells under a microscope as well as the other physical characteristics of the tumor such as size and shape from tumor samples taken at biopsy and/or surgery.

Based on these findings, a treatment plan was decided upon using those treatment protocols having greatest past success with patients whose tumors had similar physical characteristics and which originated in the same area of the body.
Now it’s possible to go beyond what is learned under the microscope and identify the underlying molecular alternations responsible for the person’s cancer. DNA sequencing and genomic profiling are done to learn what is different genetically in these cells from normal tissue and where did things go wrong?

Broad testing for all known actionable molecular alterations gives patients the best opportunity to find available or investigational targeted therapies that have been specifically designed to treat their cancers. This includes the process of selecting and providing access to appropriate clinical trials that would otherwise not have been considered.

Thus treatment is personalized to the genomic characteristics of the tumor, and could include therapies developed for other tumor types which have similar genetic mutations.

As DNA sequencing and genomic profiling can potentially open up new treatment options, we advocate that these tests be made available to GBM and other cancer patients as part of Standard of Care.

Note that several tests for genomic alterations exist, and that these questions need to be asked and arrangements for the test need to be made prior to surgery so that tissue samples can be taken and forwarded to the appropriate lab for analysis.

Immunotherapy and Vaccines

Harnessing the body’s own immune system to kill cancer cells

In the last few decades immunotherapy has become increasingly important in the treatment of cancer and holds great promise for the future. Immunotherapy harnesses the body’s own immune system to attack cancer cells, either through boosting its overall response or through targeting those specific pathways that can hamper its effectiveness causing cancer cells to grow and spread. It works very differently from chemotherapy and other cancer treatments which kill cancer cells by interfering with cell division.

Immunotherapy can either be used by itself or in combination with other types of treatment.

Although cancer vaccines are perhaps the best known type of immunotherapy, there are several different types:

Monoclonal antibodies: These are man-made versions of immune system proteins which can be designed to attack a very specific part of a cancer cell.
Immune checkpoint inhibitors: These drugs basically take the ‘brakes’ off the immune system helping it to recognize and attack cancer cells.

Cancer vaccines: Vaccines are substances put into the body to start an immune response against certain diseases. While we usually think of vaccines as being given to healthy people to help prevent certain diseases, some vaccines can help prevent or treat cancer.

Non-specific immunotherapies: These treatments boost the immune system in a general way, but this can still help the immune system attack cancer cells
Recently, cancer vaccines have received a lot of media attention as a potential game-changer in treating glioblastoma. Early results from studies, one using an HIV drug and another using polio vaccine, have greatly extended the lives of some patients. These studies are still very much a work in progress, and appear to hold much promise.

Other types of immunotherapy currently in clinical trials include therapies targeted at tumor-specific oncogenes with patients whose tumors have specific tumor markers, and ones targeted at the proteins some tumors use to evade the immune system. In this case, immunotherapy can directly target these proteins, enabling the body to better detect and fight cancer.

As immunology and vaccines can potentially open up new treatment options, we advocate that these options be made available to GBM and other cancer patients where appropriate for their particular tumor type.