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10 Key Takeaways from the 2016 National Brain Tumor Society Scientific Summit

National Brain Tumor Society hosted its Annual Scientific Summit on Thursday, September 29, 2016, convening experts from some of the world’s top brain tumor research labs; leading brain tumor clinical centers; the U.S. Food and Drug Administration (FDA) and the National Cancer Institute (NCI); biopharmaceutical company researchers and executives; and patient advocates.

Dr. Elizabeth Jaffe

Unlike most scientific-based gathering in the field, this year, instead of focusing mainly on report-outs and presentations of new data, the Summit embraced a theme of “Capitalizing on the Inflection Point in Cancer Research.” In this way, the conversation focused on charting the field’s next steps and the best paths toward greater progress and accelerated treatment development and approval and included a keynote address from Dr. Elizabeth Jaffe (Johns Hopkins School of Medicine), Chair of the president’s National Cancer Advisory Board and Co-Chair of the Cancer Moonshot’s Blue Ribbon Panel.

Here were 10 of the key takeaways from the day’s discussions (in no particular order):

1. Engagement Between Stakeholder Groups

Patient Engagement

Engaging patients more often – and more deeply – in the research and drug development process was a major subject throughout the day.

The consensus was that patients are much more than end-users of products produced through biomedical R&D, but have important roles to play in shaping, improving, and accelerating research and the drug development process.

Dr. Elizabeth Claus

Dr. Elizabeth Claus of Yale University, who was awarded a grant from NBTS earlier this year for her International Low-Grade Glioma Registry, talked about how collecting real-world data from low-grade glioma patients via smartphone- and web-based applications can help researchers understand more about the effects of low-grade gliomas – and subsequent treatments – on patients’ daily life including the ability to work, drive, sleep, exercise, or take care of oneself and/or family.

Additionally, a number of speakers – including Dr. Jaffe, Dr. Andrew Joe of Merck, Dr. Joohee Sul of the FDA, and Dr. Patrick Wen of Dana-Farber Cancer Institute, along with a number of advocates and patients in the audience – spoke to the critical need to educate and engage patients on the importance of clinical trials. Many spoke of the myths and misconceptions that exist regarding clinical trials, and the need to get patients better informed and help them overcome barriers to enrollment. Greater patient participation in clinical research could significantly speed-up, as well as lead to more, clinical trials – which are necessary to get new treatments approved.

Dr. Wen summed-up the feelings, poignantly saying, “We owe it to patients to find trials for them, that they might be eligible for, even if our institutions aren’t offering any.”

Biopharmaceutical Industry Engagement

Dr. Joohee Sul

The field of neuro-oncology research is thankfully progressing to the point where many scientific discoveries made over the past few years are poised to be tested in clinical trials. However, much of the last parts of research and development needed before a potential new treatment enters the clinical setting, as well as financing for the trials themselves, often falls to biotechnology and pharmaceutical companies (“Industry”). Thus it is important for researchers working in academic medical center laboratories to continue to improve engagement with Industry. A number of brain tumor researchers – as well as Industry executives on hand – shared their thoughts and perspectives, including the importance of developing relationships and communication with potential Industry partners early in the research process.

Dr. Roger Packer of Children’s National Health System, and the Scientific Director of the newly announced Defeat Pediatric Brain Tumor Research Collaborative, offered how – in pediatrics – progress against pediatric low-grade gliomas has shown how great scientific advances can convince Industry to invest, even in small population diseases.

Finally, a major outstanding challenge in the neuro-oncology field – the inability of many drugs to get past the blood-brain-barrier (BBB) – led to another discussion on how the neuro-oncology field, with patient advocates, needs to work with Industry to encourage companies to develop drugs that are brain-penetrable and designed specifically to cross the BBB.

NBTS, in the last 1-3 years, has really revolutionized the landscape of neuro-oncology.

Attribution: Dr. Patrick Wen

Government Engagement

In addition to Industry, the other major sponsor of clinical trials in cancer is the National Cancer Institute (NCI) – a government institution. And, of course, the FDA is the government agency tasked with ensuring all cancer drugs are both safe and effective and are the ultimate approvers of any potential new treatment for brain tumors. Thus, it was noted how important it is for brain tumor researchers, drug developers, and clinical trialists and sponsors to maintain ongoing dialogue and engagement with these two government bodies. Specifically, the research community’s work with the FDA on clinical trial endpoints was highlighted as an example of how engagement has led to positive outcomes, with the development of the new Brain Tumor Imaging Protocol. Similar types of engagement with the NCI’s Cancer Therapy Evaluation Program (CTEP) could benefit NCI-funded brain tumor clinical trials, speakers opined.

2. Creating Standards

NBTS CEO David Arons (middle) and Chairman of the Board Michael Nathanson (right) greet keynote speaker Dr. Elizabeth Jaffe (far left) and NBTS Board member Dr. Susan Pannullo (second from left)

A number of speakers described how the Jumpstarting Brain Tumor Drug Development Coalition and its Clinical Trial Endpoints Steering Committee has made great progress toward standardizing aspects of processes that govern brain tumor research and drug development to improve research and clinical trials. This includes the Brain Tumor Imaging Protocol (noted above) that standardizes the way in which imaging data is collected to minimize variation in basic MR images taken during clinical trials, so that trial sponsors have “cleaner” data to provide to regulators like the FDA when they are considering approving a potential new brain tumor drug. However, more work, it was noted, is needed to standardize advanced imaging protocols, as well as in other endpoints like “Clinical Outcomes Assessments” that could further improve brain tumor clinical trials.

Summit participants also spoke of the need – especially in light of the recently updated World Health Organization’s brain tumor classification – to standardize the practice, in all medical centers that see brain tumor patients, of providing molecular and genomic testing of patients’ tumors. Making these test a routine part of clinical diagnostics could allow more patients to be better stratified for molecularly-matched or biomarker-driven clinical trials, and help push the field closer to the full realization of “precision” or “personalized/individual” medicine.

3. Data-Sharing

Data sharing has been a hot topic in cancer research for a number of years now, and, in fact, is a major premise of Vice President Biden’s Cancer Moonshot. Fortunately, the neuro-oncology field has been improving in this area, as highlighted by a number of collaborative initiatives that were reported on during the Summit, including: the Defeat Pediatric Brain Tumors Research Collaborative, Defeat GBM Research Collaborative, GBM AGILE, and The Glioma Longitudinal AnalySiS (GLASS) Consortium.

Dr. Roel Verhaak, who spearheads the GLASS Consortium, and others at the Summit, however, advocated for the next step in the progression of the data-sharing:  a move toward true “open science.” This would entail researchers regularly sharing “pre-published” data and making their data readily accessible to the research community, and even broader public when appropriate.

As Dr. Verhaak put it, “institutions are only labels.”

4. Biomarker Development and Validation

Biomarkers can be defined as, “a characteristic that is objectively measured and evaluated as an indicator of normal biological processes, pathogenic processes or pharmacological responses to a therapeutic intervention,” and they are becoming a more and more fundamental part of the research landscape.

NBTS advocates and supporters talk during a break at the 2016 Scientific Summit

While, most of the headline-grabbing scientific studies in biomedical research over the years have been about the discovery of therapeutic targets (usually genes or proteins that can be targeted with medicine/treatment to create a therapeutic effect to benefit patients), biomarker identification and validation has become vital to precision medicine and understanding which patients are most likely to respond to certain treatments or not.

Summit presenters spoke throughout the day of the importance of developing better biomarkers in brain tumor research. There have been thousands of clinical trials of potential drugs for brain tumors over the years. Unfortunately, as we know most of them have failed (as highlighted by the lack of treatment options available for brain tumor patients). Perhaps even more unfortunately, is the fact that, in many cases, we don’t know WHY they failed. Biomarkers could serve as the front-line in the field’s quest to better understand why certain treatments didn’t work, and even predict earlier in the research process treatments that have the greatest likelihood of succeeding, maximizing the utilization of resources across the field.

5. Clinical Trial Platforms & Designs

Dr. Timothy Cloughsey

As biomarkers pave the way for more informed and sophisticated clinical trials, the design of clinical trials themselves needs to be improved. It is because of this, that nearly a year after it was originally announced many speakers at the Summit were still effusive when discussing the GBM AGILE clinical trial. (“AGILE” stands for Adaptive, Global, Innovative Learning Environment). Presenting specifically on GBM AGILE, Dr. Timothy Cloughsey of UCLA, one of the leaders of the trial (and also a Defeat GBM researcher), told the group that GBM AGILE will serve as a continuous learning system where individual patient outcomes are analyzed constantly to inform the trial’s next steps based on real patient data.

Adaptive trial design is rapidly gaining interest and support in the cancer field because it can more quickly help identify drugs and biologics that do and don’t work, eliminating false positive and false negatives via biomarkers and quickly moving promising agents onto later stage trials. The hope is that GBM AGILE will eventually provide an opportunity for patients to benefit from the type of precision medicine that is tailored to each and every patient

Dr. Sul of the FDA answers a question during the NBTS 2016 Scientific Summit

The talk of GBM AGILE’s benefits dovetailed nicely with a lunchtime session from Dr. Sul of the FDA on “Read World Evidence for Drug Development,” in terms of emphasizing the need for clinical trial sponsors to learn to capture all data and info that matters, while weeding out data that doesn’t.

Later in the day, discussion shifted to how the field needs to move forward on clinical trials in a more coordinated manner in order to maximize resources – including patient and researcher time, as well as funding. Dr. Sul suggested this may also include better collaboration between leaders in the field and smaller community hospitals to identify the factors that will incentivize them to guide patients to, and through, clinical trials.

Dr. Wen followed on with some additional thoughts that would help improve the clinical trial environment in neuro-oncology:

  • Dr. Wen noted that more GBM patients should be allowed to participate in phase I trials and that many phase II clinical trials that enroll patients from across the cancer community to test targeted therapies (e.g basket or umbrella clinical trials) also fail to include brain tumor, particularly GBM, patients. Dr. Wen says the community needs to continue to advocate that brain tumor patients be considered for inclusion in these important trials.
  • In addition to greater inclusion of brain tumor patients in pan-cancer phase II clinical trials, Dr. Wen also believes – as well as many other experts – that the neuro-oncology field, independently, needs to develop a high-quality clinical trial group or network to conduct phase 0/1 trials to vet clinical candidates for larger trials like AGILE.

Finally, Summit participants mentioned the need for the field to ensure that there are adequate clinical trials available to newly diagnosed patients. Currently, many clinical trials are geared toward recurrent patients, or patients whose tumors have progressed after initial treatment. However, limiting clinical trials to primarily recurrent patients perpetuates the myth that clinical trials should just be considered as a “last resort” option. Moreover, newly diagnosed patients tend to have more predictable mutational landscapes, meaning they may be easier to target and treat with new precision medicines, and thus we could be missing opportunities to make progress for patients if we wait to try new drugs in recurrent studies (when tumors have often changed due to the original treatment they received).

6. Utilizing New Technologies

One of the more encouraging aspects of the current state of neuro-oncology research is the convergence of biomedical science with new technologies. During the Summit, utilization of new technologies was woven through the sessions and presentations as potential solutions to help facilitate brain tumor research and overcome existing challenges.

  • Claus spoke of using smartphones – and smartphone-enabled apps, like Fitbit – to help collect information directly from, and engage with, patients in the International Low-Grade Glioma Registry.
  • Multiple speakers discussed how advanced imaging (including molecular an nuclear imaging technologies, like PET scans) could continue to improve efforts to ensure better diagnosis and tracking of tumor response to therapy.
  • Liquid biopsies was also a theme that multiple speakers touched on in relation to both pediatric and adult brain tumor research and clinical care. Because of the location within the vulnerable brain, traditional surgical biopsy of brain tumors is often difficult and risky – and in some cases impossible. Liquid biopsy (may detect circulating tumor cells or fragments of tumor-DNA released into the blood or cerebrospinal fluid) could allow for easier diagnosis, treatment planning, and tumor monitoring.

NBTS is always reaching for the next step.

Attribution: Dr. Ingo Mellinghoff

Interestingly, the idea of telemedicine and other new technologies was even mentioned as a potential tool to help overcome geographic/travel obstacles that sometimes can hinder access to neuro-oncology specialists and clinical trial participation.

7. Combination Treatments

Like a number of topics already mentioned, combination therapy was a theme that was brought up in multiple talks during the Summit.

Dr. Patrick Wen

As we’ve known for some time, brain tumors are unfortunately very adaptable and have shown they can develop resistance to many of the different treatments that have been tried against them. Thus using combinations of treatments that could target the molecular changes driving the tumor, as well as the “escape routes” the tumor might use, is an attractive strategy for researchers, clinicians, and drug developers in the neuro-oncology field.

While the realization of the need for combinatorial strategies has been apparent within the neuro-oncology field for a number of years now, most approaches to-date have attempted to utilize new targeted agents with current standard treatments and/or other targeted therapies. The discussion at the Summit focused mainly on – now that additional treatment modalities are emerging – combining therapies across different classes, such as combining two different types of immunotherapies or combining immunotherapies with targeted agents. Various participants also discussed the need for more robust preclinical testing for combinations of treatments so combinatorial clinical trials are based on sound laboratory data.

8. Exploring Emerging Scientific Avenues (Beyond Genomics Research)

As our understanding of cancer has grown deeper, it is becoming clearer that tumors can be fueled by complex interactions between genes and the environment in which we (and thus all our cells) live. This makes it crucial to explore areas of research outside of traditional genomics-based research (searching for, and targeting, genetic mutations or other gene abnormalities). Immunotherapy is, of course, one of these emerging areas that needs more exploration, but Summit participants also noted the importance of investigating the fields of epigenetics/transcriptomics (how certain molecules in cells turn genes on and off without changing the DNA sequence); metabolomics; tumor microenvironment; and even what we could learn from brain metastases.

9. Viewing Research as a Continuum  

The term and idea of a “research continuum” was cited throughout the day, as an essential perspective for the field to approach brain tumor treatment R&D.

“Research doesn’t get done by one person doing the basic discovery and then another person translating it,” said Dr. Robert Strausberg of the Ludwig Cancer Research during the Summit’s closing panel. “That’s not fair. Research is continuum and we need to collaborate across all of it.”

Drs. Al Yung (foreground) and Robert Strausberg (background)

However, long before the closing panel took the stage, Dr. Alfred Yung, stressed that the continuum of research within the Defeat GBM Research Collaborative is vital to how the scientific data produced from the Collaborative can convince Industry to partner for clinical trials. Defeat GBM (like the Defeat Pediatric Brain Tumors Research Collaborative) is designed to cover the entire spectrum of preclinical research, from basic science and target discovery to translational research and drug discovery. With expert researchers – each member with unique backgrounds and resources – teaming-up across this continuum and sharing data, information, and materials, the Collaboratives can achieve what a single researcher, working alone, could not.

Cancer cells collaborate, so we need to, too.

Attribution: Dr. Robert Strausberg

10. Increased Funding for Research and Clinical Trials

Summit attendees chat during a break in the action

Of course, funding for research and clinical trials was also a pervasive theme during presentations and sessions at the Summit. The supposition was clear: the more resources this field gets – whether it be from philanthropic or government sources – the more it could take-on. Unfortunately, scientific experiments – and particularly operating clinical trials – are very expensive ventures, which means sometimes the only thing holding back big ideas is the investment and support needed to get them off the ground.

Many speakers mentioned that we all have a role to play in this regard. Even if an individual does not have the financial means to make donations to research, there are other opportunities to help fundraise – whether it be by advocating to congress for better funding for the NIH/NCI; engaging in and/or hosting your own peer-to-peer fundraisers (organizing a bake sale, community walk, crowdfunding campaign online, etc.); or simply raising awareness to the general population of the needs of the brain tumor community.

As a final note, we’d like to extend our thanks and gratitude to the many speakers, presenters, attendees, and, of course, sponsors – especially presenting sponsors DelMar Pharmaceuticals, Abbvie, and Bristol-Myers Squibb – of this year’s NBTS Scientific Summit.

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