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Scientific research is the backbone of the National Brain Tumor Society’s (NBTS) mission and programs.
When our legacy organizations were formed in the 1980’s the general knowledge in the medical and scientific communities regarding brain tumors and how to treat this disease was very minimal. As such, our founders realized the only way to a cure would be through focused efforts to advance research.
Since that time, the field of neuro-oncology has made great advances in our understanding of brain tumors and has begun developing therapies that help extend life and improve patients’ quality of life. However, our increased understanding of brain tumors has only translated into incremental improvements in patient care – Temodar (temozolomide) was introduced in 1999; carmustine wafers in 2003; Avastin (bevacizumab) in 2009; and Optune in 2011 – thus it has become a major focus of NBTS to not just fund research, but to drive research all the way through the translational and clinical research phases toward approval.
One key to accomplishing this is to help dismantle the historical walls between the key research disciplines that play a role in advancing brain tumor research (academic scientists, clinician-scientists, biopharmaceutical industry scientists, etc.) And one of the ways NBTS accomplishes this is by convening these groups at our annual Scientific Summit.
The 2015 National Brain Tumor Society Scientific Summit brought together renowned leaders from all aspects of the brain tumor field — many actively engaged in our ground-breaking research initiatives — to share our collaborative work, explore emerging opportunities, and build strong and diverse partnerships to fast-track new discoveries toward a cure.
This year, we focused almost exclusively on the progress being made through our shared work, partnerships, and collaborations. Here are the highlights:
Defeat GBM Research Collaborative
We kicked-off with report-outs from the leaders of each of the four cores of research teams that make-up Defeat GBM.
The researchers in the Discovery (presented by Dr. Frank Furnari of Ludwig Cancer Research), Systems Biology (presented by Dr. Ingo Mellinghoff of Memorial Sloan Kettering Cancer Center), and Biomarkers (presented by Dr. Paul Mischel of Ludwig) cores all shared how their work is really starting to go deeper into mapping the total effects known molecular alterations have on GBM cells so that treatments can be developed with the ability to overcome the resistance we typically see from GBMs.
At the same time, the Drug Development core (presented by Drs. John de Groot and Erik Sulman of MD Anderson Cancer Center) is beginning to look at rational drug combinations that could work against GBM, and is concurrently developing a number of new preclinical testing tools that will continue to aid future drug testing.
The session concluded with presentations and discussion on two new areas of interest, for potential future incorporation into Defeat GBM: immunotherapy and a game-changing, global adaptive-style clinical trial.
Dr. Tiffany Hodges, a fellow in the lab of Dr. Amy Heimberger – a leader in brain tumor immunotherapy research – at MD Anderson Cancer Center, provided information on a “scorecard” approach that could help researchers prioritize which immunotherapies and approaches might be ripe for testing in GBM.
Tim Cloughesy of UCLA discussed how an adaptive-style clinical trial is needed to realize the research coming out of Defeat GBM and other efforts, as they are biomarker driven and aimed at getting the right drug to the right patient. Importantly, the learning from this style of trial would feed back into, and inform, the rest of the Defeat GBM cores.
We go wherever we have to go; where no one has gone before to find better treatments for GBM patients.Attribution: Dr. W.K. Alfred Yung, MD Anderson Cancer Center and Defeat GBM Scientific Director, comparing Defeat GBM to the Starship Enterprise from the famous TV show Star Trek
Project Impact: Defeat Pediatric High-Grade Glioma Research Collaborative
Data sharing, collaboration, and translational discoveries are key in the field of pediatric brain tumors – particularly the aggressive class known as pediatric high-grade gliomas (pHGG) – because of small patient populations. We learned more about how the Defeat Pediatric High Grade Glioma Research Collaborative and other complementary projects are supporting these scientific needs to advance research.
Dr. Nada Jabado of Montreal Children’s Hospital shared how recent discoveries from her lab and other researchers demonstrate that pHGGs are “epigenetic” disorders of the developing brain – “brain development gone wrong.” Epigenetic means alterations happening in the cells not caused by changes to the DNA sequence. In the case of pHGGs, mutations have been found in a particular protein – called a histone – that helps wind and package the DNA in a cells’ nucleosome.
Dr. Jabado said the questions that remain for research are now:
In order to answer these advanced scientific questions, Dr. Jabado said, “We need to come together, and we are.” She described Project Impact’s Defeat Pediatric High-Grade Glioma Research Collaborative as a “collaborative network” of world-class pediatric brain tumor researchers committed to “open access” data and sharing information to speed research efforts, noting that all of the sequencing studies done as part of their work will be made publicly available.
Joining Dr. Jabado in this collaborative will be Dr. Stefan Pfister of the German Cancer Research Center Heidelberg, and Suzanne Baker and Kip Guy of St. Jude Children’s Research Hospital. The group will tackle complementary research efforts that aim to answer the questions above through projects that focus on pHGG biomarkers, genomics and epigenomics, developing preclinical models, and screening of potential therapeutic compounds that selectively kill pHGGs with histone H3 mutations.
Dr. Jabado noted this collaborative network would also look to link with other consortia of pHGG researchers.
One such is the Pacific Pediatric Neuro-Oncology Consortium (PNOC). Led by Drs. Michael Prados and Sabine Mueller of the University of California, San Francisco (UCSF) this group now features 14 member institutions from across the country, and is actively working with the Translational Genomics Research Institute (TGen) on a pHGG clinical trial platform. Through this work, PNOC and TGen have already identified nine potentially actionable recurring genomic alterations, and, importantly, nearly 25 potential existing drugs that could work against some of the mutations.
Dr. Mueller concluded by stating that, as their initial feasibility study was successful, the trial is now preparing to expand in collaboration with the Defeat PHGG effort
In order to continue to learn from studies such as the Defeat Pediatric High-Grade Glioma Research Collaborative and the PNOC/TGen effort – and as Dr. Jabado stated – data needs to be available for other researchers to easily access. As such, during our pediatric session at this year’s Summit we also heard from two experts in “Big Data,” both of whom represented companies which we are exploring partnerships with to help realize our data needs in pediatric brain tumor research.
Famed computational biologist, expert in genome science and bioinformatics, Harvard School of Public Health professor, and co-founder of the company Genospace, John Quackenbush, PhD, compared cancer to a “whodunnit.” Genospace builds platforms for storing, curating, and sharing genomic and other biomedical data, and he stressed the importance of these platforms in solving the whodunit of cancer.
“Today, I can put the cost of sequencing a genome on my credit card,” said Dr. Quackenbush. “This is exciting because we can get more data. But a large pile of data…is just a large pile of data. You have to be able to make sense of it through algorithms and analytics.
Dr. Quackenbush stated that the goal is to create a resource where people who understand the disease can access data easily and use it to, “ask the questions that will get better answers for patients.”
Following up on Dr. Quackenbush’s presentation, Michael Schmidt, PhD, founder and CEO of the big data company Nutonian, discussed the need for cutting-edge technology that can use “machine learning” or artificial intelligence to generate advanced algorithms to proactively mine data stored on platforms like those created by Genospace.
Today, datasets we look at have thousands of variables that could be influencing them….and [researchers] looking at that data could spend years trying to chase equations to get the right one. Smart people in biology shouldn’t be spending tons of time doing mathematics and complex statistics – machines should be doing that.Attribution: Dr. Schmidt
By the end of this session, it was clear that the pediatric brain tumor field is now working more openly, and collaboratively than ever to find better treatments, and ultimately cures, for these vulnerable patients that deserve better.
Oligodendroglioma Research Fund
In 2013, NBTS gave out the first two grants as part of Oligodendroglioma Research Fund to Drs. Anders Persson of UCSF and Drs. David Louis and Mario Suva of Massachusetts General Hospital. These funded research projects are coming to an end, and Drs. Persson and Suva provided updates on what they have found so far that could ultimately benefit oligo patients.
Dr. Persson revealed that the grant he received from the Oligodendroglioma Research Fund allowed his lab to grow from an “embryo to a full-blown research laboratory.” His lab’s goal has been to figure out the origin of oligo tumors, and how the origin might affect treatment decisions. His team has already published three papers based on the work he has done with his oligo grant, and is preparing for three more. More importantly, his team has been able to create better models of oligo tumors that more accurately reflect the tumor in human, which will be critical to future efforts to test potential new treatments.
Dr. Suva explained how his and Dr. Louis’ work has helped provide the first, unbiased demonstration of how cancer stem cells impact the development and progression of oligodendrogliomas. By studying the evolution, genetic progression, and development of gliomas, Dr. Suva has been able to better understand possible vulnerabilities of these tumors and how to exploit them with new treatment approaches. Specifically, their work shows that oligo progenitor cells (OPCs), long thought to be the origin for oligo tumors, are actually not the cell of origin. In fact, it is neural stem cells that likely initiate oligo tumors which may be made up of a mixture of not only oligodendrocytes, but also astrocytes, and thus we should be targeting cancer stem cells for treatment.
Following the Persson and Louis/Suva grants, we issued a third grant from the Oligodendroglioma Research Fund in 2014 to Drs. Robert Jenkins and Daniel Lachance of the Mayo Clinic. At the Summit, Dr. Jenkins also presented his early findings, including how his work studying the natural progression and lineage of oligodendrogliomas is helping to better classify low-grade gliomas. As Dr. Lachance has said of this work, “This molecular data helps us better classify gliomas for patients, so we can begin to understand who needs to be treated more aggressively and who might be able to avoid unnecessary therapies.”
After looking at the science coming out of the Oligodendroglioma Research Fund, we decided to use the Summit as an opportunity to explore the current landscape of oligo research and treatment and how we might be able to accelerate this knowledge into treatments patients can benefit from. As such, NBTS’ Associate Director of Scientific Operations, Ann Kingston, PhD, presented a landscape analysis that showed an area ripe for exploiting for clinical benefit was the development of biomarkers.
We have advanced our understanding of the molecular underpinnings of oligodendroglial tumor development…this knowledge has provided some initial direction for exploring targets and biomarkers in the disease pathways for the development of more effective targeted therapeutics. Clinical biomarkers are the foundation of precision-based medicine and finding biomarkers to improve our assessment of efficacy and safety in small numbers of patients is critical to advancing targeted therapies…biomarkers are an important bridge between early research studies and later clinical evaluations.Attribution: Dr. Kingston
Finally, Dr. Susan Chang of UCSF presented on an emerging effort being established by the Dabbiere family, UCSF, and seven other institutions to eradicate low-grade gliomas called “Loglio.”
Loglio has a goal of immediate impact through focused efforts of a collaborative group of low-grade glioma experts working in teams. Dr. Chang discussed how there is great synergy between Loglio and NBTS’ oligo efforts, saying that Loglio would “leverage current projects and expertise of investigators, while adding new researchers and projects” to the fight against low-grade glioma, including oligodendrogliomas.
We believe if we focus on the target; provide the funding; get multiple institutions involved; we can hit the target faster. All of these approaches are helping each other.Attribution: Dr. Yung
Our final session of the day focused on the state of the field of metabolic therapy and the opportunities for exploration and investment, including as potentially NBTS’ next Community Research Fund.
Robert Strausberg, PhD, Ludwig Institute for Cancer Research, opened the session by explaining that tumor metabolism was once a big area of interest in cancer therapy development, but that the field lost interest in the past decade. However, Dr. Strausberg noted that just in the past couple of years, metabolic therapies are receiving renewed interest as more and more studies indicate how genetics and our environment conspire to cause cancer.
During this session, presentations were given by a range of speakers including Chandri Navarro (NBTS Board of Directors member), Dr. Que Collins (a cancer immunologist and cancer widower), Dr. Jen Helfer (Associate Director of Scientific Operations, NBTS) as well as leading researchers in the field, including Drs. Adrienne Scheck (Barrow Neurological Institute), Jaishri Blakeley (Johns Hopkins School of Medicine), and Paul Mischel (Ludwig Institute for Cancer Research, UCSD). These presentations highlighted several studies showing how changes to cancer cell metabolism can fuel tumor growth and resistance to treatment as well as how possible lifestyle changes that are currently being studied could aid therapeutic response.
We’ve heard all day about how cells and cell signals collaborate to form cancer. And just as they do, we must – in the cancer research word – collaborate and team up to overcome this disease.Attribution: Dr. Strausberg
Photos courtesy of Garnick Moore Photographers.
Presenting sponsor: DelMar Pharmaceuticals.