National Brain Tumor Society Cites Ten Reasons for Brain Tumor Community Excitement in 2017

Brain tumor field enters new year with momentum after significant developments & progress in 2016

National Brain Tumor Society (NBTS), the largest nonprofit dedicated to the brain tumor community, enters 2017 with greater enthusiasm about its mission – to find better treatments and ultimately a cure for brain tumors – than ever before. A host of important advancements within the neuro-oncology and oncology fields during the past year, have set the stage for even greater progress in 2017. Many of these discoveries and advancements were accomplished as a result of NBTS research funding, public policy advocacy, and other activities.

In neuro-oncology, there has been encouraging news in the movement towards more precision-based medicine (tailoring treatment to the individual patient) and immunotherapeutic approaches in the past couple of years. Recent advances in the molecular analysis, understanding, and characterization of brain tumors – including the potential role cancer stem cells, epigenetics, tumor metabolism, and tumor microenvironment play in the initiation, progression, and maintenance of tumors – have created optimism that more effective and beneficial treatments are within reach. This includes a number of ongoing phase 3 clinical trials awaiting data and results in 2017 and 2018, as well as the publishing of long-term data confirming that adding Optune to temozolomide, following surgery and radiation, extends survival in newly diagnosed glioblastoma patients.

Below are 10 examples of key developments and discoveries in the field of neuro-oncology in 2016:

1. In early 2016, the World Health Organization (WHO) updated its classification system for brain tumors, including, for the first time, molecular characteristics (genetic information) along with traditional histopathology (how a tumor looks under a microscope). The updated classifications allow for more accurate diagnosis, prognosis, treatment planning, and clinical trial stratification in the field of brain tumor research and clinical care.
2. Researchers at Ludwig Cancer Research, San Diego – funded through NBTS’ Defeat GBM Research Collaborative – discovered, how glioblastoma (GBM) tumors exploit the brain’s unique metabolism to import vast amounts of cholesterol, creating a dependency, and, importantly, how attacking the mechanisms these tumors use to do so may halt this deadly cancer’s growth.
3. In 2016, the potential use of non-invasive “liquid” biopsies and liquid biomarkers began to emerge, with multiple papers – including a seminal work also funded by the Defeat GBM Research Collaborative showing that CSF harbors clinically relevant genomic alterations in patients with CNS cancers. This work is paving the way for new advances in the diagnosis, monitoring, treatment planning, and research of brain tumors – both primary and metastatic.
4. A new paper, funded through NBTS’ Oligodendroglioma Research Fund, identified for the first time the existence of cancer stem cells (CSC’s), defined as cells that can self-renew and differentiate into multiple types of cells found in tumors, in human oligodendroglioma tumors and that CSCs are primarily responsible for fueling the growth of these tumors in humans. These findings highlight that targeting a specific stem cell phenotype may be an actionable therapeutic approach for oligodendroglioma patients.
5. Advanced immunotherapeutic approaches in neuro-oncology – like checkpoint inhibitors and CAR T-Cell therapy – are, unfortunately, behind where they are in some other cancers. But, encouragingly, in 2016 three different check-point inhibitors – nivolumab (Opdivo), pembrolizumab (Keytruda), and durvalumab (MEDI4736) – all showed that they were safe and tolerable for GBM patients. And though phase 1 trials aren’t designed to necessarily measure effectiveness, all showed early signals of efficacy and feasibility, as did a potential approach to CAR T-Cell therapy for GBM.
6. In 2016, the push continued to move the field toward more patient-centric research and drug development. Early in the year, NBTS and its partners in the Jumpstarting Brain Tumor Drug Development Coalition helped publish a supplemental journal issue providing details from a workshop the coalition previously hosted on the topic. Later in the year, NBTS provided funding for The International Low-Grade Glioma Registry which will help researchers understand more about the effects of low-grade gliomas – and its treatments – on patients’ daily life. Finally, the year ended with the announcement that Dr. Terri Armstrong was joining the Neuro-Oncology Branch at the National Cancer Institute in a new role focusing primarily on improving the assessment of patient outcomes measures. Dr. Armstrong, previously of MD Anderson Cancer Center, has served on several of NBTS’ own volunteer committees related to the topic of patient-centered research.
7. A banner year for brain tumor public policy advocacy, saw NBTS hold a record-setting “Head to the Hill” advocacy day in Washington; NBTS’ CEO represent brain tumor patients on the Blue Ribbon Panel of Vice President Joe Biden’s Cancer Moonshot Initiative; the passage of the NBTS-supported 21^st Century Cures Act; and the NBTS-supported Childhood Cancer STAR Act moved to the verge of becoming a law. The ultimate result of these efforts is an increase in federal support for cancer research initiatives important for brain tumor research.
8. In 2016, low-grade glioma research got a major boost with two separate funding announcements from NBTS’ Oligodendroglioma Research Fund and partners, totaling over $1.25 million. This work is already helping create new disease models, understand the molecular underpinnings of these tumors, and uncovering potential treatment targets. Also in 2016, researchers presented data at major conferences showing, for the first time in a well-controlled clinical trial, that adjuvant temozolomide (after radiation or chemo/radiation) has benefit to grade III gliomas, creating a new treatment standard for these patients.
9. Quality neuro-imaging is important in helping researchers and doctors understand if a treatment is successfully shrinking a patient’s tumor. Brain tumor imaging (neuro-imaging) was a major topic in the field this year, and the subject of multiple sessions at SNO 2016. NBTS and the Jumpstarting Brain Tumor Drug Development Coalition also continued its own imaging initiative with plans to pursue field-wide adoption of the new Brain Tumor Imaging Protocol developed in 2015 (covering basic MR-imaging (MRI) for multi-center clinical trials) while also establishing a path forward on the creation of standardized protocols for advanced imaging modalities (like perfusion and diffusion MRI) beyond basic anatomic MRI.
10. In September, NBTS and the St. Baldrick’s Foundation partnered to gather the best-of-the-best in pediatric brain cancer research to launch the Defeat Pediatric Brain Tumors Collaborative, which has been called a, “national and international priority” and, “a major opportunity for the field, patients and their families.” The Collaborative will be looking to add new partners in 2017. Elsewhere in pediatric brain tumor research, a significant new finding was published in 2016 by the International Cancer Genome Consortium Pediatric Brain Tumor Project that highlighted a new mechanism of tumor recurrence in pediatric GBM, providing strong rationale for investigating MET inhibitors, in combination with other targeted therapies, in future pediatric GBM clinical trials.

“There is much for the brain tumor community to be thankful for, in terms of the critical momentum generated toward a future where brain tumors can be treated effectively,” said David Arons, Chief Executive Officer, National Brain Tumor Society. “More importantly, there’s a lot to be optimistic about in terms of the developments that we could see in 2017, based on past progress and where the field is heading.”

National Brain Tumor Society anticipates that these scientific insights, partnerships and collaborations, improved policies, funding increases, and beneficial changes to the brain tumor R&D ecosystem will provide the groundwork to dramatically transform the brain tumor treatment landscape in the very near future.

In 2017, NBTS is aiming to help move at least one clinical candidate into human trials, while reaching other key research and scientific-related mile markers along the way in all of our ongoing programs, and securing increased federal funding for brain tumor research while driving forward federal precision medicine policy in our nation’s capital.

About National Brain Tumor Society

National Brain Tumor Society (NBTS) is the largest nonprofit organization in the U.S. dedicated to the brain tumor community. We are fiercely committed to finding better treatments and driving rapid progress toward a cure for brain tumors. We drive a multi-faceted and thoughtful approach to aggressively influence and fund strategic research, as well as advocate for public policy changes, in order to achieve the greatest impact, results, and progress for brain tumor patients. Money raised by the generous donations of our supporters has directly funded groundbreaking discoveries, programs, clinical trials and policy initiatives. To learn more visit www.braintumor.org