Neuro-Oncology Branch 2020 Year in Review

Together, our clinical team, research laboratories, and staff have made meaningful advancements to improve outcomes for patients and families affected by brain and spine tumors.

By Neuro-Oncology Branch Staff

January 22, 2021


Researcher typing on a computer at her lab bench

A harmonious team of scientists and health care providers at the NCI Center for Cancer Research's Neuro-Oncology Branch (NOB) collaborate to understand the complexities of brain and spine tumors and translate their research to improve treatments. The partnership between our neuro-oncology providers and researchers strengthens the commitment to make significant progress in research and care for patients with central nervous system (CNS) cancers.

As the New Year begins, the NOB shares accomplishments made throughout 2020. 

Neuro-Oncology Branch

In March, NIH officially went into maximum telework status, and it seemed that we were all thrust into a situation that required us to pivot and adapt in a short amount of time. Despite all of this, the NOB was able to adjust and make progress.

The NOB continued to hold regular Branch meetings, educational lectures, journal clubs, and tumor boards virtually. These virtual events allowed NOB staff to be productive and further their research knowledge and skills. The NOB now also uses technology to provide care for patients through telehealth appointments, so patients do not need to travel or make unnecessary trips outside their home.

Since the beginning of the pandemic, NOB also had 26 staff, fellows, and special volunteers begin new positions. This includes staff working on developing new therapies, researching patient outcomes, and striving to improve the lives of those with brain and spine tumors. “Although we found creative, new ways to keep advancing care and research at NOB and come together as a team, we all look forward to the time when we can all be together in person once again,” says Mark Gilbert, M.D., chief of the NOB.

Patient Outcomes Research Program

Led by Terri Armstrong, Ph.D., the Patient Outcomes Research Program seeks to gain a better understanding of how a patient feels and functions through their disease trajectory. The program also explores how clinicians could better time therapeutic intervention for maximal benefit, as well as how this will improve long-term care for this subset of patients.

In collaboration with leaders in the field, representatives from regulatory agencies, and patient advocacy representatives, Dr. Armstrong led a working group exploring core constructs to be measured in clinical trials and clinical research, which was published in Lancet Oncology this year. “Because the goal of any new treatment is to benefit the patient, it is important to include parameters that measure their clinical outcomes—how the patient feels and functions,” says Dr. Armstrong. The findings in this study support incorporating patient outcomes data into all clinical trials.

Translational Research Program

Led by Jing Wu, M.D., Ph.D., the Translational Research Program develops brain and spine cancer clinical trials that have a strong preclinical rationale. Dr. Wu strives to address gaps in knowledge in neuro-oncology and investigate novel treatment approaches to improve patient clinical outcomes.

In 2020, Dr. Wu’s laboratory investigated the effects of a novel treatment, MRX-2843, in a glioblastoma mouse model. MRX-2843 is a MerTK inhibitor. When MerTK is activated, it causes glioblastoma-associated macrophages and microglia (GAMs) to suppress the immune system and promote tumor growth.

Our findings suggest that MRX-2843 has a therapeutic benefit by causing GAMs to move away from immunosuppressive conditions, preventing neoangiogenesis (formation of new blood vessels) in the glioblastoma microenvironment and inducing tumor cell death,” says Dr. Wu. The findings of this study support developing clinical trials of MRX-2843 in glioblastoma.

Basic Immunology Research Program

Masaki Terabe, Ph.D., leads the Basic Immunology Research Program. His research focuses on various aspects of basic immunology, such as examining the biology of natural killer T (NKT) cells in brain and spine cancers. NKT cells are a type of immune cell that plays an important role in the body’s immune response and can kill certain cells, such as cancer cells. If NKT cells become inactive, this may contribute to cancer progression.

Using a glioblastoma mouse model (SB28 syngeneic orthotopic), Dr. Terabe's research laboratory found that CD1d-restricted NKT cells are involved in the suppression of the immune response against glioblastoma.

“This is the first observation, with our knowledge, strongly indicating that NKT cells are involved in the immune regulation in glioblastoma," says Dr. Terabe. "By revealing the mechanism used by NKT cells to regulate tumor immunity against glioblastoma, we may be able to discover a new therapeutic target for glioblastoma."

Translational Immunology Research Program

Led by Mark Gilbert, M.D., the Translational Immunology Research Program develops combination treatments with immunotherapy that can increase immune cell recruitment to the tumor. The researchers aim to improve patient selection in clinical trials to maximize benefit for those enrolled.

Dr. Gilbert’s group worked collaboratively with Javier Gonzalez Alarcon, M.D., to generate an important clinical trial that is designed to determine if they can predict which patients with brain tumors are most likely to respond to immunotherapy.

“This involves a blood test performed before treatment with the hope that we will be able to determine which patients should get these treatments,” says Dr. Gilbert. The study will also determine if an immune response in the blood is correlated with a response in the brain tumor.

Cancer Stem Cell Biology Research Program

Zhengping Zhuang, M.D., Ph.D., leads the Cancer Stem Cell Biology Research Program. His program uses critical patient observations to drive bench research, which can then translated to clinical use. His laboratory has a longstanding interest in understanding the functional impact of genetic changes that may uncover early disease states and potential treatment opportunities.

In 2020, his lab had an exciting development regarding a promising new drug effective against preclinical glioma models. This drug, TP5, was developed by our collaborator in the National Institute of Neurological Disorders and Stroke a decade ago. The drug inhibits cyclin-dependent kinase 5 hyperactivity in other disease models, including Alzheimer’s and Parkinson’s disease. Dr. Zhuang’s laboratory is looking to repurpose this agent. They found that TP5 was highly effective at treating glioma and colon carcinoma mouse models, and the treatment was well tolerated. “We are excited about the translational potential, and we are working on developing preclinical studies on glioblastoma,” says Dr. Zhuang.

Molecular and Cell Biology Research Program

Led by Chunzhang Yang, Ph.D., the Molecular and Cell Biology Research Program focuses on the biological effects of cancers with isocitrate dehydrogenase (IDH) mutations, in order to uncover selective vulnerabilities. By understanding the vulnerabilities of brain tumors, researchers can generate experimental treatments to improve the current standard of care for patients.

In 2020, his laboratory continued to investigate the mechanism of glioma therapy resistance, looking for a novel therapeutic strategy. “Specifically, we revealed that glutathione metabolism is critical for gliomas with an IDH mutation, which was proven to be effective through animal studies,” says Dr. Yang. Gliomas with an IDH mutation are a recently defined disease cluster. Curative therapy remains limited. “Our findings revealed the cancer vulnerability of IDH-mutated gliomas, which provides indications for drug discovery and clinical translation,” says Dr. Yang.

Cancer Metabolism Research Program

The Cancer Metabolism Research Program, led by Mioara Larion, Ph.D., is interested in the metabolic needs of cancer cells to delay tumor growth. Her laboratory also focuses on studying IDH-mutant gliomas, given the effect that this mutation has on tumor metabolism.

Dr. Larion’s laboratory has found that lipid pathways are important for IDH1-mutant glioma growth. They also found that targeting specific enzymes from either fatty acid synthesis or sphingolipid pathways leads to cellular death in these cells. “These findings have the potential to translate into targets for developing metabolic therapies," says Dr. Larion, "which may promote improved outcomes for patients diagnosed with IDH1-mutant gliomas."


NCI-CONNECT (Comprehensive Oncology Network Evaluating Rare CNS Tumors) aims to advance education about rare central nervous system (CNS) cancers by building patient-advocacy-provider partnerships and networks to improve care and treatment.

NCI-CONNECT collaborative efforts in research, education, and outreach expanded in 2020 through live virtual events. In June, NCI-CONNECT partnered with the National Brain Tumor Society to educate people with brain and spine tumors and their loved ones about how to navigate care through telehealth. In August, it partnered with the Society for Neuro-Oncology (SNO) to educate neuro-oncology providers on patient-centered communications via telehealth. Both events were timely and helped further patient care safely and successfully during a global pandemic.

In September, NCI-CONNECT partnered with SNO to host a virtual symposium focused on transdisciplinary global research in rare CNS tumors. The symposium allowed world-renowned researchers and health care providers to share their work—and address the challenges and possible solutions to improve patient outcomes. The event demonstrated that global collaborations are essential to advancing rare cancer research and education.

Brain Tumor Trials Collaborative

In an effort to develop and perform state-of-the-art clinical trials, the NOB leads a network of investigators across the nation called the Brain Tumor Trials Collaborative (BTTC). The BTTC strives to advance treatments for primary brain and spine tumors through novel and innovative clinical trial development and recruitment.

In 2020, the NOB restructured their Tumor Board so it could be held virtually and integrate BTTC institutions. The Tumor Boards brings together experts in neuro-oncology, neurosurgery, radiation oncology, and neuropathology to review and discuss patient cases. These events became increasingly important for patients who were unable to travel at this time—physicians could still work together to provide the best care and treatment plan for patients and families.

The BTTC sites were also invited to join the NCI-CONNECT consortium to perform clinical trials on 12 rare brain and spine tumors. “Using the BTTC multi-center infrastructure to expand rare tumor trials is valuable to this subset of patients, who often lack access to treatments specific for their tumor type,” says. Dr. Armstrong.

Continuing Advancements in Research and Clinical Care

Even through the pandemic, we have witnessed some remarkable and highly laudable activities by many in the NOB. “This is exemplified by the teamwork from our clinical group, enabling our clinical trials and patient care to continue while maximizing the safety for our patients and staff," says. Dr. Gilbert. "Similarly, in the laboratory, the disruption has been met with education and productivity in alternative venues that—combined with an increasing spirit of collegiality—has enabled continued progress.”

We are extremely optimistic for what we can accomplish in 2021 and the years to come.

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