Chief
Mark R. Gilbert, M.D.
Deputy Chief
Terri S. Armstrong, Ph.D.
Building 82, Room 225
Bethesda, MD 20892-8202
240-760-6030

Neuro-Oncology Clinic at NIH


Dr. Jing WuBrain and Spine Tumor Clinical Trials

The Neuro-Oncology Clinic has a number of studies that are open for adult patients with brain and spine cancers.


The Neuro-Oncology Clinic is comprised of a multidisciplinary team of physicians, other healthcare providers, and scientists who are dedicated to developing new therapies and improving outcomes for patients with primary brain and spinal cord tumors.

We offer our patients:

  • Expert evaluation, examinations, tests and imaging
  • State-of-the-art neurosurgery and radiation therapy
  • Therapies based on the genetic characteristics of your specific tumor
  • Consultations and treatments at NIH are free-of-charge

The Neuro-Oncology Clinic is located at the National Institutes of Health (NIH) Clinical Center in Bethesda, Maryland.

This is a joint program of the Center for Cancer Research at the National Cancer Institute and the National Institute of Neurological Disorders and Stroke.


Special Update

Our Neuro-Oncology Clinic is changing normal operating procedures due to the novel coronavirus (COVID-19). 

  • If you are scheduled for an appointment within the next 30 days, you will receive a phone call from a patient care coordinator or health care provider to discuss your visit and what to expect.
  • If you need to schedule an appointment, your health care provider should contact us first.

Your care and safety remain our top priority and we appreciate your understanding during this time of continuous change. You can contact us at NCINOBReferrals@mail.nih.gov or 240-760-6010 with your questions or concerns.

- Posted March 20, 2020


Our Mission

The Neuro-Oncology Branch (NOB) is a trans-institutional initiative that launched in 2000. NOB is sponsored by both the National Cancer Institute (NCI) and the National Institute of Neurological Disorders and Stroke (NINDS). Our mission is to develop novel diagnostic and therapeutic agents for patients with primary central nervous system tumors. The branch's strategic direction is founded on the belief that the continued application of the traditional and largely empiric approach to cancer drug development that has dominated oncology for three decades will likely result in only small, incremental progress in the treatment of patients with malignant gliomas.

Meaningful advances in patient outcomes will more likely result from therapeutic approaches based on a greater understanding of glioma genetics and biology with the subsequent application of that understanding toward individualized and targeted treatments for patients based on their specific tumor characteristics. Additionally, clinical investigations will incorporate tools to measure the impact of the disease on patient function.  This will typically include measures of symptom burden, quality of life, and cognitive function.

Although we understand that the idea of "personalized medicine" will only be fully realized through the engagement of the entire cancer research and cancer care enterprise, the NOB functions under the premise that within the protected clinical environment and immense scientific freedom of the NIH intramural program, we are ideally situated to build a small prototype of a biology-driven, individualized, patient-centric rational therapeutics program.


Patients and Families


NOB_Patient_ClinicBeing told you have a brain or spine tumor can be shocking and overwhelming. As you find yourself facing complex decisions about treatment options, self-care and supportive resources, we offer comprehensive care together with your doctors back home.

At the NCI’s Neuro-Oncology Branch at National Institutes of Health (NIH), we have over 20 years of experience working with patients worldwide along with their primary oncologists to offer a comprehensive approach to patient care. We provide consultations free-of-charge for patients who are recently diagnosed with a primary brain or spinal cord tumor, those seeking a second opinion on their diagnosis or treatment, or those who have recurrent tumors. Patients can also visit us if they do not have a formal brain or spine tumor diagnosis but have recent imaging suggesting a primary brain or spine tumor.

To make an appointment, contact us at NCINOBReferrals@mail.nih.gov or call 1-866-251-9686 or 240-760-6010 (Monday to Friday, 9:00 a.m.–5:00 p.m. ET).

Care at the Neuro-Oncology Clinic

A Multidisciplinary Approach to Care

Consultations with branch providers at the Neuro-Oncology Clinic in Bethesda, Maryland, include our neuro-oncology physicians, nurse practitioners, research nurses, and social workers. While NIH does not cover expenses for medical services at other facilities, tests and treatments at NIH are free-of-charge after a patient is accepted on clinical study.

Our multidisciplinary approach to care paves the way for us to help patients and their families deal with both the physical and emotional aspects of this disease with resources to help cope with the diagnosis and treatment of a brain or spine tumor.

We also work closely with our colleagues in neurosurgery, radiation oncology, and radiology, as well as the patient’s primary doctor at home, to provide personalized and expert care.

  • Pathology: we use tumor tissue to precisely diagnose your tumor type, which helps us determine clinical trial eligibility and make accurate treatment decisions for your specific tumor to maximize clinical outcomes.
  • Imaging: we use the latest imaging techniques to give a detailed understanding of the potential for tumor growth and response to treatment. This helps our clinical team more accurately detect small but potentially significant changes in a tumor.
  • Surgery: we remove as much tumor as safely possible while not damaging healthy tissue using state-of-the-art surgical techniques and extensive pre-surgical planning.
  • Radiation: we use cutting-edge radiation therapy technologies that intensely focuses energy targeting the tumor while minimizing the radiation delivered to normal brain or spine tissue.

Clinical Studies

In addition to a general consultation or second opinion, patients have the potential to participate in one of our many clinical studies. We typically have between 5-10 active clinical studies for primary brain or spine tumors open, ranging from observational studies and symptom management to testing new drugs and tumor-focused interventions.

If the patient decides to participate in a clinical study, they will visit the NIH for consultations, follow-up, and imaging as needed free-of-charge. Much of their direct care will be transferred to our physicians and nurses for the duration of the study, although the patient will continue to be closely monitored by their referring or primary physician in collaboration with our clinical team.

Appointments

Patients can contact the Neuro-Oncology Clinic directly to make an appointment on their own or through a physician referral. We request that the patient or family member contact us directly to facilitate the consent process for the visit. Following consent, our patient care coordinators will provide instructions to send medical records.

We provide care for people with primary brain and spine tumors at various stages of their disease, including if:

  • they are newly diagnosed or have a recurrence and need to make a treatment decision
  • they are interested in a second opinion on follow-up care
  • they are interested in improving their care and the care of future patients

After you contact us, our patient care coordinators will follow-up with you within 48 hours. Please expect appointments to be scheduled a minimum of 2-3 weeks after we receive all medical records, forms and pathology materials.

  1. To make an appointment or refer a patient, contact us at NCINOBReferrals@mail.nih.gov or call 1-866-251-9686 or 240-760-6010 (Monday to Friday, 9:00 a.m. - 5:00 p.m. ET). If you are an international patient, you must be referred by your local oncologist.
  2. Our patient care coordinators will briefly discuss your diagnosis and send you the intake form to complete. This will include demographic information and your medical history. Next, you will talk with a research nurse where you will be able to ask any questions you have. They will then send you a consent form to complete. After your forms are complete, our patient care coordinators will schedule an appointment with you.
  3. Before your first visit, we will ask you to provide medical records. This includes all available MRI and CT scans related to your brain or spine tumor diagnosis, corresponding radiology reports, and a list of medications and pathology material from the most recent surgery (including biopsies). Your most recent MRI should be within 30 days of the appointment and we also require a recent progress note from your treating physician. Other records, including surgery, radiation, and treatment summaries may be requested to determine trial eligibility.

The Neuro-Oncology Clinic is located on the NIH campus in Bethesda, Maryland, on the 13th floor of the NIH Clinical Center (Building 10).

The NIH and the Clinical Center provide information on transportation, patient services, places to stay while you visit and how to get around the hospital.

Resources

A brain or spine tumor diagnosis can greatly impact all aspects of your life. Physical, cognitive or memory changes can be caused by the tumor, and removal of the tumor or treatment can affect your quality of life.  This can be challenging for you and your loved ones, and you will face unique challenges in each stage of your cancer journey. To help you cope and be informed, visit our recommended resources listed below.

Neuro-Oncology Branch Resources

Neuro-Oncology Branch Twitter
Follow us at @NIHBrainTumor to receive updates on the latest in brain and spine cancer research, care and treatment.

NCI-CONNECT Community
Join our Facebook page, NCI-CONNECT Community, to connect with other tumor patients and their caregivers.

NCI-CONNECT Rare Brain and Spine Tumor Network
If you have a rare brain or spine tumor, find more information on diagnosis, grading, tumor types and treatment options.

Living With a Brain or Spine Tumor
View questions to ask your health care team, ways to manage your self-care, and treatment and support resources for each stage of your cancer journey.

Other Resources

NIH Wellness Toolkit
Guidance to help you improve your health needs. Learn how to manage your surroundings, feelings, body, relationships and health.

NCI Coping with Cancer
Learn how to cope with feelings and cancer, adjusting to cancer, self-image and sexuality, day-to-day life, support for caregivers, survivorship and questions to ask your doctor.

Brain and Spine Tumor Organizations
Find support and connect with others impacted by a brain or spinal cord tumor.

NCI Brain and Spine Tumor Information
Learn more about the many different brain and spinal cord tumor types and how they are treated. Find information about brain cancer statistics, research and clinical trials.

Brain and Other Nervous System Cancers | Did You Know?



Brain Tumor Trials Collaborative


Brain Tumor Trials Collaborative

The mission of the Brain Tumor Trials Collaborative (BTTC) is to develop and perform state-of-the-art clinical trials in a collaborative and collegial environment, advancing treatments for patients with brain and spine tumors, and merging good scientific method with concern for patient well-being and outcome.

The BTTC was created in 2003 as a network of professionals who investigate new treatments, allowing patients across a broad geographic range to participate in cutting-edge clinical trials and help medical professionals more rapidly determine the benefits of various therapies. The National Cancer Institute's (NCI) Center for Cancer Research serves as the lead institution and provides the administrative infrastructure, clinical database and oversight for the collaborative.

NCI-CONNECT (Comprehensive Oncology Network Evaluating Rare CNS Tumors) aims to advance the understanding of rare adult central nervous system (CNS) cancers by establishing and fostering patient-advocacy-provider partnerships and networks to improve approaches to care and treatment. The BTTC network serves as the infrastructure for NCI-CONNECT clinical studies and trials focused on rare adult brain and spine tumors. By partnering with advocacy organizations and BTTC doctors and institutions, NCI-CONNECT researchers hope to improve approaches to care and treatment.

Each of the participating BTTC and NCI-CONNECT institutions and expert physicians are committed to discovering new and effective therapies for the treatment of brain and spine tumors while providing compassionate care to enhance patient outcomes and well-being. Contact the BTTC Coordinating Center if you have questions about BTTC clinical trials at NCI_BTTC@mail.nih.gov or call (240) 760-6060.

 

BTTC Interactive Map of Doctors and Institutions

Salt Lake City, UT

Dr. Howard Colman
Dr. Howard Colman University of Utah Huntsman Cancer Institute 801-585-0260 Director of Medical Neuro-Oncology Clinical Interests: The treatment of primary and metastatic brain tumors. The diagnosis and management of neurologic complications of cancer. His clinical research is focused on the development and testing of new therapies for brain tumors.

Bethesda, MD

Dr. Terri Armstrong
Dr. Terri Armstrong National Institutes of Health 240-760-6010 Co-Principal Investigator and Director of Patient Outcomes Research for BTTC; Deputy Chief, Neuro-Oncology Branch, Center for Cancer Research, National Institutes of Health Clinical Interests: Gauging impact of treatment on symptoms, health related quality of life and neurocognitive functions in patients with primary brain tumors. Evaluation of toxicities of therapy. Symptom cluster analysis.
Dr. Mark Gilbert
Dr. Mark Gilbert National Institutes of Health 240-760-6010 Principal Investigator, Research Group for BTTC; Chief, Neuro-Oncology Branch, Center for Cancer Research, National Institutes of Health Clinical Interests: Development of new chemotherapy agents, phase I testing, pharmacology and drug resistance modulation, development of novel clinical trial strategies.
Dr. Marta Penas-Prado
Dr. Marta Penas-Prado National Institutes of Health 240-760-6010 Associate Research Physician, Neuro-Oncology Branch, Center for Cancer Research, National Institutes of Health Clinical Interests: Adult medulloblastoma, development of clinical trials with targeted agents for malignant gliomas, neurological complications of cancer and cancer therapies.
Dr. Jing Wu
Dr. Jing Wu National Institutes of Health 240-760-6010 Investigator, Clinical Fellowship Director, Neuro-Oncology Branch, Center for Cancer Research, National Institutes of Health Clinical Interests: Development of pre-clinical studies and investigator-initiated clinical trials of personalized, biology-based treatments of primary CNS tumors.
Dr. Brett Theeler
Dr. Brett Theeler National Institutes of Health 240-760-6010 Clinical Collaborator, Neuro-Oncology Branch, National Institutes of Health Clinical Interests: Rare CNS neoplasms.

Houston, TX

Dr. Carlos Kamiya Matsuoka
Dr. Carlos Kamiya Matsuoka University of Texas MD Anderson Cancer Center 877-632-6789 Assistant Professor, Department of Neuro-Oncology Clinical Interests: Neurology and psychiatry.

Kansas City, KS

Dr. Michael Salacz
Dr. Michael Salacz University of Kansas Cancer Center 913-574-1049 Assistant Professor Clinical Interests: Neuro-oncology. Developing therapies for treatment of primary and metastatic cancers of the brain and spinal cord, including cytotoxic chemotherapies, targeted therapies and immunotherapies.

Boston, MA

Dr. Patrick Wen
Dr. Patrick Wen Dana-Farber Cancer Institute 617-632-3703 Director, Center For Neuro-Oncology, Dana-Farber Cancer Institute Professor of Neurology, Harvard Medical School Clinical Interests: Focus on novel treatments of brain tumors, especially targeted molecular agents. His other clinical interests include neurologic complications of cancer.
Dr. Isabel Arrillaga
Dr. Isabel Arrillaga Massachusetts General Hospital 617-726-2862 Associate Clinical Director, Neuro-Oncology Clinical Interests: Early phase clinical trials in glioma.

Dallas, TX

Dr. Karen Fink
Dr. Karen Fink Baylor Health Care System 214-820-8690 Lead Neuro-Oncologist Clinical Interests: Bring advanced methods of treatment to her patients. It is her mission to attain the best possible outcome and an improved quality of life for all of her patients coping with a brain tumor diagnosis.
Dr. Edward Pan
Dr. Edward Pan University of Texas Southwestern Medical Center 214-645-8186 Medical Director, Annette G. Strauss Center for Neuro-Oncology Department of Neurology & Neurotherapeutics Clinical Interests: Clinical trials involving novel targeted therapies for primary malignant gliomas, low-grade gliomas, neoplastic meningitis, brain metastases, quality of life assessments and early phase translational studies.

Evanston, IL

Dr. Ryan T. Merrell
Dr. Ryan T. Merrell NorthShore University HealthSystem 847- 570-2570 Clinical Assistant Professor Clinical Interests: Primary and metastatic brain tumors, CNS lymphoma, meningiomas, novel neuroimaging techniques.

Detroit, MI

Dr. Tobias Walbert
Dr. Tobias Walbert Henry Ford Health System 313- 916-2723 Neuro-Oncologist, Hermelin Brain Tumor Center Clinical Interests: Clinical trial development, quality of life, socio-economic evaluation.

Chicago, IL

Dr. Priya Kumthekar
Dr. Priya Kumthekar Northwestern University Feinberg School of Medicine 312-695-0990 Disease Team Lead in Neuro-Oncology; Director of the Brain Metastases Program Clinical Interests: Brain cancer, brain metastases, clinical trials and quality of life.

Chapel Hill, NC

Dr. Simon Khagi
Dr. Simon Khagi University of North Carolina 984-974-8432 Director, Brain Tumor Program Clinical Interests: Novel stem cell-based therapies, neuro-immunotherapeutics, design of early phase clinical trials, and enhanced imaging techniques for brain tumors.

Cleveland, MD

Dr. David Peereboom
Dr. David Peereboom Cleveland Clinic 866-320-4573 Director, Clinical Research; Brain Tumor and Neuro-Oncology Center Clinical Interests: Benign and malignant tumors of the brain and spinal cord, benign brain tumors (meningiomas, schwannomas), brain metastases, brain tumors, CNS metastatic disease, experimental therapeutics, molecular biology of brain tumors, clinical and translational trials, neuro-oncology, tumors of the brain and spine, cancer.

Winston-Salem, NC

Dr. Glenn Lesser
Dr. Glenn Lesser Wake Forest Baptist Health 336-713-5433 Louise McMichael Miracle Professor in Oncology; Associate Chief, Section on Hematology and Oncology; Wake Forest Baptist Medical Center Clinical Interests: Neuro-oncology, cancer pain control, cancer symptom management, brain tumors.

Orlando, FL

Dr. Nicholas Avgeropoulos
Dr. Nicholas Avgeropoulos Orlando Healthcare 321-843-2584 Director, Section of Neuro-Oncology Assistant Professor, University of Central Florida Clinical Interests: Expertise includes the design and conduct of clinical and translational research studies advocating the health and benefit of neuro-oncology patients.

Milwaukee, WI

Dr. George Bobustuc
Dr. George Bobustuc Aurora Health Care 414-384-5111 Medical Director, Neuro-Oncology Clinical Interests: Working on several projects focusing on brain, breast and pancreatic cancer. He has one patent in method and composition for enhancing PGE1 production in vascular and endothelial and smooth muscle cells.

Los Angeles, CA

Dr. Jeremy Rudnick
Dr. Jeremy Rudnick Cedars-Sinai Medical Center 310-423-4818 Attending, Neuro-Oncologist Clinical Interests: Clinical trials, brain tumor vaccines, immunotherapy and molecular targeted therapy.

Columbus, OH

Dr. Vinay K. Puduvalli
Dr. Vinay K. Puduvalli Ohio State University Wexner Medical Center 614-293-4448 Professor and Director, Division of Neuro-Oncology, Arthur G. James Cancer Hospital Clinical Interests: Developing novel therapies for patients with primary brain tumors and specifically focusing on malignant gliomas. His laboratory efforts are focused on: 1) understanding the role of epigenetics in brain tumor biology and translating these findings into new treatment options; 2) identifying means to accelerate tumor cell death by activating death pathways in brain tumors.

Seattle, WA

Dr. Lynne Taylor
Dr. Lynne Taylor University of Washington Medical Center 206-598-2282 Attending Physician, University of Washington Medical Center, Seattle Cancer Care Alliance; Clinical Professor, Department of Neurology; Neuro-Oncologist, Co-Director Alvord Brain Tumor Center Clinical Interests: Clinical neuro-oncology; paraneoplastic disorders; improving conversations about goals of care; palliative care in neurology.

Austin, TX

Dr. Morris Groves
Dr. Morris Groves Texas Oncology 512-421-4105 Director of the Austin Brain Tumor Center Chair, Neuro-Oncology Research Clinical Interests: All primary and metastatic CNS tumors, leptomeningeal spread of cancer. Research interests include development of novel and targeted agents for primary and metastatic CNS cancers. Biomarkers of disease, and molecular targeted drug development, and clinical trial design.

New York, NY

Dr. Fabio Iwamoto
Dr. Fabio Iwamoto Columbia University Medical Center 212-342-0571 Assistant Professor of Neurology Deputy Director, Division of Neuro-Oncology Clinical Interests: Diagnosis, management and treatment of brain and spinal cord tumors as well as neurological complications of cancer.

Charleston, SC

Dr. David Cachia
Dr. David Cachia Medical University of South Carolina 843-792-7749 Assistant Professor, Neuro-Oncology Clinical Interests: Primary brain tumors and molecular characterization of brain tumors.

BTTC Clinical Trials

Clinical trials are medical research studies conducted to understand whether promising approaches to cancer prevention, diagnosis and treatment are safe and effective in patients. Since its inception, the BTTC has experienced unparalleled advancements in the research of brain and spine tumors and completed a number of clinical trials. Several exciting new trials are in development. Clinical trials through the BTTC hope to provide a safer and effective treatment to control the tumor, increase survival, and improve quality of life.

Interested in participating in a BTTC clinical trial? Contact the BTTC Coordinating Center at NCI_BTTC@mail.nih.gov or call (240) 760-6060.

Openly Recruiting BTTC Clinical Trials

  • GBM Pembrolizumab HSPPC-96 - Newly Diagnosed Glioblastoma Before Surgery.
    This phase II clinical study will evaluate the combination of a personalized cancer vaccine made from the patient’s own tumor tissue (HSPPC-96) and PD-1 inhibition (pembrolizumab) in newly diagnosed glioblastoma. Learn more about the trial here and view the flier for more information.
  • Everolimus/Sorafenib Study - Recurrent High-Grade Gliomas
    This phase I-II clinical study will evaluate the safety and efficacy of everolimus and sorafenib in patients with recurrent high-grade gliomas. Learn more about this trial here.

BTTC Clinical Trials in Development

  • Pazopanib/Topotecan Study - Recurrent Glioblastoma
    This phase II clinical study’s primary objective is to determine if pazopanib when given in combination with topotecan is safe, effective and can control glioblastoma. Learn more about this trial here.

Completed BTTC Clinical Trials

  • Bevacizumab/Vorinostat Study - Recurrent Glioblastoma
    This phase I-II clinical study evaluated the occurrence of symptoms and their relations to disease progression, as well as the tolerance to the treatment regimen with bevacizumab versus bevacizumab plus vorinostat in adults with recurrent glioblastoma. Learn more about this trial here.
  • Bevacizumab/Tarceva Study - Newly Diagnosed Malignant Glioma
    This phase II clinical study evaluated the effects and determined overall survival rates of newly diagnosed malignant glioma patients administered a combination of bevacizumab and erlotinib after completion of radiation therapy with temozolomide. Learn more about this trial here.
  • Temodar Combination Study - Newly Diagnosed Glioblastoma
    This phase II clinical study sought to determine if temozolomide administered alone or in combination with thalidomide, isotretinion and/or celecoxib was effective in the continued treatment of newly diagnosed glioblastoma patients after radiation therapy. Learn more about this trial here.

Published BTTC Papers and Features

Published Papers

BTTC Meetings

  • BTTC Annual Meeting (July 2019) – The  NCI-CONNECT (Comprehensive Oncology Network Evaluating Rare CNS Tumors) and Brain Tumor Trials Collaborative (BTTC) Annual Meetings were held on April 29-30, 2019 at the National Institutes of Health (NIH).
  • BTTC Annual Meeting (July 2018) – The inaugural NCI-CONNECT Meeting was held in conjunction with the BTTC Annual Meeting on June 11-12, 2018 at the NIH in Bethesda, Maryland with record attendance.
  • BTTC Annual Meeting (June 2017) – The BTTC held a productive meeting on March 31, 2017 at the NIH. At the meeting, which included neuro-oncologists from 26 hospitals across the United States, BTTC members shared information about current and upcoming clinical trials.

Comprehensive Oncology Network Evaluating Rare CNS Tumors


NOB_NCI-CONNECTNCI-CONNECT (Comprehensive Oncology Network Evaluating Rare CNS Tumors) aims to advance the understanding of rare adult central nervous system (CNS) cancers by establishing and fostering patient-advocacy-provider partnerships and networks to improve approaches to care and treatment.

Primary CNS cancers are tumors that start in the brain and spinal cord. They account for less than 2% of all cancers diagnosed a year in the United States, with over 130 different types. Although all primary adult CNS tumors can be considered rare, NCI-CONNECT is starting with 12 types, each with less than 2,000 people diagnosed a year.

View NCI-CONNECT Website

The low incident rate of these 12 tumors means patients and researchers face unique challenges. Patients struggle to find expert care and treatments. Researchers have limited resources to study the disease and develop new therapies.

NCI-CONNECT intends to address these challenges and unmet needs by connecting patients, providers, researchers and community organizations to work in partnership.

NCI-CONNECT selected rare adult CNS tumor types:

  • Atypical Teratoid Rhabdoid Tumor (ATRT)
  • Choroid Plexus Tumors
  • Diffuse Midline Gliomas
  • Ependymoma
  • Gliomatosis Cerebri
  • Gliosarcoma
  • Medulloblastoma
  • Meningioma (High Grade)
  • Oligodendroglioma / Anaplastic Oligodendroglioma
  • Pineal Region Tumors
  • Pleomorphic Xanthoastrocytoma / Anaplastic Pleomorphic Xanthoastrocytoma
  • Primitive Neuro-Ectodermal Tumors

Studies

NCI-CONNECT has a number of studies that are open for adult patients with rare central nervous system cancers. Check back as we plan to add more.

View NCI-CONNECT Studies

Receive Updates

Receive the latest news in rare adult central nervous system cancer research, care, and treatment.

Connect with Others

Join our Facebook page, NCI-CONNECT Community, to connect with other patients and caregivers.

Contact us: NCICONNECT@mail.nih.gov or call (240) 760-6530


About

NCI-CONNECT is a program within the Rare Tumor Patient Engagement Network (RTPEN), an initiative supported by the National Cancer Moonshot. RTPEN aims to advance research and discover new treatments for rare tumors through a collaborative network of national and international institutions that will provide expertise, personalized health care and education to patients with rare tumors.

NCI-CONNECT as part of RTPEN focuses on rare central nervous system tumors and is managed at the National Institutes of Health, National Cancer Institute, Center for Cancer Research, Neuro-Oncology Branch.


Brain and Spine Cancer Research


With support from the investigators in the Neuro-Oncology Branch (NOB), we have developed robust programs addressing critical areas of brain and spine cancer research. Our investigators translate laboratory findings into clinical trials which are designed to assess whether a treatment is working and what the impact is on the patient. Each of the laboratory programs have the same goal – to improve brain and spine cancer patient care and outcomes. 


Clinical Research Program

Led by Mark Gilbert, M.D. and Terri Armstrong, Ph.D., this program encompasses a growing portfolio of therapeutic brain and spine cancer clinical trials as well as a series of complementary non-therapeutic studies. The infrastructure that has been developed for clinical research has enabled our investigators to launch studies including phase 0 studies using both microdialysis techniques as well as post-treatment tumor resection to determine tumor drug delivery, phase I studies with pharmacokinetics and pharmacodynamics, and single arm phase II studies. Larger scale clinical trials are being done in the Brain Tumor Trials Collaborative and the NCI-CONNECT Clinical Trials Network, both led by the NOB. Notably, patient outcomes measures are integrated into each clinical trial, complementing ongoing work in the Patient Outcomes Program. 


Patient Outcomes Program

Led by Terri Armstrong, Ph.D., this program is focused on implementing outcomes measures that assess brain and spine cancer patients during the course of their disease. Utilizing the Natural History Study with longitudinal collection of clinical characteristics, Patient Reported Outcomes measures to learn cancer risk information, and biologic samples including germline DNA for single nucleotide polymorphisms are collected for evaluation. Dr. Armstrong is also developing preclinical models for treatment-related toxicities including radiation-induced somnolence which may have an association with polymorphisms of select CLOCK genes.


Translational Research Program

Led by Jing Wu, M.D., Ph.D., this program is focused on the development of brain and spine cancer clinical trials that have strong preclinical science and rationale, enabling the clinical trial to be hypothesis-based with strong correlative biology. Studies with the novel targeted agent, TG02, led to the creation of a successful phase I trial. This program is also investigating the malignant transformation and hypermutation that occurs in a subset of patients harboring IDH-mutated gliomas. This translational research complements the work by Dr. Mioara Larion and the Cancer Metabolism Research Program, translating preclinical models and imaging biomarkers to clinical trials. 


Immunology Program

Initially led by Mark Gilbert, M.D., this program has recently transitioned its leadership to Masaki Terabe, Ph.D., and is focused on various aspects of translational and basic immunology, such as examining the biology of NK-T cells, potent mediators of immune response, in brain and spine cancers. These topics include:

  • Developing techniques to assess the immune phenotype of brain and spine tumors
  • Patient immunocompetence and metrics of peripheral response to immune modulators
  • Evaluating the impact of treatment agents such as chemotherapy and corticosteroids on immune reactivity
  • Achieving a better understanding of the mediators of an effective immune response such as immune cell trafficking
  • Chemokine production in the umor microenvironment 

The Cancer Stem Cell Biology Program

Led by Zhengping Zhuang, M.D., Ph.D., this program focuses on his longstanding interest and expertise in evaluating hypoxia and pseudohypoxia as well as the role of HIF2α abnormalities in brain and spine cancer biology. The ongoing research is focused on understanding the functional impact of the genetic changes that may uncover early disease states and potential therapeutic opportunities. 


Molecular and Cell Biology Program

Led by Chunzhang Yang, Ph.D., this program is focused on the biologic impacts of cancer-associated IDH mutations to uncover selective vulnerability of brain and spine cancers. These investigations have demonstrated that DNA repair pathways are compromised due to the effects of the IDH mutation and that Nrf2-mediated metabolic pathways, such as glutathione de-novo synthesis, support IDH-mutated cells by relieving metabolic stress and oxidative damage. These studies may have a direct link to clinical research as Dr. Jing Wu is developing a robust therapeutic program for patients with IDH-mutant tumors. 


Cancer Metabolism Program

Led by Mioara Larion, Ph.D., this program is studying IDH-mutant gliomas, given the impact that this mutation has on tumor metabolism. Her investigations are exploring the impact of IDH mutations on cellular requirements for carbon sources, lipid metabolism at the molecular, organelle, and cellular levels as well as preclinical development of hyperpolarized MRI using 13C substrates to measure in vitro metabolic flux. Her research compliments the cell biology-focused research by Dr. Chunzhang Yang and the translational and clinical research on IDH-mutant tumors being performed by Dr. Jing Wu.


Training Programs


NOB_TrainingJob Vacancies

The Neuro-Oncology Branch (NOB) Clinic has various job vacancies. The Neuro-Oncology Clinic is comprised of a multidisciplinary team of physicians, other healthcare providers, and scientists who are dedicated to developing new therapies and improving outcomes for patients with primary brain and spinal cord tumors. Click here to view current openings.


Clinical Fellowship

This is a joint program with the Neuro-Oncology Program at the Johns Hopkins Hospital in Baltimore, MD.  The goal is to prepare neurologists, medical oncologists, radiation oncologists and neurosurgeons for research careers in neuro-oncology.

Training includes:

  • Formal coursework in pharmacology and clinical research methodology, 
  • Academic biweekly conferences
  • Patient management from a pool of more than 2,000 brain tumor patients who are seen each year in our Brain Tumor Clinic

Trainees will participate in developing and implementing clinical trials for adult brain tumor therapeutics. Fellows also have the opportunity to attend national scientific meetings and are mentored in pursuit of their clinical, basic, and translational brain tumor research interests. 

Click here for more information or to submit an application. For further questions, email NCINeuroOncology@mail.nih.gov.


Clinical Elective

The National Institutes of Health (NIH) Clinical Electives Program emphasizes the important connection between research and patient care in neuro-oncology. Students participate in inpatient and outpatient evaluation of patients, learn about the conduct of clinical research trials, observe surgical procedures, attend clinical care and research conferences, and participate in weekly clinical and laboratory meetings. Students will participate in the workup, evaluation, and planning of treatment for patients they see.

Objectives include:

  • Prepare trainees to deliver state-of-the-art diagnostic and consultative services in the field of neuro-oncology
  • Introduce trainees to research in neuro-oncology and to inspire them to pursue a lifelong career of academic inquiry

The elective affords an opportunity to gain an understanding of the techniques of clinical research, as well as experience in the evaluation and treatment of patients with diseases of the CNS. Although laboratory research is closely coordinated with clinical activities in the NOB, the student will not engage in laboratory investigations during this course.

Click here for more information or to submit an application. For further questions, email NCINeuroOncology@mail.nih.gov.


Clinical Base Training

The NIH Clinical Center offers an extensive range of clinical research training to help prepare the next generation of clinician-scientists. The innovative curriculum includes courses in pharmacology, principles and practice of clinical research and bioethics.

Training programs include:

  • Summer internship program
  • Internships throughout the year
  • Undergraduate scholarship program
  • Intramural research training program
  • Postdoctoral training program
  • Research fellowship
  • Clinical fellowship

Click here for more information or to submit an application. For further questions, email NCINeuroOncology@mail.nih.gov.


Summer Internship Program

Trainees accepted into one of the NIH Summer Internship programs and working in the Neuro-Oncology Branch can participate in the NOB’s Translational Research Immersion Program (NOB-TRIP). This program is designed to immerse interns in the patient-centered research conducted within the NOB. Interns will have the opportunity to observe and engage with clinical and laboratory professionals that lead clinical, translational, and basic research. This 10-week program consists of five structured areas of educational focus.

Training includes:

  • Didactic lectures
  • Weekly seminars with the NOB Chief on research rigor and career planning
  • Cross-focus observational experiences
  • Integration of wellness
  • Mentoring in training and public speaking

Opportunities are available for high school, college, grad school and professional school students through the NIH Summer Internship Program in Biomedical Research program.

Click here for more information or to submit an application. For further questions, email NCINeuroOncology@mail.nih.gov.


News

NOB_NewsDeveloping Clinical Trials to Advance Care for Brain and Spine Cancers 

November 13, 2020

Developing clinical trials for brain and spine tumors is vital to improving care for patients. However, neuro-oncology trials have unique challenges given the rarity and characteristics of these tumors. Learn more...


Glioblastoma Survivor, Bob, Finds Hope at the NIH

July 1, 2020

Bob was diagnosed with glioblastoma in 2015. A previously active bicyclist and outdoor enthusiast, read how he made many lifestyle changes to accommodate his needs as he underwent treatment. Learn more...


BTTC Colleagues Integrated into Weekly Tumor Board

June 25, 2020

In a continuing effort to increase collaboration, the weekly Neuro-Oncology Tumor Board held at the NIH is now inclusive of BTTC doctors at remote institutions to improve patient care. Learn more... 


Andrew, An Astrocytoma Survivor Building a New Life

June 19, 2020

Andrew, an astrocytoma survivor, visits the Neuro-Oncology Branch regularly for treatment advice from our experienced neuro-oncology physicians and nurses. Read about his experience with our clinic and new outlook on life after diagnosis. Learn more... 


Telehealth: What You Need to Know About a Virtual Doctor Visit

June 19, 2020

Neuro-oncology experts share what telehealth is, the benefits and limitations, how to prepare and what to expect during and after your visit. Learn more... 


Spotlight on Matt Lindsley

May 26, 2020

Matt Lindsley, a research nurse at the Neuro-Oncology Branch, also serves as a Lieutenant in the U.S. Public Health Service. His passionate service to the NOB clinical trial patients is integral to our branch's mission. Learn more... 


Celebrating Nurses Week

May 12, 2020

We highlight the tireless contribution our nurse practitioners, research nurses and nurse-scientists make to provide the best care for our brain and spine tumor patients. Learn more... 


Advances in Clinical Care and Research 

April 29, 2020

Our Neuro-Oncology Branch is advancing care and treatment for people with brain and spine tumors through collaborative scientific workshops, research discovers, and successful clinical trials. Learn more... 


New Strategy for Treating Brain Tumors with Mutations in Etabolic Enzymes​

April 10, 2020

Dr. Chunzhang Yang and his team uncover the molecular effects of IDH mutations in gliomas, including stress, reactive oxygen species and changes in a protein named NRF2. The group also explored how a small molecule inhibitor for NRF2 can make glioma cells more responsive to therapy in preclinical mouse models. Learn more... 

Tracking Patient Outcomes to Improve Clinical Trials

March 6, 2020

In collaboration with leaders in the field, Dr. Terri Armstrong published a paper outlining the importance of tracking patient outcomes in clinical trials. Including these patient-reported outcomes can help physicians better understand the impact of the tumor on the patient and their lives. Learn more... 


Women Scientist Appreciation Month

March 1, 2020

In celebration of International Women and Girls In Science Day, we celebrated female scientists, physicians and mentors in the Neuro-Oncology Branch that strive everyday to make advances in ground-breaking research for brain and spine tumor patients. Learn more... 


Exploratory Adapatation Explains How Glioblastoma Cells Adapt and Survive

February 5, 2020

Dr. Orieta Celiku and Dr. Mark Gilbert publish an article on the concept of exploratory adaptation in glioblastoma. Using bioinformatics and mathematical modeling, they have found tools to explain how glioblastoma cells may adapt to survive in stressful conditions. Learn more... 


Celebrating National Mentoring Month

January 31, 2020

We celebrate our impactful mentors in the Neuro-Oncology Branch for National Mentoring Month. Our postdoctoral fellows share their love for science and how their mentors have made their experience at NIH more rewarding. Learn more... 


FDA Grants Orphan Drug Designation to Zotiraciclib for the Treatment of Glioma​

January 9, 2020

Dr. Jing Wu's investigational drug, zotiraciclib, receives orphan drug approval by the FDA for the treatment of glioma, which comprises up to 30% of all brain and central nervous system tumors. Learn more... 


Why a Precise Diagnosis is Vital to Treat Brain and Spine Tumors​

October 10, 2019

NCI neuropathologist Dr. Kenneth Aldape is using the latest technologies to precisely diagnose brain and spine tumors. He shares his techniques and why they’re important to precision medicineLearn more...


Clinical Trial Tests New Drug for Recurrent Ependymomas

May 28, 2019

Ependymomas are rare tumors that arise in the fluid-filled cavities in the brain and spinal cord. A new clinical trial is testing a drug that targets ependymomas that have a specific genetic signature. Learn more... 


New Strategy for Treating Brain Tumors with Mutations in Metabolic Enzymes​

February 20, 2019

Dr. Chun Zhang Yang studies how IDH-mutant gliomas and reactive oxygen species (ROS) can allow glioma cells to become malignant and survive. Learn more... 


Clinical Trial Studies Antitumor Effect of Sunitinib in Central Nervous System Sarcomas

February 4, 2019

A new clinical trial studying the cancer drug sunitinib for recurrent gliosarcoma and previously treated sarcomas of the CNS, which comprises the brain and spinal cord. Learn more...


Targeting Survival: Living Well with Brain Cancer in the Era of Precision Treatments

November 1, 2018

Dr. Mark Gilbert outlines his work in immunotherapy brain cancer research at the Cooperative Trials Group for Neuro-Oncology (COGNO) 2018 Learn more...


See Brain Cancer as More Than Just the Sum of Biology

September 26, 2018

Dr. Terri Armstrong shares how neurological symptoms, as well as biological changes in cells and tissues, contribute to the disease’s characteristics. Learn more...


NCI-CONNECT Rare Brain and Spine Tumor Network Website Launches

September 24, 2018

New network fosters patient-advocacy-provider partnerships and networks to improve care and treatment of rare brain and spine tumors. Learn more...


Can Immunotherapy Suceed in Glioblastoma?

May 24, 2018

Despite continued efforts to develop new therapies for glioblastoma, none have been able to improve how long patients live appreciably. Despite some setbacks, researchers are hopeful that immunotherapy might be able to succeed where other therapies have not. Learn more...


Rare Cancer Moonshot Project

December 6, 2017

Dr. Mark Gilbert shares information about the new moonshot project focusing on rare brain tumors: bringing together scientists and clinicians while closely following the patient experience. Learn more...


Adding Immune-Based Treatments to Standard Glioblastoma Therapy

October 11, 2017

This clinical trial is testing standard therapy (surgery, radiation and temozolomide) plus immunotherapy with pembrolizumab with or without a cancer treatment vaccine for patients with newly diagnosed glioblastoma. Learn more...


Clinical Trial Aims to Study Immunotherapy for Central Nervous System Tumors

August 23, 2017

A new clinical trial aims to determine whether nivolumab, an immune checkpoint inhibitor, can improve control of cancer for patients with several types of tumors of the central nervous system (CNS). The CNS is composed of the brain and spinal cord and the cause of most CNS tumors in adults is unknown. Learn more...


Glioblastoma – Unraveling the Threads

August 3, 2017

Progress against the brain cancer glioblastoma has been slow. Drs. Gilbert and Armstrong of NCI’s Neuro-Oncology Branch discuss why and what’s being done to change that. Learn more...


Clinical Trial Explores New Glioblastoma Treatment Regimen

May 22, 2017

On average, a patient with glioblastoma lives for only about a year after diagnosis. In a new clinical trial, CCR researchers will test whether a combination of a vaccine made from the patient’s tumor with another immunotherapy treatment can increase survival rates. Learn more...


PARP Inhibitors May be Effective in Brain, Other Cancers With IDH Mutations

April 24, 2017

Two separate studies have revealed new insights into the function of genetic mutations commonly found in a form of brain cancer as well as several other cancers and, in the process, identified a potential new treatment strategy. Learn more…


Brain Cancer’s Cholesterol Addiction May Offer New Strategy

December 26, 2016

Brain cancer cells are heavily dependent on a constant supply of cholesterol to survive, a new study suggests. And in mice with brain tumors, treatment with a cholesterol-depleting drug slowed tumor growth and improved survival. Learn more…


Study Tests the Safety and Efficacy of Combination Therapy in Adults with Astrocytoma and Glioblastoma

December 2, 2016

A two-part clinical trial of a multikinase inhibitor plus chemotherapy in patients with two types of brain tumors is enrolling. The study will be open to patients with anaplastic astrocytoma, an uncommon malignant brain tumor that develops from star-shaped brain cells called astrocytes and glioblastoma, the most common and aggressive form of astrocytoma. Learn more…


Breaching the Blood–Brain Barrier

August 10, 2016

Despite the development of gene sequencing and targeted cancer treatments se advances, the biological equivalent of a brick wall — the blood–brain barrier — has remained almost impenetrable. Dr. Sadhana Jackson shares how researchers are on a quest to breach the blood-brain barrier to treat brain cancer. Learn more…


Clinical Trial to Study Long-Term Progression of Brain and Spine Cancers

August 8, 2016

Dr. Mark Gilbert describes an ambitious new clinical trial that, for the first time, will study the long-term progression of brain and spine cancers. The 10,000 atient trial is the largest of its kind and will follow patients throughout the course of their disease. In addition to identifying optimal treatments for common brain and spine cancers, the study focuses on treatment discovery for rare, overlooked cancers. Learn more…


Trials Produce Practice-Changing Results for Brain Cancer

June 9, 2016

Results from two clinical trials show that treating patients with the chemotherapy drug temozolomide in addition to radiation therapy increased overall and progression-free survival. Learn more…


With Immunotherapy, Glimmers of Progress Against Glioblastoma

March 8, 2016

Researchers who specialize in brain cancer are optimistic about immunotherapy’s prospects for improving outcomes in patients with this aggressive form of brain cancer. Learn more…


Genome Study Links Misfolded DNA to Brain Tumors

January 12, 2016

Changes in the packaging of DNA in the cell nucleus may activate growth-promoting genes that cause cancer. Learn more…


Patient with Brain Tumor Responds Dramatically to Targeted Therapy

December 3, 2015

A patient with a papillary craniopharyngioma saw his tumor shrink more than 80 percent after treatment with a targeted drug that inhibits a mutant form of the BRAF protein. Learn more…


Genomic Underpinnings of Brain Tumors Expanded

June 10, 2015

TCGA researchers analyzed nearly 300 cases of diffuse low- and intermediate-grade gliomas, which together comprise lower-grade gliomas. LGGs occur mainly in adults and include astrocytomas, oligodendrogliomas and oligoastrocytomas. Learn more…


"Flipping the Switch": An Interview with Dr. Mark Gilbert, Chief of NIH’s Neuro-Oncology Branch

February 18, 2015

In November 2014, Mark Gilbert, M.D., became chief of the NIH Neuro-Oncology Branch. NCI's Dr. Mark Gilbert discusses new developments in neuro-oncology and the priorities of the Neuro-Oncology Branch. Learn more…


Neuro-Oncology Branch Welcomes Dr. Mark Gilbert as New Branch Chief

October 30, 2014

Dr. Gilbert's vision is to build a highly collaborative, robust translational research program centered on finding treatments for brain and other central nervous system tumors.  Learn more about Dr. Gilbert…


A New "America's Neuro-Oncologist" at the National Cancer Institute

October 28, 2014

"Having had the pleasure of working together with Dr. Gilbert on a number of projects in recent years, I can attest to his expertise, professionalism, and dedication to the brain tumor community, said David Arons, Chief Public Policy and Advocacy Officer, National Brain Tumor Society. "Dr. Gilbert is an excellent fit, and we look forward to working with him as he develops his programs and priorities for the Neuro-Oncology Branch moving forward."  Read more…