
Mark R. Gilbert, M.D.
- Center for Cancer Research
- National Cancer Institute
- Building 82, Room 235A
- Bethesda, MD 20892
- 240-760-6023
- 301-480-5124
- mark.gilbert@nih.gov
RESEARCH SUMMARY
Dr. Gilbert has developed a unified team within the Neuro-Oncology Branch (NOB) to perform cutting-edge research that advances knowledge in the brain tumor field. These findings are used to develop robust clinical trials, which improve patient outcomes through innovative combination treatments and precision medicine approaches.
As the head of the NOB's Translational Immunology Research Program and Precision Medicine Research Program, his primary research concentrations are in the areas of immunotherapy and precision therapy. For immunotherapy, he and his team are working to develop combination therapies that can increase immune cell recruitment to the tumor site and improve patient selection in clinical trials to maximize benefit for those enrolled. His Precision Medicine Research Program focuses on utilizing basic and computational biology to develop synthetic lethal drug pairs that can overcome the challenges of driver mutation targeting—and eventually offer a wider percentage of patients effective therapeutic options.
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Areas of Expertise
Information for Patients
Learn about our multidisciplinary approach to patient care, our clinical trials and the highly specialized care teams who lead them.

Mark R. Gilbert, M.D.
Clinical Trials
Research
Advances in therapeutic approaches for patients with primary brain tumors have struggled to keep pace over the last few decades, as surgery, radiation, and chemotherapy remain the staples of clinical care. As immunotherapy and precision medicine begin to become relevant in the field, Dr. Gilbert has established a robust Translational Immunology Research Program at the Neuro-Oncology Branch (NOB) with a multidisciplinary research group and accomplished clinicians that can improve treatment and patient outcomes. The goal of the NOB and Dr. Gilbert’s vision is to not only find effective treatment for brain tumors as an area of unmet need, but also to establish paradigms of clinical and translational investigation that can then be utilized worldwide to help other physicians make a collective impact in neuro-oncology.
Since heterogeneity is a hallmark of brain tumors, Dr. Gilbert’s focus for the branch has been to explore precision medicine and immunotherapy options by utilizing computational, biological, and clinical trial approaches stemming from a strong basic research program. His role in founding and establishing both the Brain Tumor Trials Collaborative (BTTC), of which NOB is the lead center, and the Collaborative Ependymoma Research Network (CERN) have been crucial foundational pillars to establish a hypothesis-driven research program that supports pre-clinical and clinical studies to advance therapies for rare cancers. BTTC and CERN are now part of large, multi-institutional networks that focus on delivering clinical trial options for malignant gliomas around the country, supported by molecular profiling, patient outcomes studies, and innovative drug discovery, among other goals.
Additionally, there are various research programs within the NOB, namely Cancer Metabolism, Molecular and Cancer Cell Biology, Cancer Stem Cell Biology, Immunology, Translational Research, and Patient Outcomes, each led by an investigator with a specific area of expertise. These programs allow for a strong translational infrastructure that can result in hypothesis-driven clinical trials, and each aims to understand the trajectory of brain tumors and provide maximal clinical benefit to the patient through therapy and patient outcomes research.
Further, with Dr. Gilbert’s guidance, the NOB strives to maintain a highly collaborative environment, spanning the clinic to the laboratory. This allows both scientists and physicians to interact and develop translational programs that can be easily carried to patients. Collaborations with bioinformatics groups, harnessing the power of effective preclinical models, and extramural relationships with other NCI branches also lends significant impact to basic research that can become clinically relevant.
Mentorship is also an extensive facet of the NOB’s activities. Visiting, post-baccalaureate, postdoctoral, and summer fellows—in addition to special volunteers and medical research scholars—assist with ongoing clinical and preclinical research programs.
Publications
A Phase II Study of Dose-Dense Temozolomide and Lapatiniv for Recurrent Low-Grade and Anaplastic Supratentorial, Infratenrial, and Spinal Cord Ependymoma
Dexamethasone-induced immunosuppression: mechanisms and implications for immunotherapy.
Randomized phase II adjuvant factorial study of dose-dense temozolomide alone and in combination with isotretinoin, celecoxib, and/or thalidomide for glioblastoma.
A randomized trial of bevacizumab for newly diagnosed glioblastoma
Dose-dense temozolomide for newly diagnosed glioblastoma: a randomized phase III clinical trial
Biography

Mark R. Gilbert, M.D.
Dr. Gilbert received his Doctor of Medicine degree from Johns Hopkins University in 1982 (Alpha Omega Alpha). He remained at Johns Hopkins to complete residencies in internal medicine (1982-1985), neurology (1984-1988), and neuro-oncology fellowship training with the Keck Foundation Fellowship (1986-1987).
He subsequently held academic research positions at Johns Hopkins University’s Department of Neurology and Oncology Center, the University of Pittsburgh Cancer Institute’s Department of Neurology, and Emory University’s Department of Neurosurgery. He then had a 14-year tenure at the Department of Neuro-Oncology at The University of Texas’s MD Anderson Cancer Center before becoming branch chief of the Neuro-Oncology Branch (NOB) at the NCI’s Center for Cancer Research.
Over the course of his career, Dr. Gilbert has been an integral part of various national and international clinical trial initiatives. Beginning in 2005, he was appointed director of the Brain Tumor Trials Collaborative (BTTC), a multi-center clinical trials consortium that is a large part of the NOB’s ongoing efforts to deliver accessible clinical trial options to patients across the country. Additionally, Dr. Gilbert is the founder, former director, and principal investigator of the Collaborative Ependymoma Research Network (CERN), a consortium formed in 2006 to study ependymoma—a rare central nervous system cancer. CERN supports basic research, clinical trials, patient outcomes research, and educational efforts in North America and Europe.
Notably, Dr. Gilbert received the Blanche Bender Endowed Professorship in Cancer Research in 2009 from the MD Anderson Cancer Center, and the National Institutes of Health Director’s Award in 2016 for his impactful contributions to the field.
Honors, Awards and Leadership
- Top Doctors - 2002-present
- Best Doctors - 2002-present
- Joel A. Gingras Jr. Award, American Brain Tumor Association - 2022
- Federal Technology Transfer Award, Cancer Center Research - 2016-2020
- National Institute of Health Director’s Award - 2016
- Award for Excellence in Clinical Research, Society for Neuro-Oncology - 2010, 2011, 2013
- Matthew T. Moore Distinguished Lecturer - 2010
- Blanche Bender Endowed Professorship in Cancer Research - 2009
- Who's Who in America - 2006
- Vice President, Society for Neuro-Oncology - 2005-2007
- Phi Beta Kappa, The Johns Hopkins University - 1982
- Alpha Omega Alpha, The Johns Hopkins University - 1982
- Scholars Program, Department of Chemistry, Brooklyn College - 1976
Societies and Initiatives
- Clinical Deputy Director, National Cancer Institute’s Center for Cancer Research (CCR)
- Co-Chair, Brain Tumor Committee in the Radiation Therapy Oncology Group (RTOG)
- Chair, Center for Cancer Research Science Board at National Cancer Institute
- Founder, former Director and Principal Investigator, Collaborative Ependymoma Research Network (CERN)
- Director, Brain Tumor Trials Collaborative (BTTC)
Professional Memberships
- Brain Tumor Trials Collaborative (BTTC)
- Collaborative Ependymoma Research Network (CERN)
- Radiation Therapy Oncology Group (RTOG)
- Society for Neuro-Oncology (SNO) Foundation Board
- Adult Brain Tumor Consortium (ABTC)
- American Academy of Neurology (AAN)
- American Association for Cancer Research (AACR)
- New Approaches to Brain Tumor Treatment Consortium (NABTT)
Job Vacancies
There are no open positions at this time. Check back again later, or take a look at CCR's Careers page.
Team
News
Mark Gilbert, M.D., Earns American Brain Tumor Association’s Joel A. Gingras Jr. Award
September 5, 2022
The Neuro-Oncology Branch chief was recognized for his work spearheading brain tumor research collaborations and expanding clinical trial access. Read more >
August 11, 2022
Summer interns investigated methods to improve immunotherapy, tailor survivorship programs, and target cancer cells using antibiotics. Read more >
Translational Immunology: Making Immunotherapy Effective for Patients with Brain Tumors
Dr. Mark Gilbert leads the Translational Immunology Program, which investigates the unique nature of the immune response in the brain to improve patient outcomes. Read more >
New Immunotherapy Study for Glioblastoma
A clinical trial investigates immune checkpoint inhibitors with standard treatment in people with aggressive brain cancers to understand the immune system response and improve long-term outcomes. Read more >