Headshot of Mark Gilbert

Mark R. Gilbert, M.D.

  • Center for Cancer Research
  • National Cancer Institute
Neuro-Oncology Branch


As the former Neuro-Oncology Branch (NOB) chief from 2014 to 2024, Dr. Gilbert developed a unified team to perform cutting-edge research that advances knowledge in the brain tumor field. These findings were used to develop robust clinical trials, which improved patient outcomes through innovative combination treatments and precision medicine approaches. 

Today, Dr. Gilbert is a scientist emeritus. In his previous role as chief and senior investigator, he led the NOB's Translational Immunology Research Program and Precision Medicine Research Program. His primary research concentrations were in the areas of immunotherapy and precision therapy. For immunotherapy, he and his team worked to develop combination therapies that 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 focused on utilizing basic and computational biology to develop synthetic lethal drug pairs that can overcome the challenges of driver mutation targeting—offering a wider percentage of patients effective therapeutic options.

Explore the NOB's Research Programs >

Areas of Expertise

Rare Tumors
Cancer Immunology and Immunotherapy
Brain and Spine Cancer
Combination Immunotherapy
Cancer Patient Outcomes


Routes for leukocyte trafficking in the CNS
Three main routes have been described for leukocyte trafficking from the blood vasculature to the brain. A parallel lymphatic system allows for drainage of cerebrospinal fluid and interstitial fluid to the cervical lymph node. 

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. In his previous role as a senior investigator at the Neuro-Oncology Branch (NOB), Dr. Gilbert established a robust Translational Immunology Research Program with a multidisciplinary research group to improve treatment and patient outcomes. Dr. Gilbert’s vision was to not only find effective treatments for brain tumors, but to also establish paradigms of clinical and translational investigation that can then be utilized worldwide to help other physicians make a collective impact in neuro-oncology.

Because heterogeneity is a hallmark of brain tumors, Dr. Gilbert’s focus was exploring precision medicine and immunotherapy options by utilizing computational, biological, and clinical trial approaches stemming from a strong basic research program. His roles in founding and establishing the Brain Tumor Trials Collaborative (BTTC) and the Collaborative Ependymoma Research Network (CERN) were crucial foundational pillars to establish a hypothesis-driven research program that supported pre-clinical and clinical studies to advance therapies for rare cancers. The 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.


Selected Recent Publications

A Phase II Study of Dose-Dense Temozolomide and Lapatiniv for Recurrent Low-Grade and Anaplastic Supratentorial, Infratenrial, and Spinal Cord Ependymoma

Gilbert MR, Yuan Y, Wu J, Mendoza T, Vera E, Omuro A, Lieberman F, Robins HI, Gerstner ER, Wu J, Wen PY, Mikkelsen F, Aldape K, Armstrong TS
Neuro-Oncology. 23(3): 468-477, 2021. [ Journal Article ]

Dexamethasone-induced immunosuppression: mechanisms and implications for immunotherapy.

Giles AJ, Hutchinson MND, Sonnemann HM, Jung J, Fecci PE, Ratnman NM, Zhang W, Song H, Bailey R, Davis D, Reid CM, Park DM, Gilbert MR
Journal of Immunotherapy. 6(1): 51, 2018. [ Journal Article ]

Randomized phase II adjuvant factorial study of dose-dense temozolomide alone and in combination with isotretinoin, celecoxib, and/or thalidomide for glioblastoma.

Penas-Prado M, Hess KR, Fisch MJ, Lagrone LW, Groves MD, Levin VA, De Groot JF, Puduvalli VK, Colman H, Volas-Redd G, Giglio P, Conrad CA, Salacz ME, Floyd JD, Loghin ME, Hsu SH, Gonzalez J, Chang EL, Woo SY, Mahajan A, Aldape KD, Yumg WK, Gilbert MR, MD Anderson Community Clinical Oncology Program, Brain Tumor Trials Collaborative
Neuro-Oncology. 17(2): 266-73, 2015. [ Journal Article ]

A randomized trial of bevacizumab for newly diagnosed glioblastoma

Gilbert MR, Dignam JJ, Armstrong TS, Wefel JS, Blumenthal DT, Vogelbuam MA, Colman H, Chakravarti A, Pugh S, Won M, Jeraj R, Brown PD, Jaeckle KA, Schiff D, Stieber VW, Brachman DG, Werner-Wasi M, Tremont-Lukats I, Sulman EP, Aldape KD, Curran WJ, Mehta MP
New England Journal of Medicine. 370(8): 699-708, 2014. [ Journal Article ]

Dose-dense temozolomide for newly diagnosed glioblastoma: a randomized phase III clinical trial

Gilbert MR, Wang M, Aldape KD, Stupp R, Hegi ME, Jaeckle KA, Armstrong TS, Wefel JS, Won M, Blumenthal DT, Mahajan A, Schultz CJ, Erridge S, Baumert B, Hopkins KI, Tzuk-Shina T, Brown PD, Chakravarti A, Curran WJ, Mehta MP
Journal of Clinical Oncology. 31(32): 4085-91, 2013. [ Journal Article ]


Three neuro-oncology providers smile outside

Forming Connections to Address Rare Brain Tumor Treatment and Care

May 21, 2024

Scientists and neuro-oncology providers are taking a comprehensive approach to improve care and treatment—performing laboratory research, conducting clinical trials, and fostering a sense of community. Watch Now >

Group photo of people smiling outside with Dr. Gilbert in the center

Celebrating CCR Careers: Mark Gilbert, M.D.

April 23, 2024

After nearly a decade at the Center for Cancer Research as chief of the Neuro-Oncology Branch, Dr. Mark Gilbert is announcing his retirement. Read more >

Teenage girl speaking to a doctor

Understanding the Unique Barriers Adolescents and Young Adults with CNS Cancers Face in Clinical Care

January 30, 2024

In a new systematic review, researchers identify the specific challenges that make it difficult for this patient population to enroll in clinical trials and seek specialized treatment. Read more > 

Headshot of Mark Gilbert

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 > 

Headshots of the NOB's three summer interns

Translational Research Immersion Program Interns Share Their Projects at the Annual Summer Poster Day

August 11, 2022

Summer interns investigated methods to improve immunotherapy, tailor survivorship programs, and target cancer cells using antibiotics. Read more >

Headshot of Dr. Gilbert in lab coat

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 >

Nurse taking blood from patient

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 >

Hand holding pills and cup of water

Oral Drug Combination Shows Benefit for Adults with Ependymoma

Learn how results from the first prospective clinical trial for adults with ependymoma led to a change in treatment guidelines, offering hope to control the disease and improve symptoms. Read more >

Illustration showing the progression of tumor cells from the tumor core (CT) to the leading edge of the tumor (LE) through an intermediate state (IT), towards a proliferating state (CTmvp) close to vasculature or a necrotic state (CTpan).

Exploratory Adaptation Explains How Glioblastoma Cells Adapt and Survive 

February 5, 2020

Dr. Orieta Celiku and Dr. Mark Gilbert used bioinformatics and mathematical modeling to find tools to explain how glioblastoma cells may adapt to survive in stressful conditions. Read more >

Dr. Mark Gilbert and Dr. Nivi Ratnam

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. Read more >

Drawing of the inside of the brain showing the supratentorium (the upper part of the brain) and the infratentorium (the lower back part of the brain).

Clinical Trial Tests a New Drug Therapy for Recurrent Ependymomas of the Brain and Spinal Cord

May 28, 2019

Dr. Mark Gilbert, chief of the Neuro-Oncology Branch, is leading a trial of marizomib, a drug that targets ependymomas that have a specific genetic signature by slowing the spread of cancer cells. Read more >

Nanoparticles in the brain

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

February 4, 2019

Dr. Mark Gilbert is leading a trial of the cancer drug sunitinib for recurrent gliosarcoma and previously treated sarcomas of the CNS, which comprises the brain and spinal cord. Read more >