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Headshot of Mark Gilbert

Mark R. Gilbert, M.D.

  • Center for Cancer Research
  • National Cancer Institute
NIH Scientist Emeritus
Neuro-Oncology Branch

RESEARCH SUMMARY

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
Ependymoma
Cancer Patient Outcomes

Research

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(link is external) (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.

Publications

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 ]