Mark R. Gilbert, M.D.

Senior Investigator

Neuro-Oncology Branch


Building 82, Room 235A
Bethesda, MD 20892


Research Topics

My focus has been in the area of clinical research, emphasizing both large, multi-institutional studies for malignant gliomas as well as smaller clinical trials that are designed to advance therapies for less common cancers.  I was the Director of Clinical Research in the Department of Neuro-Oncology from 2000-2007 and was the Director of the Clinical Research Core for the ongoing Brain Tumor SPORE grant at M. D. Anderson.  I recently moved to the National Institutes of Health where I serve as the Chief of the Neuro-Oncology Branch.  I have recently been named the Co-Chair of the Brain Tumor Committee in the Radiation Therapy Oncology Group (RTOG).  In addition to my increasing leadership role, I am serving on several important clinical trials within the RTOG.  The RTOG 0525 study accrued nearly 1200 patients and will determine whether a dose-dense schedule of temozolomide is therapeutically superior to standard dosing.  Patient stratification required upfront tumor tissue to determine MGMT methylation status.  The study includes a quality of life component including longitudinal measures of symptom burden and neurocognitive function.  RTOG 0625 was a randomized study of bevacizumab and irinotecan versus bevacizumab and temozolomide for patients with recurrent glioblastoma.  This led to RTOG 0825 evaluating the role of bevacizumab for newly diagnosed glioblastoma which completed accrual in 2011 with 975 patients. Upfront tumor collection and molecular stratification, in addition to the placebo-control design and expansion of the quality of life studies signify advances in clinical trial opportunities in the field. Both 0525 and 0825 provide outstanding opportunities for correlative studies.  The results of RTOG 0825 were presented at ASCO in the plenary session in 2013 and were published in the New England Journal of Medicine in 2014.

I have also been involved in the creation of two multicenter clinical trial efforts.  Eight years ago, with support from philanthropy, I established the Brain Tumor Trials Collaborative (BTTC), a 24 center consortium that is geographically distributed in the United States and designed to do creative, mid-range (100 -250 patient) clinical trials.  This group completed accrual to a 176 patient, 8 arm randomized study using a factorial design.  A second study completed accrual with 115 patients testing an alternative adjuvant treatment for poor prognosis patients with glioblastoma. Ongoing studies include those with novel trial designs, such as adaptive randomized design, novel treatment for bevacizumab refractory glioblastoma and a randomized double blinded placebo controlled trial of prophylactic anticonvulsants in patients with malignant gliomas.

I am also the founder and former leader of the Collaborative Ependymoma Research Network (CERN).  This consortium, supported by philanthropy, to focus basic and clinical research efforts on this uncommon adult cancer.  Five projects ranging from clinical trials, tumor molecular profiling – patient outcomes study, drug discovery by both screening and novel drug synthesis, tumor model creation, exploration of ependymoma stem cells and patient outcomes studies are all ongoing. The CERN recently expanded beyond North America and is now collaborating with centers in Europe. Three clinical trials are now ongoing.


Dr. Gilbert received his medical doctorate (M.D.) degree in medicine from Johns Hopkins University (JHU) in 1982 (Alpha Omega Alpha).  At JHU, he completed residencies in internal medicine (1982-1985) and neurology (1984-1988) and fellowship training (Keck Foundation Fellowship) in both neurology and neuro-oncology.  In 2000, after serving on the faculties of the University of Pittsburgh and Emory University in Atlanta, Georgia, Dr. Gilbert joined the University of Texas MD Anderson Cancer Center as Deputy Chair of the Department of Neuro-oncology.  In 2005, he was appointed as Director of the Brain Tumor Trials Collaborative, a multi-center clinical trials consortium.   In 2009, Dr. Gilbert received the Blanche Bender Endowed Professorship in Cancer Research.  Recently, he was named the Co-Chair of the Brain Tumor Committee in the Radiation Therapy Oncology Group (RTOG).  Dr. Gilbert is also the founder and former leader of the Collaborative Ependymoma Research Network (CERN), a consortium studying ependymoma, a rare central nervous system cancer, by supporting basic research, clinical trials, patient outcomes research and educational efforts in North America and Europe.

Honors, Awards and Leadership

Award for Excellence in Clinical Research, Society for Neuro-oncology (2011, 2010)
Matthew T. Moore Distinguished Lecturer (2010)
Who's Who in America (2006)
Vice President, Society for Neuro-oncology (2005-2007)
Top Doctors (2002-2009)
Best Doctors (2002-2009)
Phi Beta Kappa, The Johns Hopkins University (1982)
Alpha Omega Alpha, The Johns Hopkins University (1982)
Scholars Program, Department of Chemistry, Brooklyn College (1976)

Selected Publications

  1. Gilbert MR, Dignam JJ, Armstrong TS, Wefel JS, Blumenthal DT, Vogelbaum MA, Colman H, Chakravarti A, Pugh S, Won M, Jeraj R, Brown PD, Jaeckle KA, Schiff D, Stieber VW, Brachman DG, Werner-Wasik M, Tremont-Lukats IW, Sulman EP, Aldape KD, Curran WJ Jr, Mehta MP. A randomized trial of bevacizumab for newly diagnosed glioblastoma. N Engl J Med. 2014;370(8):699-708.

  2. Gilbert MR, Gonzalez J, Hunter K, Hess K, Giglio P, Chang E, Puduvalli V, Groves MD, Colman H, Conrad C, Levin V, Woo S, Mahajan A, de Groot J, Yung WK. A phase I factorial design study of dose-dense temozolomide alone and in combination with thalidomide, isotretinoin, and/or celecoxib as postchemoradiation adjuvant therapy for newly diagnosed glioblastoma. Neuro Oncol. 2010;12(11):1167-72.

  3. Gilbert MR, Kuhn J, Lamborn KR, Lieberman F, Wen PY, Mehta M, Cloughesy T, Lassman AB, Deangelis LM, Chang S, Prados M. Cilengitide in patients with recurrent glioblastoma: the results of NABTC 03-02, a phase II trial with measures of treatment delivery. J Neurooncol. 2012;106(1):147-53.

  4. Armstrong TS, Cao Y, Scheurer ME, Vera-Bolaños E, Manning R, Okcu MF, Bondy M, Zhou R, Gilbert MR. Risk analysis of severe myelotoxicity with temozolomide: the effects of clinical and genetic factors. Neuro Oncol. 2009;11(6):825-32.

  5. Hegi ME, Liu L, Herman JG, Stupp R, Wick W, Weller M, Mehta MP, Gilbert MR. Correlation of O6-methylguanine methyltransferase (MGMT) promoter methylation with clinical outcomes in glioblastoma and clinical strategies to modulate MGMT activity. J Clin Oncol. 2008;26(25):4189-99.

This page was last updated on September 12th, 2018