Mark Gilbert, M.D.
Mark Gilbert, M.D., has been a neuro-oncologist for nearly four decades, working to find better treatments for people with rare brain and spine tumors. He will retire at the end of May, after nearly a decade at CCR as Chief of the Neuro-Oncology Branch (NOB).
Gilbert obtained his M.D. in 1982 from Johns Hopkins University. After appointments at Hopkins, the University of Pittsburgh and Emory University, Gilbert moved to The University of Texas’s MD Anderson Cancer Center, serving as a professor and deputy chair of the Department of Neuro-Oncology.
Gilbert became chief of CCR’s NOB in 2014, revitalizing the branch after a long period without leadership and pursuing his research interest of examining the unique nature of the immune response in the brain. In 2017, he co-launched NCI-CONNECT (Comprehensive Oncology Network Evaluating Rare CNS Tumors) with Terri Armstrong, Ph.D. NCI-CONNECT is a program within CCR’s Rare Tumor Patient Engagement Network supported by the Cancer Moonshot℠, which advances care and treatment for rare central nervous system (CNS) cancers. Gilbert also took on additional leadership roles as the Chair of the CCR Scientific Review Board, a CCR Deputy Scientific Director and an Adjunct Investigator at the National Institute of Neurological Disorders and Stroke.
Throughout his career, in addition to his laboratory work, Gilbert spearheaded over 30 clinical trials — including two of the largest neuro-oncology studies in the world to date. He was inspired by the CCR “true translational model,” where laboratory discoveries are transferred to the clinic and clinical observations are evaluated in the laboratory in a seamless, collaborative process.
Gilbert has earned multiple accolades, including the MD Anderson Cancer Center’s Blanche Bender Endowed Professorship in Cancer Research, the Society for Neuro-Oncology (SNO) Award for Excellence in Clinical Research, the National Institute of Health Director’s Award and the American Brain Tumor Association’s Joel A. Gingras Jr. Award. He also served in leadership roles for SNO from 2005 to 2016. Gilbert authored nearly 450 peer-reviewed research papers in prestigious publications, such as The New England Journal of Medicine and the Journal of Clinical Oncology.
In the Q&A that follows, Gilbert reflects on his career highlights, his efforts to find new collaborative approaches to clinical trials and the patients he has seen over the years.
What made you decide to be a neuro-oncologist?
While I was a medical student at Johns Hopkins, I joined the lab of Dr. Richard Humphrey, a respected researcher who studied multiple myeloma, a type of blood cancer. I would “round” with him to see patients. From that point on, I was sure I wanted to pursue oncology.
About three weeks before I was about to graduate from medical school, I received a call on my answering machine. It was from the secretary of the chairman of neurology at Johns Hopkins, where I had recently finished a clinical rotation. I was asked to meet with the chairman as soon as possible. Fearing the worst, I stayed awake all night and arrived early to his office the next morning.
As it turns out, he asked if I was interested in a career in neurology. I told him I'd matched at Johns Hopkins for internal medicine and really wanted to be an oncologist. That’s when he mentioned this new field called neuro-oncology that combines neurology and oncology. The rest is history.
Much of your research has focused on immunotherapy and precision medicine to treat brain and spine tumors. What are the most exciting frontiers in these areas?
I hope to see continued efforts to bring precision medicine into cancer immunology. An exciting development will be utilizing cutting-edge molecular technologies to better characterize tumors. Researchers will leverage advanced informatics to identify targets in an individual patient's tumor and predict the patient's prognosis more accurately. That will allow us to conduct informed clinical trials and determine whether a particular immunotherapy will work for a patient.
Why is brain tumor research an area of unmet need — and how does the NCI-CONNECT program help to fill that need?
There are very few clinical trials for patients with brain and spine tumors, because it can be hard to accrue enough participants to answer scientific questions.
NCI-CONNECT allowed us to establish partnerships and networks to develop clinical trials and accrue patients faster. Since its beginning, the program has launched 13 clinical studies. Our work led to updating two National Comprehensive Cancer Network (NCCN) Guidelines for rare diseases — one for recurrent ependymoma and one for medulloblastoma. We also helped identify a new ependymoma subtype, which was added to the 2021 WHO Classification of CNS Tumors.
In many instances, we don't know the best treatment because we don’t know enough about the cancer biology, especially in the case of rare tumors. NCI-CONNECT is helping to fill this knowledge gap.
What have you learned from your patients?
Throughout my career, I have taken care of over 5,000 people with CNS cancers. In doing so, I have met some of the bravest, strongest, and most wonderful people. They have each risen to the task of facing a terrible disease — almost always with incredible support from their families. They taught me a lot about remaining positive and kind even when life throws obstacles your way. They are truly inspirational.
What is the importance of mentorship in science, and what role has it played in your career?
Mentorship is important for so many reasons. Young scientists and clinicians benefit from role models who can provide guidance and a broader perspective. Seeing how your mentors handle various situations will also help inform your core values and build your persona. I’ve had many mentors throughout my career. Some were fellow scientists and healthcare providers, while others were philanthropists.
What advice would you give someone entering the neuro-oncology field today?
I would tell those entering the field that it's going to take a collaborative effort, both nationally and internationally, to make significant advances. It will require outstanding science, ranging from the most basic laboratory work to translational research and patient outcomes investigations. This multidisciplinary approach will be key to future innovations in care and treatment.
What are you looking forward to most in your retirement?
During my retirement, I’m looking forward to exploring new adventures. I hope to reflect on where my experience and expertise can be most impactful. I’ll also continue to communicate with my CCR colleagues — and watch with pride as they continue to push intellectual boundaries and design comprehensive clinical trials that incorporate patient outcomes measures. The future is bright. I hope to leave behind a legacy of integrating laboratory and clinical research that leads to exceptional patient care.
Dr. Mark Gilbert will retire on May 31, 2024.