Mark Gilbert

March 2019

Mark Gilbert

CERN Advisor Reports on Clinical Trials and Advances in Care

As the CERN Foundation continues to evolve to better serve the ependymoma community, so do its members. In November 2014, CERN Advisor Mark Gilbert, M.D., was named senior investigator and chief of the Neuro-Oncology Branch in the Center for Cancer Research, National Cancer Institute (NCI), at the National Institutes of Health (NIH). There, Dr. Gilbert continues to conduct clinical trials, measure patient outcomes and develop best practices in patient care.

He also continues his collaborative and translational research focused on finding new treatments for patients with primary brain and spine tumors. Specifically, in November 2018, he presented the final report of the CERN 08-02 study at the Society for Neuro-Oncology Annual Scientific Meeting. The phase II study of dose-dense temozolomide and lapatinib is the first prospective clinical trial in adult patients with ependymoma.

“The results are very dramatic,” Dr. Gilbert said. The report showed that the drug combination had objective responses and prolonged progression-free survival. For patients with spine tumors, improvements in performance status, disease-related symptoms and symptom interference was seen. For patients with brain tumors, improvements in performance status and stability of most disease-related symptoms was reported.

“These results suggest that the combination temozolomide-lapatinib has both anti-cancer activity and clinical benefit in adult patients with recurrent ependymoma,” Dr. Gilbert said. “It should be considered as a standard salvage regime for adults with recurrent ependymoma.”

At NIH, Dr. Gilbert is also continuing the CERN 09-02 adult ependymoma clinical trial studying the chemotherapy drug Carboplatin in conjunction with the drug Bevacizumab (Avastin). The first phase of the trial is a preliminary assessment of effectiveness in over 20 patients. If this phase is effective, a second study will be initiated. The trial is accruing patients at four participating CERN sites, including NIH, Memorial Sloan Kettering Cancer Center, Harvard Cancer Center and the University of Pittsburgh Cancer Institute.

The studies are part of a larger initiative led by Dr. Gilbert and Terri Armstrong, Ph.D., senior investigator and deputy chief of the Neuro-Oncology Branch at NIH, to focus research efforts on rare central nervous system (CNS) cancers in adults. “We recognized an unprecedented opportunity to focus our research efforts with funding from the Cancer Moonshotâ„ ,” Dr. Gilbert said.

In October 2017, Drs. Gilbert and Armstrong launched NCI-CONNECT (Comprehensive Oncology Evaluating Rare CNS Tumors) to study 12 rare adult CNS cancers, including ependymoma. This new effort was inspired by their experience with CERN, which provided a model for a collaborative program, Dr. Gilbert said.

“Our approach is to connect basic science research to patient outcomes research and scientifically-based clinical trials. With the aim of establishing better therapies and standard of care. This model could have a huge impact,” Dr. Gilbert said.

NCI-CONNECT has expanded the original ependymoma outcomes project to study outcomes and risk in patients with rare CNS cancers. The project led by Dr. Armstrong aims to improve the understanding of outcomes and risk factors related to the occurrence of rare CNS cancers. Patients participating complete a web-based survey and submit a saliva sample to be used for genetic testing.

NCI-CONNECT is also conducting a Natural History Study to better understand rare CNS cancers and areas for further research. This study includes an evaluation at NIH and submitting a tumor tissue sample. Dr. Gilbert is also investigating the immunotherapy drug nivolumab as an effective treatment for people with rare CNS tumors, including ependymoma.

NCI-CONNECT Partnerships to Advance Care

The CERN Foundation is part of another important piece of NCI-CONNECT: Partnering with non-profit organizations to overcome the challenges of reaching patients. CERN works with NCI-CONNECT to identify and engage patients with ependymoma by disseminating information about clinical trials and new approaches to improve care and treatment.

“It’s difficult to complete studies to better understand CNS cancers because there are few patients for clinical trials and few tumor samples to study,” Dr. Gilbert said. “We value our relationships with non-profit organizations because they can help identify patients who would benefit from our program activities.”

NCI-CONNECT also plans to develop web-based educational content that will be shared by the CERN Foundation for physicians, nurses and researchers, and collaborate closely with them. Community-based providers also have the opportunity be part of a larger network of investigators who collaborate to advance treatments for patients with brain and spine tumors.

Importantly, over the past 32 years as a neuro-oncologist, Dr. Gilbert said he has learned that every patient is unique. When he is helping patients navigate their illness, the trivial things seem really trivial. “I have gained a unique perspective about life,” Dr. Gilbert concludes.

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