New clinical trial to study long-term progression of brain and spine cancers

A new clinical trial at the Neuro-Oncology Branch (NOB) of the Center for Cancer Research proposes to map the natural history of tumors of the central nervous system (CNS), marking the first time that a research center will conduct a large-scale, longitudinal study on patients with CNS tumors.

“We don’t have any study yet that looks at the full course of these diseases,” said Dr. Mark Gilbert, chief of the Neuro-Oncology Branch and the trial’s lead investigator. “It’s an area of unmet need.”

Longitudinal studies, in which patients are tracked for years or decades, are challenging for small research institutions because most centers don’t have the capacity to collect enough patients. The current trial, titled the “Evaluation of the Natural History of and Specimen Banking for Patients with Tumors of the Central Nervous System,” seeks to fill that void.

The NOB has already begun actively recruiting trial participants, with the goal of enrolling a total of 10,000 patients, over the age of 3 years, who have been diagnosed with a CNS cancer. NOB researchers will then collaborate with the patients’ physicians to collect and analyze patient data on parameters such as the impacts of disease on a patient’s quality of life, the molecular drivers of tumor development, the potential genetic and environmental factors that contribute to disease development, and the outcomes of various treatment interventions.

By serving as a centralized hub coordinating the flow of massive amounts of patient information, the NOB can achieve a breadth and depth of clinical analysis that smaller institutions can’t achieve on their own. It takes years to collect the critical mass of data, Gilbert said, but “if you start the clock now, then five, seven, 10 years from now we’ll have an incredible data resource.”

Anonymized data on the various CNS cancers will be made available to clinics and research institutions outside of the NOB to fine-tune and identify new treatment strategies, to design therapeutic trials, and to locate patients who may qualify for enrollment in future trials.  “This trial is one of the keys that will form the foundation of all future trials designed to discover the causes of and treatments for brain and CNS tumors,” Gilbert says.

The trial also focuses on gathering data on 10 rare CNS cancers that currently receive little research funding. These include ependymoma, adult medulloblastoma, pineoblastoma and pineal parenchymal tumor, choroid plexus carcinoma and papilloma, chordoma, gliomatosis, midline glioma, brainstem glioma, ATRT (atypical teratoid rhabdoid tumor), and malignant and atypical meningioma. Because of their rarity, the trial researchers only expect to recruit between 2 and 12 patients per year for each rare CNS cancer on the list into the trial, but the pace or recruitment is still much more rapid than could be achieved by smaller institutions alone.

The long term goal of the trial, which follows participants from diagnosis through the entire course of their disease, “is a better understanding of the spectrum of diseases we see in neuro-oncology,” Gilbert said. This longitudinal approach to studying CNS cancer development promises a much more thorough understanding of these diseases and better insight into future treatments.