Defining Spinal Ependymoma: A New CERN-Funded Effort to Clarify Diagnosis and Improve Care
— Categories: CERN Research Articles Ependymoma Research Articles Press Release Ependymoma Community Posted on February 24, 2026
This year, the Collaborative Ependymoma Research Network (CERN), a program of the National Brain Tumor Society, is launching a new multi-year research project designed to address one of the most persistent gaps in the field: how spinal ependymomas are defined, classified, and understood at the molecular level. The project, Creating a Reference Landscape for Spinal Ependymoma, is led by Eric Holland, MD, PhD, at Fred Hutchinson Cancer Center, with international collaboration to ensure sufficient data for this rare disease.
Ependymoma is a rare tumor of the brain and spinal cord that occurs in both children and adults. While spinal tumors are more common in adults, they also affect pediatric patients. On paper, spinal ependymoma often appears to have a relatively favorable five-year survival rate—but that statistic masks a highly variable reality. Some patients experience long periods of stability. Others face repeated recurrences, progressive neurological impairment, chronic pain, or disease spread. The difference between these trajectories often comes down to tumor biology, not just tumor location.
Over the past two decades, advances in molecular profiling have transformed how ependymomas are diagnosed. These efforts culminated in major updates to the World Health Organization (WHO) classification in 2021, which now recognizes multiple biologically distinct ependymoma subgroups.
Today, within the broad spinal ependymoma category, there are currently four distinct subgroups:
- Spinal subependymoma (grade 1),
- Spinal myxopapillary ependymoma (grade 2),
- Spinal ependymoma (grade 2 or 3), and
- Spinal ependymoma with MYCN amplification (grade 3).
CERN supported the work of Dr. Kristian Pajtler, Dr. Mark Gilbert and Dr. Terri Armstrong that led to the recent identification of the subgroup of spinal ependymoma with the MYCN amplification. This discovery was a critical finding as it was observed that patients with this subtype tend to experience a more aggressive disease with worse overall- and progression-free survival compared to other types of ependymoma. This demonstrates the importance of an accurate diagnosis. These advancements in disease classification and diagnosis were milestone changes for ependymoma patients changing how patients are diagnosed and affecting treatment decisions.
Despite this progress, ependymoma that originates in the spinal cord remains incompletely understood and defined, even compared to those that occur in the brain. “After 20 years of providing personalized support and navigation to ependymoma families, I can tell you the experience of our spinal survivors is remarkably different and brings its own set of unique challenges and hardships,” said Kim Wallgren, Executive Director of CERN. “Being equipped with the proper diagnostic information that can help shape decisions for care and awareness of severity of disease, is invaluable to families. With some frequency, patients are told not to worry and to consider themselves ‘lucky’ to have a disease as the overall survival rate is much higher than other CNS tumors. Even today, some people are not diagnosed according to the latest WHO criteria and are not offered proper support and care from a neuro-oncology clinic. I can tell you wholeheartedly that our spinal ependymoma survivors go through a great deal of unseen suffering and oftentimes, a significant amount of pain and unmet needs through the trajectory of their illness.”
To address this issue, CERN has announced a new grant to Dr. Eric Holland at Fred Hutchinson Cancer Center to fund a multi-year project that will analyze spinal ependymoma samples on a molecular level and generate data sufficient to better define, classify, diagnose, and potentially treat patients with these tumors. A specific focus for this project will be myxopapillary ependymoma, as this subtype is still one of the most mischaracterized of all the subtypes.
“New technologies are allowing us to use…tumor samples from pathology archives to divide diagnosis into distinct subgroups based on their biology,” said Dr. Holland. “This is especially important for rare tumors where an informative population is spread across multiple sites and across time. With samples from across the country and globe, we aim to determine how many different spinal ependymomas subtypes there are and what drives them.”
Dr. Holland was selected after CERN Executive Director, Kim Wallgren convened a small group of international experts at the Society for Neuro-Oncology Annual Meetings in 2024 to create a plan to tackle this important issue. Through continued discussions, Dr. Holland emerged as a leader with access to key technology and keen interest to focus on the issues. He’s a world renowned researcher with recognized expertise in rare cancers and experience in ependymoma specifically. He previously was awarded a CERN grant in 2016 that later led to the multimillion dollar grant from the National Cancer Institute for ependymoma work in 2020.
Dr. Holland and collaborators will seek to analyze spinal ependymoma tumor samples using RNA sequencing, a technique that provides a comprehensive snapshot of tumor biology. The study will determine whether tumors – again, particularly myxopapillary ependymoma – currently grouped together are truly the same disease—or whether important biological differences have been obscured by older diagnostic methods. These insights may ultimately identify additional subtypes (i.e., it is suspected, but needs to be confirmed, that the group of tumors currently considered one uniform group of myxopapillary ependymoma may in fact better be defined as two separate, unique subgroups) which can then help generate more targeted potential treatment approaches and optimize patient care. The analysis may also uncover misdiagnosis that would have important patient management implications.
“I think what is incredibly important here is the holistic role advocacy is actively playing in the formation and launch of this research project in addition to being the sole financial supporter of the project,” said Wallgren. “Having a rare tumor doesn’t mean we should avoid investing in research to further understand and define the disease. However, the reality is it takes a concerted and designated effort to fuel momentum in the overlooked disease. The team at National Brain Tumor Society feels privileged to partner with Dr. Holland and other international leaders to advance our understanding of spinal ependymoma on behalf of the entire community.”
Because spinal ependymoma is rare, no single institution can collect and generate enough data or answer these questions alone. This project is intentionally structured as an international, collaborative effort, partnering with Dr. Ulrich Schuller at the University Medical Center in Hamburg Germany, to openly share data and accelerate progress across the field. This work exists because of advocacy-driven investment. It builds directly on CERN’s mission to define the biology of ependymoma so that meaningful treatments can follow—particularly for patients whose needs have historically been overlooked. CERN remains the most influential and impactful advocacy force focused on defining the biology of ependymoma to uncover potential therapeutic opportunities for the future. CERN funded the hallmark Nature publication establishing the initial nine subgroups, supported the efforts refining the ZFTA-RELA fusion, contributed to the research that defined the newly added MYCN amplification subgroup, and is now responsible for setting out to define myxopapillary ependymoma once and for all.
“This project means a lot to our community. If we don’t invest in this project, we could go another ten years without understanding the disease and continuing to diagnose the disease based on old and less than accurate methods,” continued Wallgren. “We can’t let that stagnation happen. The critical nature of dedicated support to the ependymoma fund at NBTS is evident and imperative in order for progress to happen.”