Embryonal tumor with multilayer rosettes (ETMR) is one of the most challenging and complex brain tumor diagnoses for a physician to manage. Whether fresh out of fellowship or a seasoned pediatric neuro-oncologist, each ETMR patient we treat presents a unique set of circumstances that will test the limits of our clinical expertise. As a relatively new clinical entity with little guiding medical literature, ETMR appears to be a locked box. The key to solving this rare tumor is collaboration.
Enabling Open Access
The rarity of ETMR makes dedicated, large multi-institution clinical trials extremely difficult to carry out. At the same time, we believe that patient access to cutting-edge ETMR care should not be limited to only select institutions, certain states, or even countries. As an international registry, ETMR One opens the door for collaboration across hospitals and across borders, allowing physicians to research and refine a consensus therapy for ETMR and partner together to deliver comprehensive, complex medical care to each individual patient.
Standardizing Biology-driven Treatment
To date, the literature regarding the clinical treatment and outcomes of children ETMR is extremely limited. Interpreting this data is challenging as the information is retrospective, often incomplete, and involves a variety of therapeutic regimens. While the aggressive nature of ETMR warrants an equally aggressive therapeutic approach, historical data suggests that extensive surgical resection, radiotherapy, and high-dose chemotherapy are not sufficient treatment in the vast majority of cases.
As physicians trusted with the lives of these children, we cannot accept the poor outcomes of past regimens and continue this same pattern of care. If we want to see a change occur, we must create that change ourselves. A collaborative, large-scale effort aimed at discovering an effective treatment standard for ETMR must be carried out. The ETMR One consensus protocol is our approach to delivering a biology-based treatment regimen, built by leading experts and based on the latest available medical literature, to our children with ETMR.
Accelerating Innovative Research
Perhaps the largest impediment to discovering breakthrough treatments for ETMR is the scarcity of viable ETMR tumor models. Every ETMR tumor specimen is precious and, when used properly, can bring us one step closer to finding a cure. Through coordinated biobanking and genomic sequencing of participant tumor tissue, we hope to accelerate pre-clinical research of ETMR. Specimens collected through the registry will be used to develop ETMR cell lines and transplantable tumor models which will be made available to dedicated researchers from around the globe.