About the Study

International Research for Children Diagnosed with Embryonal Tumor with Multilayer Rosettes (ETMR)

ETMR One is an international registry and research platform for children diagnosed with embryonal tumor with multilayer rosettes (ETMR).

The project has the following aims:

• To collect the epidemiologic, clinical, and molecular data of patients with ETMR.
• To evaluate tumor responses and patient outcomes using expert consensus therapy.
• To obtain tumor tissue for research of new biology-based therapies for children with ETMR.
• To provide patient families with guidance and access to clinical trials for ETMR.

Our Mission

Our goal is to improve patient outcomes through the development of innovative therapy based on the most up-to-date medical literature surrounding ETMR.

ETMR is a rare and highly aggressive brain tumor which occurs almost exclusively in young children and has an extremely poor prognosis. To date, only a few hundred cases of ETMR have been reported. Due to rarity of this tumor, no large clinical trials have been carried out to determine the best therapy for ETMR. From the cases that have been reported, we know that extensive surgery, radiation, and high-dose chemotherapy are not sufficient treatment for most children. Newer, better treatment approaches are desperately needed for ETMR.

To that end, we aim to develop and support further research focused on delivering more effective, biology-based therapy to children with ETMR using the following approach:

1. The Registry

   The registry is a collection of clinical information from ETMR patients from across the globe which includes their demographic data, treatments received, responses to therapy, imaging studies, and available tumor specimens. This information is being collected as a resource for physicians and researchers to improve our understanding of ETMR and develop better approaches to treatment and overall care of children with ETMR.

2. Consensus Therapy

   The physicians of patients enrolled into the registry will receive recommendations for a consensus therapy plan derived from the ETMR medical literature, the experience of leading pediatric neuro-oncologists, and clinical data derived from other infant brain tumor protocols. The consensus therapy plan is not considered to be experimental, but rather a recommendation from available experts. While not required, physicians of registry participants are encouraged to follow the consensus therapy to help gather uniform outcome data and optimize the treatment for children with ETMR.

3. Medical Advisory Board

   The management of patients with ETMR is complex and requires multidisciplinary expertise in the fields of pediatric neuro-oncology, pediatric neurosurgery, neuropathology, neuroradiology, and radiation oncology. Our medical advisory board is available as a resource for physicians and families. The board reviews the case of every registry participant and provides expert guidance for the clinical management of each individual patient.

4. Tumor Specimen Collection

   To date, the majority of ETMR laboratory research has been performed using a single cell line that is unlikely to be representative of all ETMR cases. Using the tumor specimens collected from the registry, we aim to produce multiple ETMR cell lines that will allow potential therapies to be tested against a range of ETMR models. The data obtained from studies will be used to improve the consensus protocol therapy or used to generate additional clinical trials for ETMR.

5. Clinical Trial Referrals

   There is a tremendous need for further clinical research aimed at improving outcomes in children with ETMR. Conducting clinical trials in rare tumors is highly challenging, with patient accrual being perhaps the largest hurdle to overcome. The ETMR One registry will aid in identifying eligible study participants and provide a pathway for enrollment onto relevant clinical trials.