A Global Platform for Translational Research in Rare Pediatric Brain Tumors
Abstract
Objective and Rationale: Brain cancers are as common as leukemia in children, but treatment for brain cancers have been less effective. They cause the greatest number of deaths and disability due to cancer-related treatment. Pediatric brain cancer is not one disease, but a collection of many different diseases. Rare types of brain cancer make up 40% of the most common category of pediatric brain cancers called embryonal brain tumors (EBTs), which can spread to the brain and spinal cord and requires intensive therapy. Treatment for these aggressive cancers is very challenging as there is very limited research and data to guide treatment, and most children with rare EBTs are young (0-4years) and developmentally fragile. The RBTC (Rare Brain Tumor Consortium) was formed in 2002 with the mission to create a worldwide collaborative clinical database and biobank to fill this long-standing lack of data and information for families and clinicians faced with a rare brain tumor diagnosis. The RBTC is now a collaborative of 180+ centers, in 40+ countries. RBTC houses a unique, and one of the largest resources dedicated to rare brain tumors which has enabled world leading research that has improved recognition, diagnosis, and management of these patients that are rare even in high-volume hospitals. In this proposal, our goal is to maintain, grow, and diversify RBTC data and biomaterials, improve access to stimulate global research worldwide, and create an information portal for families/patients. We will future-proof these unique global resources by updating our platform. We will increase collection of new types of biomaterials (e.g., spinal fluid) and tissues from recurrent cancer or postmortem tissue to support innovative science and technologies with the mission to cure children with minimal harm - powerfully guided by parent advocates and partners. Applicability and Advancing Rare Cancer Research: Families and physicians have long faced the frustrating lack of information and studies on rare brain cancers. Over the last 20 years, the efforts of the RBTC global collaborative have organized and generated large amounts of clinical and biological data from approximately 3,000 rare brain tumor patients worldwide. These efforts have led to game changing studies which showed that rare brain tumors were often misdiagnosed. By applying the most innovative tools and linking them to carefully curated patient data, they were able to show that some diseases with different names were the same, while others group together were different diseases. This information has now helped create the new World Health Organization diagnostic classification for rare EBTs. The improved recognition and precision in diagnosis of rare brain tumors has now opened critical new opportunities to study mechanisms by which these tumors grow, how and why treatment fails, and to identify new types of drugs. These new areas of research will need appropriate biomaterials such as recurrent tumor samples or tissues from postmortem as well as matched detailed data, for which the RBTC is creating a dedicated registry and biobank that will be built on its existing collection of matching tumor samples at diagnosis and will leverage its established global network. Currently, failure in treatment of brain tumors is detected late and with MRI or CT, which are crude tools. To make brain tumor therapy more precise and effective, we need more sensitive, less resource intense, and rapid methods of detecting treatment failure early so that ineffective treatment can be stopped, and alternative treatments can be instituted earlier. In the context of the very young age of rare brain tumor patients, it will also be invaluable to be able to confirm with certainty that tumor cells are all eliminated and potentially save a child the devastation of brain and spine radiation. The RBTC proposal to collect plasma and cerebrospinal fluid from patients with rare brain tumors
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Jan 04, 2024
- Source ID
- HT94252310732
Entities
People
- Annie Huang
Organizations
- Hospital for Sick Children
- United States Army