Rare Central Nervous System Cancers Initiative

Division Of Basic Sciences - Nci

Investigators

Linked publications & trials

Abstract

The NIH is uniquely suited to create a rare CNS cancer initiative. This effort utilizes the following specific resources of the NIH. This has required developing a clinical trial infrastructure, including data management system, to oversee the disease specific therapeutic and natural history studies. Collaborations with experts in imaging technologies to create imaging biomarkers and routine imaging for monitoring response to therapies are planned. A tissue repository has been established to store both tumor and normal tissue for molecular analyses including genomics, metabolomics, and proteomics with the intent of advancing the understanding of tumor biology and pathogenesis leading to better prognostic information, sub-classification and ultimately markers predictive of response to specific treatments. Additionally, specific testing capabilities have been developed such as methylation testing for accurate diagnosis, whole exome DNA and RNA sequencing to look for novel mutations and gene alterations such as fusions. A variety of clinical studies have been developed for this population of patients with rare cancers. For example, an immunotherapy trial is underway and actively recruiting. This study includes immunologic monitoring that are being performed in Dr. Gilbert's laboratory. NIH-based outreach efforts will be utilized to interact with patient advocacy groups to enhance patient participation in these efforts and provide a venue for educational programs to improve patient and caregiver understanding of their uncommon disease. Finally, an important component of the rare CNS Tumor program is outreach and education. Formal Memorandum of Understanding Agreements has been signed with 9 advocacy groups and the NCI-CONNECT website has been highly successful with over 500,000 unique visits and over 900,000 page views in calendar year 2021. Additionally, the website content has been translated into Spanish and this is garnering over 20,000 visits per month.