Rare Diseases Research

CCR has a mandate to confront the special challenges presented by rare cancers and diseases as well cancers that may be predominant in medically underserved populations. CCR investigators, both at the laboratory bench and in the clinic, are actively engaged in this research endeavor.  In many cases, the knowledge CCR’s investigators gain from studying and treating rare cancers and related diseases provides them with insights into the mechanisms that underlie other more common malignancies.


CCR, working with the NIH Clinical Center, brings together patients from around the world who share rare disease diagnoses. CCR’s clinical experts can mobilize quickly to establish clinical trials on-site and identify agents that may bring relief to these patients.

In an effort to leverage CCR’s expertise in basic and clinical research studies of rare tumors, CCR launched the NCI Rare Tumor Initiative in 2013 with the goal of identifying and translating potential new therapies for rare tumors.

Clinical Studies: Rare Diseases and Cancers