ID-CTP Clinical Core

Division Of Basic Sciences - Nci

Investigators

Linked publications & trials

Abstract

Recent advances in the molecular basis of disease have created exciting opportunities for the development and implementation of allogeneic transplant and gene and cellular therapy protocols to treat inborn errors of immunity and other genetic blood disorders. While reconstitution of immunity is the goal, current techniques frequently worsen immunodeficiency transiently before successful immune reconstitution. Furthermore, complications such as acute and chronic graft-versus-host disease (cGVHD) interfere with immune cell development and function. The ID-CTP clinical core provides support for clinical trials led by members of the ID-CTP. Currently, investigators within the ID-CTP are leading several clinical trials focused on improving treatment and outcomes for patients with cGVHD and those undergoing hematopoietic stem cell transplantation for treatment of immunodeficiency and immune dysregulation, such as GATA2 deficiency (MonoMac syndrome) and DOCK8 deficiency. Progress in the past year includes the expansion of the clinical core staff to accommodate the increasing volume of patients, and the opening of a new protocol for hematopoietic stem cell transplantation of patients with a broad variety of primary immunodeficiencies.