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 continued enrollment on a protocol for treatment of inborn errors of immunity including an amendment to adjust the dosing of alemtuzumab and investigation of alemtuzumab levels. Additional advanced practitioner staff have been added to support the clinical transplantation service that cares for malignant disorders. Members of the clinical core also support the work of other investigators in the branch. A protocol for virus-specific T cells is in development.