ID-CTP Clinical Core

Division Of Basic Sciences - Nci

Investigators

Linked publications, trials & patents

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, germline genetic disorders predisposing to hematologic malignancy, and acquired diseases such as myelodysplastic syndrome and acute myelogenous leukemia. 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 clinical trials in several key areas. In the area of inborn errors of immunity, we have open and enrolling trials of transplantation for GATA2 deficiency (MonoMac syndrome), DOCK8 deficiency and others. There is a robust and active program of research in the natural history and treatment of cGVHD. In the area of germline predisposition to myeloid malignancy, we have opened a protocol to test the effect of imatinib, a tyrosine kinase inhibitor, in patients with germline mutations in RUNX1. We have opened a natural history study of patients with germline mutations in DDX41. Transplant protocols for both of these disorders are in development as is a protocol for treatment of acute myelogenous leukemia with a novel conditioning agent.