About Our Program

The Immune Deficiency Cellular Therapy Program (ID-CTP) seeks to accelerate progress in the treatment of children, adolescents and young adults with genetic diseases of the blood and immune system, collaborating across multiple specialties to synergize disease-specific and gene mutation-specific approaches. 

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 these diseases. Trans-laboratory projects engage both basic scientists and clinicians in the detailed molecular and cellular assessment of the restoration of normal immune function. Multidisciplinary studies focus on clinical issues that transcend individual protocols, such as detection and management of infections, classification and treatment of graft-versus-host disease, and detection and management of hyperinflammatory immune reconstitution syndromes driven by cytokine release.

The Program's goals:

1) coordinate basic, preclinical and clinical research efforts across multiple specialties and several NIH Institutes to accelerate progress in treatment of patients with genetic diseases of the blood and immune system;

2) engage basic scientists and clinicians in the detailed molecular and cellular assessment of the restoration of normal immune function;

3) develop and implement allogeneic transplant and gene and cellular therapy protocols to treat children, adolescents and young adults with these diseases; and

4) provide multi-disciplinary expertise to focus on key clinical questions such as detection and management of infections, classification and treatment of graft-versus-host disease, and detection and management of hyperinflammatory immune reconstitution syndromes driven by cytokine release to improve patient safety and outcomes.

Science

Our Research Interests

Sung-Yun Pai, M.D., is focused on cellular therapy for several disorders, including severe combined immunodeficiency (SCID), Wiskott-Aldrich syndrome (WAS) and DOCK8 deficiency, and developing new gene therapy approaches for these disorders.

Dennis D. Hickstein, M.D., is working on the recently described bone failure syndrome GATA2 deficiency.  In particular, his lab is investigating mutations outside of the GATA2 exons in patients with MonoMAC syndrome, mutations associated with myeloid transformation in patients with GATA2 deficiency who have evidence of myeloid progression, and developing new approaches to the treatment of GATA2 deficiency with hematopoietic stem cell transplantation.

Steven Z. Pavletic, M.D., M.S., is directing clinical trials focused on chronic graft versus host disease (cGVHD) and hematopoietic stem cell transplantation.  Dr. Pavletic was instrumental in helping to develop develop national and international consensus guidelines for cGVHD clinical trials.