Acting Director
Brigitte C. Widemann, M.D.

Center for Cancer Research
National Cancer Institute

Building 10, Room 1W-3750
Bethesda, MD 20892
240-760-6560

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.

 


Patient Services


We are committed to improving outcomes for children, adolescents and young adults with genetic diseases of the blood and immune system.

Patients are provided guidance and assistance with appointments, travel, counseling and social support. We believe it is important to collaborate with our patients' primary care physicians in order to provide expert continuity of care. Before visiting, patients may be asked to provide medical records, disks with scans or x-ray studies and a list of medications. Important facts for our patients:

  • Consultations are provided at no cost to the patient
  • After a patient is accepted onto a study, treatments and tests performed at NIH are also at no cost to the patient
  • NIH does not cover the cost of medical services provided at other facilities
  • We are located in Bethesda, Maryland, approximately 8 miles northwest of Washington, D.C.
  • Our clinics are located in the NIH Clinical Center (Building 10)
  • Visitor information is available at NIH Clinical Center 

Questions?  Visit NIH Clinical Research Trials and You.

Patient Referrals

Patients, family members and physicians may contact our referral nurse from 8:30 AM to 5:00 PM, Monday-Friday:

Cara Kenney, MPH, BSN, RN, OCN
Referral Nurse
Immune Deficiency Cellular Therapy Program
NCI Center for Cancer Research

Ph: 240.760.6233
Cara.Kenney@nih.gov

 

Our care team contacts any prospective patients within 24 business hours after your call to collect basic information and give further instructions. We require a medical summary, operative notes, relevant radiographic scans (MRI, CT, PET), pathology report, and additional materials as indicated.

With the patient’s permission, we keep their primary physician fully informed and involved in their care. After consultation, the physician (or nurse practitioner) will contact the patient's primary physician to coordinate care and follow-up plans.

Educational Resources for Patients

Visit the NIH Genetic and Rare Diseases Information Center to learn more about:


Our Teams


  • Physicians and Investigators
  • Physican Assistants, Nurse Practitioners, and Research Nurses 
    • Daniele Avila, MSN, CRNP
    • Bazetta Blacklock-Schuver, RN
    • Kristan Cole, RN
    • Michael Emmanuel, RN
    • Tonya Jenkins, CRNP
    • Jeannette Nashed, CRNP
    • Laura Parsons-Wandell, RN
  • Patient Care Coordinators
    • Shaneil Flucker
    • Nina Manaye
  • Staff Scientists
  • Biologists
    • Lisa Embree
    • Robert West, Ph.D.

 

 


Our Research Interests


Brigitte C. Widemann, M.D., is focused on a more rational and targeted approach to drug discovery and development.  As head of the Pharmacology and Experimental Therapeutics Section in the Pediatric Oncology Branch, Dr. Widemann directs studies centerd on the application of new molecularly targeted agents to treat childhood cancers and neurofibromatosis type 1 (NF1).

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.


Training Opportunities


The NCI Center for Cancer Research is committed to training and supporting the next generation of cancer researchers; this is an important part of our mission. CCR offers a training environment that is second-to-none in quality of science and quality of life. Learn about all of the training/fellowship programs that are available at Training Opportunities at NCI CCR. For current openings, visit CCR Careers.

Unique programs have been set up for:

  • Medical School Graduates (MD, DDS, DVM)
  • Postdoctoral Fellows (PhD, MD/PhD, DVM)
  • Graduate/Professional School Students
  • College Graduates
  • College Students
  • High School Students

Why Train at CCR?

For information on benefits, services and programs of interest to trainees/fellows, visit Why Choose CCR? 

 

About


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.

 

For Patients


Patient Services


We are committed to improving outcomes for children, adolescents and young adults with genetic diseases of the blood and immune system.

Patients are provided guidance and assistance with appointments, travel, counseling and social support. We believe it is important to collaborate with our patients' primary care physicians in order to provide expert continuity of care. Before visiting, patients may be asked to provide medical records, disks with scans or x-ray studies and a list of medications. Important facts for our patients:

  • Consultations are provided at no cost to the patient
  • After a patient is accepted onto a study, treatments and tests performed at NIH are also at no cost to the patient
  • NIH does not cover the cost of medical services provided at other facilities
  • We are located in Bethesda, Maryland, approximately 8 miles northwest of Washington, D.C.
  • Our clinics are located in the NIH Clinical Center (Building 10)
  • Visitor information is available at NIH Clinical Center 

Questions?  Visit NIH Clinical Research Trials and You.

Patient Referrals

Patients, family members and physicians may contact our referral nurse from 8:30 AM to 5:00 PM, Monday-Friday:

Cara Kenney, MPH, BSN, RN, OCN
Referral Nurse
Immune Deficiency Cellular Therapy Program
NCI Center for Cancer Research

Ph: 240.760.6233
Cara.Kenney@nih.gov

 

Our care team contacts any prospective patients within 24 business hours after your call to collect basic information and give further instructions. We require a medical summary, operative notes, relevant radiographic scans (MRI, CT, PET), pathology report, and additional materials as indicated.

With the patient’s permission, we keep their primary physician fully informed and involved in their care. After consultation, the physician (or nurse practitioner) will contact the patient's primary physician to coordinate care and follow-up plans.

Educational Resources for Patients

Visit the NIH Genetic and Rare Diseases Information Center to learn more about:

Teams


Our Teams


  • Physicians and Investigators
  • Physican Assistants, Nurse Practitioners, and Research Nurses 
    • Daniele Avila, MSN, CRNP
    • Bazetta Blacklock-Schuver, RN
    • Kristan Cole, RN
    • Michael Emmanuel, RN
    • Tonya Jenkins, CRNP
    • Jeannette Nashed, CRNP
    • Laura Parsons-Wandell, RN
  • Patient Care Coordinators
    • Shaneil Flucker
    • Nina Manaye
  • Staff Scientists
  • Biologists
    • Lisa Embree
    • Robert West, Ph.D.

 

 

For Trainees


Training Opportunities


The NCI Center for Cancer Research is committed to training and supporting the next generation of cancer researchers; this is an important part of our mission. CCR offers a training environment that is second-to-none in quality of science and quality of life. Learn about all of the training/fellowship programs that are available at Training Opportunities at NCI CCR. For current openings, visit CCR Careers.

Unique programs have been set up for:

  • Medical School Graduates (MD, DDS, DVM)
  • Postdoctoral Fellows (PhD, MD/PhD, DVM)
  • Graduate/Professional School Students
  • College Graduates
  • College Students
  • High School Students

Why Train at CCR?

For information on benefits, services and programs of interest to trainees/fellows, visit Why Choose CCR? 

 

Our Science


Our Research Interests


Brigitte C. Widemann, M.D., is focused on a more rational and targeted approach to drug discovery and development.  As head of the Pharmacology and Experimental Therapeutics Section in the Pediatric Oncology Branch, Dr. Widemann directs studies centerd on the application of new molecularly targeted agents to treat childhood cancers and neurofibromatosis type 1 (NF1).

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.