Research

The UCSF Pediatric Allergy, Immunology, and Bone Marrow Transplantation research programs aim to advance our understanding of complex disorders of the immune system, and to improve outcomes of children with severe allergic, immunologic, malignant, and non-malignant diseases (including marrow failure, primary immunodeficiencies, immunodysregulatory disorders, and metabolic diseases) using cutting-edge care that includes all available cellular modalities. Some of the exciting work that our researchers are involved in includes:

Allergy

  • Clinical research on oral immunotherapy for treatment of food allergy
  • Translational research on IgE profiling in food allergy
  • Translational research on eosinophilic esophagitis

Immunology

  • Laboratory and translational research on genetic and molecular basis of immune system disorders
    • Newborn screening for T-cell disorders: spectrum and outcomes
    • Functional analysis of candidate genes in primary T-cell immunodeficiencies
    • Sequencing of newborn blood spot DNA to improve and expand newborn screening
    • Measurements of T-cell production and diversity pre- and post- treatment for severe combined immunodeficiency disease (SCID)
    • CRISPR/Cas9 modification of iPS and hematopoietic stem cells to study T-cell development and treatment for SCID
  • United States Immune Deficiency Network (USIDNET): Registry and clinical research on the natural history of primary immunodeficiencies
  • UCSF Jeffrey Modell Foundation Diagnostic Center for Primary Immunodeficiencies: Laboratory studies to uncover molecular mechanisms of immune defects
  • Laboratory studies on novel genetic susceptibilities to autoimmunity
  • Clinical trial of humanized pegylated adenosine deaminase for patients with ADA-Deficient SCID
  • Clinical research on the psychosocial Impact of infant SCID diagnosis on parents and relatives
  • Clinical research on the optimization of pre-transplant management of patients with chronic granulomatous disease

Bone Marrow Transplant & Cellular Therapy

  • Laboratory studies on mechanisms of aberrant hematopoiesis
  • Laboratory and clinical research on the use of genetically-corrected autologous hematopoietic cells (“Gene Therapy”) to treat SCID
  • Clinical research on optimizing the dose of the conditioning agents to be administered during the pre-transplant period
  • Clinical research on non-chemotherapy-based methods to engraft hematopoietic stem cells
  • Clinical research on optimizing the method of ex vivo graft manipulation for mismatched donor transplantation
  • Clinical research on the role of post-transplant immunomodulation in preventing leukemic relapse following allogeneic hematopoietic cell transplantation
  • Clinical research on preventing transplant-related morbidity and mortality, especially that related to opportunistic infections and organ toxicity
  • Expanded access studies of investigational medications and cellular therapies to diagnose and treat rare or severe transplant-related complications, including opportunistic infections and graft-versus-host-disease (GVHD)

In addition, we actively participate in and lead studies through several national clinical trial consortiums, including:

  • The UCSF Division of Pediatric Allergy, Immunology, and Bone Marrow Transplantation is the lead site for the national Primary Immunodeficiency Treatment Consortium (PIDTC). Active protocols include:
    • 6901: A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with Severe Combined Immune Deficiency Disorders
    • 6902: A Retrospective and Cross-Sectional Analysis of Patients Treated for Severe Combined Immune Deficiency (1968-2010)
    • 6903: Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995
    • 6904: Analysis of Patients Treated for Wiskott-Aldrich Syndrome Since January 1, 1990
  • The Pediatric Blood and Marrow Transplant Consortium
  • The Children’s Oncology Group
  • The International Pediatric Fungal Network
  • The Center for International Blood and Marrow Transplant Research

Faculty Research:

Alice Chan, MD, PhD

Morton Cowan, MD

Morna Dorsey, MD, MMSc

Christopher Dvorak, MD

Craig Forester, MD, PhD

Alexis Melton, MD, PhD

Janel Long-Boyle, PharmD, PhD

Jennifer Puck, MD

Justin Wahlstrom, MD

Jennifer Willert, MD

Sandhya Kharbanda, MD

Jasmeen Dara, MD

Angela Chang, MD

Kristin Shimano, MD

Clinical Research Staff

Kenny Truong, Research Nurse

Carol Fraser-Brown, Clinical Research Coordinator

Kevin Magruder, Clinical Research Coordinator

Ying Lu, Clinical Research Coordinator

Jueleah Expose-Spencer, Clinical Research Coordinator

Jueeli Dahake, Data Manager & Clinical Research Coordinator

To Reach a Member of the Clinical Research Team, please call 415-476-2656

PIDTC

Elizabeth Dunn, Clinical Research Coordinator PIDTC Senior Project Manager

Megan Murnane, Clinical Research Coordinator, PIDTC Assistant Project Manager

Tara Bani-Hashemi, Clinical Research Coordinator, PIDTC Assistant Project Manager

To Reach a Member of the PIDTC Studies Team, please call (415) 502-0203

Research Resources

Search for a Clinical Trial at UCSF Benioff Children’s Hospital

National Directory of Ongoing Clinical Trials: http://clinicaltrials.gov/

PIDTC Trials: https://www.rarediseasesnetwork.org/cms/pidtc/Get-Involved/Studies