Immune Matters: a PIDTC Podcast

In this episode of Immune Matters, hosts Jack McDonnell and Elie Haddad speak with Dr. Joseph Oved, a pediatric hematologist-oncologist and transplant specialist at Columbia University, about the biologic therapy emapalumab (Gamifant). The discussion explores how this anti–interferon-gamma monoclonal antibody is used to treat hyperinflammatory conditions such as primary hemophagocytic lymphohistiocytosis (HLH) and macrophage activation syndrome. Dr. Oved explains the underlying immunology of interferon-gamma–driven inflammation and how targeted blockade of this pathway can interrupt the cycle of immune dysregulation. The conversation also examines emerging applications of emapalumab in hematopoietic stem cell transplantation, including its potential role in preventing graft rejection, managing hyperinflammation, and improving transplant outcomes in patients with inborn errors of immunity. The episode concludes with a discussion of ongoing research, the importance of precision medicine in immune dysregulation disorders, and new PIDTC initiatives that allow clinicians to collaborate on complex cases and share expertise across institutions. Note: for more information on how you can get the PIDTC's help with a complex case, please email Jessica.Ni@ucsf.edu or Lisa.Lim@ucsf.edu . Keywords: emapalumab, Gamifant, HLH, macrophage activation syndrome, interferon-gamma, hyperinflammation, hematopoietic stem cell transplant, graft rejection, inborn errors of immunity, PIDTC, immune dysregulation, biologics, precision medicine, clinical immunology

In this episode of Immune Matters, Dr. Jack McDonnell and Dr. Elie Haddad welcome Dr. Jen Heimall, an expert in severe combined immune deficiency (SCID). They discuss the complexities of SCID, including its diagnosis, management, and the importance of newborn screening. Dr. Heimall shares insights from her research on treatment outcomes and the significance of collaboration in SCID care. The conversation also touches on the challenges faced by patients and families, the evolving landscape of gene therapy, and the urgent questions that remain in the field of immunology. Keywords SCID, immune deficiency, pediatric immunology, bone marrow transplant, gene therapy, patient experience, newborn screening, PIDTC, immunology research, immune system

In this episode of Immune Matters: a PIDTC Podcast, Dr. Jack McDonnell and Dr. Elie Haddad, along with guests Dr. Blachy Davila and Dr. Jessie Alexander, delve into Wiskott-Aldrich syndrome, a rare X-linked immunodeficiency affecting boys. They discuss the clinical manifestations, management strategies, and the importance of bone marrow transplantation as a definitive treatment. The conversation highlights a recent study analyzing transplant outcomes and conditioning regimens, emphasizing the need for timely intervention and collaboration in research to improve patient care. The Primary Immune Deficiency Treatment Consortium (PIDTC) is a part of the Rare Diseases Clinical Research Network of the Office of Rare Diseases Research (ORDR), National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH). This work was supported by the Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases (DAIT, NIAID) and the ORDR under cooperative agreements U54-AI082973 and U54-NS064808 and grants R13-AI094943 and 1R24AI184316-01. The content and opinions expressed are solely the responsibility of the authors and do not represent the official policy or position of the NIAID, ORDR, NCATS, NIH, HRSA, or any other agency of the US Government.