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Medical Advisory Board

Dr. David Lewis



Dr. Alice Bertaina 



Dr. David Lewis

Clinical Focus

  • Primary Immunodeficiency

  • Pediatrics

Academic Appointments

Administrative Appointments

  • Acting Associate Professor, Stanford University, Department of Pediatrics (1997 - 1997)

  • Associate Professor, Stanford University, Department of Pediatrics (1997 - 2005)

  • Director of the Jeffrey Modell Primary Immunodeficiency Center at Stanford, Supported by the Jeffrey Modell Foundation and the Lucile Packard Fund of Children's Health (2002 - Present)

  • Professor, Stanford University, Department of Pediatrics (2005 - Present)

  • Chief, Stanford Department of Pediatrics, Division of Immunology and Allergy (2008 - Present)

Honors & Awards

  • Henry J. Kaiser Award for Excellence in Preclinical Teaching (Immunology), Kaiser Foundation (2001)

  • Henry J. Kaiser Award for Excellence in Preclinical Teaching (Immunology), Kaiser Foundation (2005)

Professional Education

  • Fellowship:Seattle Children's Hospital Pediatric Infectious Diseases Fellowship (1988) WA

  • Residency:UCSF Pediatric Residency (1984) CA

  • Internship:Seattle Childrens Hospital Pediatric Residency (1982) WA

  • Medical Education:University of California at San Francisco School of Medicine (1981) CA

  • Board Certification: Pediatrics, American Board of Pediatrics (1986)

  • M.D., Univ.Calif at San Francisco, Medicine (1981)

  • B.S., Yale University, Biology (1976)



University - Faculty

Department: Pediatrics - Immunology

Position: Professor-Med Ctr Line

CCSR Bldg, Room 3215

Stanford,  California  94305-5101 

(650) 498-6077 (fax)


Alternate Contact

Elena Infeld

Administrative Assistant

650-498-6073 (office)



Pediatric Allergy and Immunology Clinic

730 Welch Rd 1st Fl Palo Alto, CA 94304

(650) 723-0290 (office)

(650) 497-0399 (fax)

A long-standing interest is to understand the cellular and molecular basis for this vulnerability of the human neonate to infection with intracellular pathogens that require T helper 1 (Th1) cells [CD4 T cell producing interferon-gamma (IFN-gamma)] for effective immune control. We have previously shown that CD4 T cells of the newborn have a unique limitation in the ability to produce certain effector molecules, such as CD40-ligand (CD154) and IFN-gamma compared to these cells in adults due to both reduced gene transcriptional and impaired signals that lead to gene transcription. Recently, we have shown that these limitations apply to physiological T-cell activation, e.g., using allogeneic dendritic cells. Defining the molecular mechanisms for decreased IFN-gamma production by neonatal CD4 T cells is a current focus. 
We have also found that recent thymic emigrants, which predominate in the newborn and young infant, are less able to differentiate into T helper 1 cells, which produce IFN-gamma. These studies required the development of a novel marker for recent thymic emigrants (RTEs) of the CD4 T-cell lineage in humans. Using a combination of approaches, we have identified protein tyrosine kinase 7 (PTK7) as such a marker. In progress are to studies to define the role of PTK7, an orphan member (no known ligand) of the receptor tyrosine kinase family, in T-cell development and immunity, and to determine how this marker can be used to follow the output of recent thymic emigrants in health and disease. We are also interesed in determining the molecular mechanisms for the reduced RTE function and to what extent these mechanisms are shared by neonatal CD4 T cells and CD4+CD8-CD3+ thymocytes, the immediate precursors of antigenically naive CD4 T cells. 

We have also found that limitations in T-cell immunogenicity to viruses and viral vaccines extend beyond the neonatal period to childhood. These studies highlight a need to develop more potent vaccines to overcome developmental and other factors, such as genetic inheritance, in mounting adaptive immunity. With this as an ultimate goal, we have previously examined the ability of a novel adjuvant, cationic liposome DNA complexes (CLDC)(Juvaris Biotherapeutics), to induce durable CD4 and CD8 T-cell immunity and humoral immunity to influenza A. The molecular and cellular components of the innate immune system that are required for immunogenicity are of particular interest. We are currently embarking on studies of vaccine immunity using novel influenza A virus antigens produced by our collaborators at Sutrovax. We are also beginning studies to determine if universal anti-influenza A viral protection can be achieved using catalytically inactive Cas proteins combined with appropriate guide RNAs. This work is being carried out in collaboration with Stanley Qi's laboratory at Stanford. 

As part of an on-going collaboration with Dr. Neal Boerkoel, University of British Columbia, we are defining the mechanism of T-cell lymphopenia in genetic deficiency of SMARCAL1, a protein that plays a novel role in relieving stalled DNA replication forks. Patients with SMARCAL1 deficiency (Schimke immuno-osseous dysplasia) suffer from not only T-cell immunodeficiency but also progressive renal dysfunction due to collapsing variant focal glomerulosclerosis, short stature due to growth place abnormalities, and vascular disease, with an increased risk of TIAs and strokes. How a block in DNA repair selective influences these disease programs is unclear and is a major current focus of research. This research is being supported in part by funds from the Kruzn' for a Kure Foundation, a philanthropic foundation that was started by parents of two children with SIOD.

Current Research and Scholarly Interests


Dr. Alice Bertaina

Dr. Alice Bertaina completed her MD degree at the University of Pavia in Italy, her fellowship in hematopoietic stem cell transplantation (HSCT) at the Bambino Gesù Children’s Hospital in Rome, and her PhD degree in Immunology and Biotechnology at Tor Vergata University in Rome. Until joining Stanford University in 2017, she was Head of the Stem Cell Transplant Unit in the Department of Hematology and Oncology at the Bambino Gesù Children’s Hospital in Rome (this institution currently has the largest number of children transplanted with hematopoietic progenitors/stem cells in Europe).

Dr. Bertaina is an expert in the field of allogeneic HSCT in pediatric patients affected by hematological malignancies or nonmalignant disorders. In particular, she has pioneered the novel approach of graft manipulation based on the physical elimination of alfa/beta T cells and B cells. Dr. Bertaina has excellent clinical and biological expertise, as demonstrated by her publications in the field of pediatric hematology and oncology. Moreover, she is expert in different aspects of immunological reconstitution of children given an allograft of hematopoietic stem cell, paying particular attention to innate immunity.


University - Faculty

Department: Pediatrics - Ped Stem Cell TransplantationPosition: Assoc Prof - Med Ctr Line


Administrative Associate

Beatrice Ochoa

650-498-1054 (office)



300 Pasteur Dr Rm H320 Stanford, CA 94305

(650) 497-2447 (office)

(650) 724-1164 (fax)


Clinical Focus

  • Pediatric Hematology-Oncology

Academic Appointments

Administrative Appointments

  • Head of Stem Cell Transplant Unit, Department of Hematology and Oncology, Bambino Gesù Children’s Hospital Rome, Italy (2013 - 2017)

  • Physician Assistant and Researcher, Department of Hematology and Oncology, Bambino Gesù Children’s Hospital, Rome, Italy (2010 - 2013)

  • Residency in Pediatrics, Pediatric Hematology and Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy (2008 - 2010)

  • Medical and Research Fellowship, Department of Pediatrics, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy (2004 - 2008)

  • Medical Assistant, Ergonometric Service, Division of Cardiology, Fondazione IRCCS Policlinico San Matteo, Pavia (2002 - 2003)

Honors & Awards

  • Press release 'alfa/beta T-cell depleted haplo-transplant in children with acute leukemia', 55th ASH Meeting, Engineering Cell Therapies Press Program, New Orleans (December 2013)

  • Abstract Travel Award 'Identification of deregulated microRNAs in JMML', 2016 International JMML Symposium, San Diego (December 2016)

  • Invited to participate in the Annual National Ceremony for Cancer Research, Palazzo del Quirinale, By Invitation from the President of the Italian Republic (October 2015)

Boards, Advisory Committees, Professional Organizations

  • Member, Child Health Research Institute Stanford (CHRI) (2017 - Present)

  • Peer Reviewed Journal Reviewer, Frontiers in Immunology Bone Marrow Transplantation (2016 - Present)

  • Member, American Society of Hematology (2013 - Present)

  • Member, European Society for Blood and Marrow Transplantation (2010 - Present)

  • Member, Comitato per il Controllo delle Infezioni Ospedalerie (CCIO) at the OPBG, Rome (2011 - 2017)

  • Board Member, EBMT PDWP (European Society for Blood and Marrow Transplantation Pediatric Disease Working Party) (2014 - 2017)

  • Peer Reviewed Journal Reviewer, Bone Marrow Transplantation (2015 - Present)

  • Member, Associazione Italiana di Ematologia ed Oncaologia Pediatrics (2010 - Present)

  • Head, HSCT Working Party Group of AIEOP (Italian Association of Pediatric Hematology and Oncology) (2015 - 2017)

  • Member, Inborn Errors Working Group, EBMT. (2016 - Present)

  • Chair, Paediatric transplantation Session, EBMT 43 rd Annual Meeting, Marseille, France, March 26-29, 2017 (2017 - 2017)

  • Member, GITMO project entitled ‘3 Steps’, aiming at defining strategies of prophylaxis and treatment of veno-occlusive disease in transplant recipients (2016 - Present)

  • Member, Italian Group for Bone Marrow Transplantation (2017 - Present)


Professional Education

  • Board Certification: Medicine, Provincial Association of Surgeons and Dentists of Turin (2006)

  • Fellowship:Ospedale Bambino Gesu' (2013) Italy

  • Residency:University of Pavia (2010) Italy

  • Medical Education:University of Pavia (2005) Italy

  • PhD, Tor Vergata University, Immunology and Biotechnology (2013)

  • Residency in Pediatrics, University of Pavia, Pavia, Italy, Thesis title: “NK-alloreactivity and outcome of pediatric patients with acute leukemia given an HLA-haploidentical stem cell transplantation” under Prof. Franco Locatelli supervision (2010)

  • Post-graduate fellowship, Bambino Gesù Children’s Hospital, Rome, Italy, Hematopoietic Stem Cell Transplantation (basic and applied research) (2010)

  • Medical Degree, University of Pavia, Pavia, Italy, Thesis title: “Human Metapneumovirus II in acute pediatric respiratory infection: epidemiology, clinical picture and diagnosis” (2005)

Current Research and Scholarly Interests

Dr. Bertaina is a highly experienced clinician and will play a key role in supporting Section Chief Dr. Rajni Agarwal and Clinical Staff in the Stem Cell Transplant Unit at Lucile Packard Children’s Hospital. She will also continue her research on immune recovery and miRNA, understanding the mechanisms underlying immune reconstitution, Graft-versus-Host Disease (GvHD), and leukemia relapse after allogeneic HSCT in pediatric patients affected by hematological malignant and non-malignant disorders.

Clinical Trials

KIR Favorable Mismatched Haplo Transplant and KIR Polymorphism in ALL/AML/MDS Allo-HCT ChildrenRecruitingMore

  • This is a phase II, open-label, non-randomized, prospective study of haploidentical transplantation using KIR-favorable donors for children with acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (HCT). The relationship of KIR2DL1 polymorphisms to survival in children with these diseases undergoing any approach to allogeneic HCT during the study time frame will also be determined.

    Lead Sponsor

    • Michael Pulsipher, MD


    • University of California, San Francisco

    • Children's Hospital of Philadelphia

    • Medical College of Wisconsin

    • Ann & Robert H Lurie Children's Hospital of Chicago

    • New York Medical College

    • Children's Hospital & Research Center Oakland

    • Vanderbilt University

    • Rady Children's Hospital, San Diego

Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell TransplantNot RecruitingMore

  • This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially matched family donor. The study will assess whether immune cells, called T cells, from the family donor, that are specially grown in the laboratory and given back to the patient along with the stem cell transplant can help the immune system recover faster after transplant. As a safety measure these T cells have been programmed with a self-destruct switch so that they can be destroyed if they start to react against tissues (Graft versus host disease).

Stanford is currently not accepting patients for this trial. For more information, please contact Cancer Clinical Trials Office (CCTO), 650-498-7061.

Lead Sponsor

  • Bellicum Pharmaceuticals

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