Morton J. Cowan, MD

Morton J. Cowan, MD

Professor, Department of Pediatrics, UCSF

Phone: (415) 476-2188 (appts)
Box 1278, UCSF
San Francisco, CA 94143-1278

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Research Summary

Current Non-NIH Awards:

TR3-05535 Cowan (PI) 11/01/2012-10/31/2015

California Institute of Regenerative Medicine vGene Correction of Autologous Hematopoietic Stem Cells in Artemis Deficient SCID

Goal is to complete the preclinical testing necessary for a clinical gene therapy trial in children with Artemis-SCID. The focus is on optimizing non-chemotherapy approaches to opening marrow niches, studying potential genotoxicity, and demonstrating that gene transduced Artemis deficient hematopoietic stem cells mature in vitro and in vivo into T and B cells.

Role: PI

DR2A-05365 Shizuru (PI) 07/01/2013-06/30/2017

California Institute of Regenerative Medicine vA monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants

Goal is to bring to a clinical trial a monoclonal antibody that targets hematopoietic stem cells. The antibody will be evaluated in a non-human primate model followed by a prospective phase I/II clinical trial in children with SCID.

Role: Co-PI


I am a pediatric immunologist, Professor of Pediatrics and Chief, Allergy Immunology and BMT Division at UCSF. I led the planning for what ultimately became the Primary Immune Deficiency Treatment Consortium (PIDTC), an NIH-funded group of 43 centers in the US and Canada focused on PID in which I am the PI and UCSF is the lead institution since 2009. I am on the Steering and Executive Committees of the Pediatric Blood and Marrow Transplant Consortium (PBMTC) and established and led for three years the Pediatric Special Interest Group within the American Society of Blood and Marrow Transplantation. My career has focused on children with PID, with over 180 peer-reviewed publications. My major research interests include SCID in Athabascan-speaking Native Americans (SCIDA), gene therapy for children with Artemis-deficient SCID, non-chemotherapy approaches to opening marrow niches, and the use of haploidentical donors and reduced-toxicity transplantation for childhood diseases. I performed one of the initial haplocompatible T cell depleted marrow transplants for SCID in North America in 1982 and reported one of the first in utero transplants in the world, the first patient with multiple biotin-dependent carboxylase deficiencies and immune deficiency, and the first children with HIV infection. I have treated the largest cohort of children with SCIDA and the largest number of babies diagnosed through newborn screening with SCID in North America, mapped the genetic locus of the gene for SCIDA and radiation sensitive SCID (RS-SCID), and identified the mutation in DCLRE1C responsible for SCIDA in Navajo and Apache children. I designed and completed one of the few prospective single institution trials of haplocompatible HCT for children with SCID and initiated and completed the first large study of children with SCID transplanted in North America and reported by the PIDTC.


Massachusetts Institute of Technology, Cambridge, MA, B.S., 1966, Electrical Engineering
University of Pennsylvania, Philadelphia, PA, M.D., 1970, Medicine
University of California, San Francisco, Intern/Resident, 1975-77, Pediatrics
University of California, San Francisco, Fellow, 1977-79, Immunology

Professional Experience

  • 1972-74
    Scientist, Division of Lung Diseases, National Heart and Lung Institute, Bethesda, MD
  • 1974-75
    Staff Investigator, Pulmonary Biochemistry Branch, National Heart and Lung Institute, NIH, Bethesda, MD
  • 1979-83
    Assistant Professor, Department of Pediatrics, University of California, San Francisco
  • 1984-89
    Associate Professor of Pediatrics, Director, Pediatric Bone Marrow Transplant Program, University of California, San Francisco
  • 1989-present
    Professor of Pediatrics, Director, Bone Marrow Transplant Program, UCSF

Honors & Awards

  • 1966
    Tau Beta Pi, Engineer Honor Society
  • 1968-70
    Charles Hovies Scholarship
  • 1979-82
    Pediatric Clinical Research Center Investigator Award

Selected Publications

  1. Walter JE, Rosen LB, Csomos K, Rosenberg JM, Mathew D, Keszei M, Ujhazi B, Chen K, Lee YN, Tirosh I, Dobbs K, Al-Herz W, Cowan MJ, Puck J, Bleesing JJ, Grimley MS, Malech H, De Ravin SS, Gennery AR, Abraham RS, Joshi AY, Boyce TG, Butte MJ, Nadeau KC, Balboni I, Sullivan KE, Akhter J, Adeli M, El-Feky RA, El-Ghoneimy DH, Dbaibo G, Wakim R, Azzari C, Palma P, Cancrini C, Capuder K, Condino-Neto A, Costa-Carvalho BT, Oliveira JB, Roifman C, Buchbinder D, Kumanovics A, Franco JL, Niehues T, Schuetz C, Kuijpers T, Yee C, Chou J, Masaad MJ, Geha R, Uzel G, Gelman R, Holland SM, Recher M, Utz PJ, Browne SK, Notarangelo LD. Broad-spectrum antibodies against self-antigens and cytokines in RAG deficiency. J Clin Invest. 2015 Nov 2; 125(11):4135-48.
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  2. Zinter MS, Dvorak CC, Spicer A, Cowan MJ, Sapru A. New Insights Into Multicenter PICU Mortality Among Pediatric Hematopoietic Stem Cell Transplant Patients. Crit Care Med. 2015 Sep; 43(9):1986-94.
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  3. Shapiro EG, Nestrasil I, Rudser K, Delaney K, Kovac V, Ahmed A, Yund B, Orchard PJ, Eisengart J, Niklason GR, Raiman J, Mamak E, Cowan MJ, Bailey-Olson M, Harmatz P, Shankar SP, Cagle S, Ali N, Steiner RD, Wozniak J, Lim KO, Whitley CB. Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment. Mol Genet Metab. 2015 Sep-Oct; 116(1-2):61-8.
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  4. Cowan MJ, Gennery AR. Radiation-sensitive severe combined immunodeficiency: The arguments for and against conditioning before hematopoietic cell transplantation-what to do? J Allergy Clin Immunol. 2015 Nov; 136(5):1178-85.
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  5. Multhaup MM, Podetz-Pedersen KM, Karlen AD, Olson ER, Gunther R, Somia NV, Blazar BR, Cowan MJ, McIvor RS. Role of transgene regulation in ex vivo lentiviral correction of artemis deficiency. Hum Gene Ther. 2015 Apr; 26(4):232-43.
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  6. Long-Boyle JR, Savic R, Yan S, Bartelink I, Musick L, French D, Law J, Horn B, Cowan MJ, Dvorak CC. Population pharmacokinetics of busulfan in pediatric and young adult patients undergoing hematopoietic cell transplant: a model-based dosing algorithm for personalized therapy and implementation into routine clinical use. Ther Drug Monit. 2015 Apr; 37(2):236-45.
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  7. Kwan A, Hu D, Song M, Gomes H, Brown DR, Bourque T, Gonzalez-Espinosa D, Lin Z, Cowan MJ, Puck JM. Successful newborn screening for SCID in the Navajo Nation. Clin Immunol. 2015 May; 158(1):29-34.
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  8. Horn B, Petrovic A, Wahlstrom J, Dvorak CC, Kong D, Hwang J, Expose-Spencer J, Gates M, Cowan MJ. Chimerism-based pre-emptive immunotherapy with fast withdrawal of immunosuppression and donor lymphocyte infusions after allogeneic stem cell transplantation for pediatric hematologic malignancies. Biol Blood Marrow Transplant. 2015 Apr; 21(4):729-37.
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  9. Punwani D, Wang H, Chan AY, Cowan MJ, Mallott J, Sunderam U, Mollenauer M, Srinivasan R, Brenner SE, Mulder A, Claas FH, Weiss A, Puck JM. Combined Immunodeficiency Due to MALT1 Mutations, Treated by Hematopoietic Cell Transplantation. J Clin Immunol. 2015 Feb; 35(2):135-46.
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  10. Pai SY, Cowan MJ. Stem cell transplantation for primary immunodeficiency diseases: the North American experience. Curr Opin Allergy Clin Immunol. 2014 Dec; 14(6):521-6.
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  11. Kwan A, Abraham RS, Currier R, Brower A, Andruszewski K, Abbott JK, Baker M, Ballow M, Bartoshesky LE, Bonilla FA, Brokopp C, Brooks E, Caggana M, Celestin J, Church JA, Comeau AM, Connelly JA, Cowan MJ, Cunningham-Rundles C, Dasu T, Dave N, De La Morena MT, Duffner U, Fong CT, Forbes L, Freedenberg D, Gelfand EW, Hale JE, Hanson IC, Hay BN, Hu D, Infante A, Johnson D, Kapoor N, Kay DM, Kohn DB, Lee R, Lehman H, Lin Z, Lorey F, Abdel-Mageed A, Manning A, McGhee S, Moore TB, Naides SJ, Notarangelo LD, Orange JS, Pai SY, Porteus M, Rodriguez R, Romberg N, Routes J, Ruehle M, Rubenstein A, Saavedra-Matiz CA, Scott G, Scott PM, Secord E, Seroogy C, Shearer WT, Siegel S, Silvers SK, Stiehm ER, Sugerman RW, Sullivan JL, Tanksley S, Tierce ML, Verbsky J, Vogel B, Walker R, Walkovich K, Walter JE, Wasserman RL, Watson MS, Weinberg GA, Weiner LB, Wood H, Yates AB, Puck JM. Newborn screening for severe combined immunodeficiency in 11 screening programs in the United States. JAMA. 2014 Aug 20; 312(7):729-38.
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  12. Dvorak CC, Hassan A, Slatter MA, Hönig M, Lankester AC, Buckley RH, Pulsipher MA, Davis JH, Güngör T, Gabriel M, Bleesing JH, Bunin N, Sedlacek P, Connelly JA, Crawford DF, Notarangelo LD, Pai SY, Hassid J, Veys P, Gennery AR, Cowan MJ. Comparison of outcomes of hematopoietic stem cell transplantation without chemotherapy conditioning by using matched sibling and unrelated donors for treatment of severe combined immunodeficiency. J Allergy Clin Immunol. 2014 Oct; 134(4):935-943.e15.
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  13. Pai SY, Logan BR, Griffith LM, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Hanson IC, Filipovich AH, Jyonouchi S, Sullivan KE, Small TN, Burroughs L, Skoda-Smith S, Haight AE, Grizzle A, Pulsipher MA, Chan KW, Fuleihan RL, Haddad E, Loechelt B, Aquino VM, Gillio A, Davis J, Knutsen A, Smith AR, Moore TB, Schroeder ML, Goldman FD, Connelly JA, Porteus MH, Xiang Q, Shearer WT, Fleisher TA, Kohn DB, Puck JM, Notarangelo LD, Cowan MJ, O'Reilly RJ. Transplantation outcomes for severe combined immunodeficiency, 2000-2009. N Engl J Med. 2014 Jul 31; 371(5):434-46.
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  14. Dvorak CC, Horn BN, Puck JM, Adams S, Veys P, Czechowicz A, Cowan MJ. A trial of alemtuzumab adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency. Pediatr Transplant. 2014 Sep; 18(6):609-16.
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  15. Dvorak CC, Horn BN, Puck JM, Czechowicz A, Shizuru JA, Ko RM, Cowan MJ. A trial of plerixafor adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency. Pediatr Transplant. 2014 Sep; 18(6):602-8.
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  16. Soni S, Boulad F, Cowan MJ, Scaradavou A, Dahake J, Edwards S, Walters MC. Combined umbilical cord blood and bone marrow from HLA-identical sibling donors for hematopoietic stem cell transplantation in children with hemoglobinopathies. Pediatr Blood Cancer. 2014 Sep; 61(9):1690-4.
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  17. Haddad E, Allakhverdi Z, Griffith LM, Cowan MJ, Notarangelo LD. Survey on retransplantation criteria for patients with severe combined immunodeficiency. J Allergy Clin Immunol. 2014 Feb; 133(2):597-9.
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  18. Shearer WT, Dunn E, Notarangelo LD, Dvorak CC, Puck JM, Logan BR, Griffith LM, Kohn DB, O'Reilly RJ, Fleisher TA, Pai SY, Martinez CA, Buckley RH, Cowan MJ. Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: the Primary Immune Deficiency Treatment Consortium experience. J Allergy Clin Immunol. 2014 Apr; 133(4):1092-8.
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  19. Schuetz C, Neven B, Dvorak CC, Leroy S, Ege MJ, Pannicke U, Schwarz K, Schulz AS, Hoenig M, Sparber-Sauer M, Gatz SA, Denzer C, Blanche S, Moshous D, Picard C, Horn BN, de Villartay JP, Cavazzana M, Debatin KM, Friedrich W, Fischer A, Cowan MJ. SCID patients with ARTEMIS vs RAG deficiencies following HCT: increased risk of late toxicity in ARTEMIS-deficient SCID. Blood. 2014 Jan 9; 123(2):281-9.
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  20. Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Pai SY, Ballard B, Bauer SC, Bleesing JJ, Boyle M, Brower A, Buckley RH, van der Burg M, Burroughs LM, Candotti F, Cant AJ, Chatila T, Cunningham-Rundles C, Dinauer MC, Dvorak CC, Filipovich AH, Fleisher TA, Bobby Gaspar H, Gungor T, Haddad E, Hovermale E, Huang F, Hurley A, Hurley M, Iyengar S, Kang EM, Logan BR, Long-Boyle JR, Malech HL, McGhee SA, Modell F, Modell V, Ochs HD, O'Reilly RJ, Parkman R, Rawlings DJ, Routes JM, Shearer WT, Small TN, Smith H, Sullivan KE, Szabolcs P, Thrasher A, Torgerson TR, Veys P, Weinberg K, Zuniga-Pflucker JC. Primary Immune Deficiency Treatment Consortium (PIDTC) report. J Allergy Clin Immunol. 2014 Feb; 133(2):335-47.
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