University of California San Francisco
Helen Diller Family Comprehensive Cancer Center
Morton J. Cowan, MD

Morton J. Cowan, MD

Professor, Department of Pediatrics, UCSF

Cancer Center Program Memberships

Affiliate Member

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

Overview:

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.

Education

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. Shah AJ, Sokolic R, Logan B, Yin Z, Iyengar S, Scalchunes C, Mangurian C, Albert M, Cowan MJ. Quality of Life of Patients with Wiskott Aldrich Syndrome and X-Linked Thrombocytopenia: a Study of the Primary Immune Deficiency Consortium (PIDTC), Immune Deficiency Foundation, and the Wiskott-Aldrich Foundation. J Clin Immunol. 2019 Oct 16.
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  2. Marsh RA, Leiding JW, Logan BR, Griffith LM, Arnold DE, Haddad E, Falcone EL, Yin Z, Patel K, Arbuckle E, Bleesing JJ, Sullivan KE, Heimall J, Burroughs LM, Skoda-Smith S, Chandrakasan S, Yu LC, Oshrine BR, Cuvelier GDE, Thakar MS, Chen K, Teira P, Shenoy S, Phelan R, Forbes LR, Chellapandian D, Dávila Saldaña BJ, Shah AJ, Weinacht KG, Joshi A, Boulad F, Quigg TC, Dvorak CC, Grossman D, Torgerson T, Graham P, Prasad V, Knutsen A, Chong H, Miller H, de la Morena MT, DeSantes K, Cowan MJ, Notarangelo LD, Kohn DB, Stenger E, Pai SY, Routes JM, Puck JM, Kapoor N, Pulsipher MA, Malech HL, Parikh S, Kang EM. Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT. J Clin Immunol. 2019 Oct; 39(7):653-667.
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  3. Mangurian C, Scalchunes C, Yoo J, Logan B, Henderson T, Iyengar S, Smith H, Cowan MJ. Psychosocial services for primary immunodeficiency disorder families during hematopoietic cell transplantation: A descriptive study. Palliat Support Care. 2019 Aug; 17(4):409-414.
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  4. Mamcarz E, Zhou S, Lockey T, Abdelsamed H, Cross SJ, Kang G, Ma Z, Condori J, Dowdy J, Triplett B, Li C, Maron G, Aldave Becerra JC, Church JA, Dokmeci E, Love JT, da Matta Ain AC, van der Watt H, Tang X, Janssen W, Ryu BY, De Ravin SS, Weiss MJ, Youngblood B, Long-Boyle JR, Gottschalk S, Meagher MM, Malech HL, Puck JM, Cowan MJ, Sorrentino BP. Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1. N Engl J Med. 2019 04 18; 380(16):1525-1534.
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  5. Dvorak CC, Long-Boyle J, Dara J, Melton A, Shimano KA, Huang JN, Puck JM, Dorsey MJ, Facchino J, Chang CK, Cowan MJ. Low Exposure Busulfan Conditioning to Achieve Sufficient Multilineage Chimerism in Patients with Severe Combined Immunodeficiency. Biol Blood Marrow Transplant. 2019 Jul; 25(7):1355-1362.
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  6. Amatuni GS, Currier RJ, Church JA, Bishop T, Grimbacher E, Nguyen AA, Agarwal-Hashmi R, Aznar CP, Butte MJ, Cowan MJ, Dorsey MJ, Dvorak CC, Kapoor N, Kohn DB, Markert ML, Moore TB, Naides SJ, Sciortino S, Feuchtbaum L, Koupaei RA, Puck JM. Newborn Screening for Severe Combined Immunodeficiency and T-cell Lymphopenia in California, 2010-2017. Pediatrics. 2019 02; 143(2).
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  7. Ferrua F, Galimberti S, Courteille V, Slatter MA, Booth C, Moshous D, Neven B, Blanche S, Cavazzana M, Laberko A, Shcherbina A, Balashov D, Soncini E, Porta F, Al-Mousa H, Al-Saud B, Al-Dhekri H, Arnaout R, Formankova R, Bertrand Y, Lange A, Smart J, Wolska-Kusnierz B, Aquino VM, Dvorak CC, Fasth A, Fouyssac F, Heilmann C, Hoenig M, Schuetz C, Kelecic J, Bredius RGM, Lankester AC, Lindemans CA, Suarez F, Sullivan KE, Albert MH, Kalwak K, Barlogis V, Bhatia M, Bordon V, Czogala W, Alonso L, Dogu F, Gozdzik J, Ikinciogullari A, Kriván G, Ljungman P, Meyts I, Mustillo P, Smith AR, Speckmann C, Sundin M, Keogh SJ, Shaw PJ, Boelens JJ, Schulz AS, Sedlacek P, Veys P, Mahlaoui N, Janda A, Davies EG, Fischer A, Cowan MJ, Gennery AR. Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study. J Allergy Clin Immunol. 2019 Jun; 143(6):2238-2253.
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  8. Yoo J, Halley MC, Lown EA, Yank V, Ort K, Cowan MJ, Dorsey MJ, Smith H, Iyengar S, Scalchunes C, Mangurian C. Supporting caregivers during hematopoietic cell transplantation for children with primary immunodeficiency disorders. J Allergy Clin Immunol. 2019 Jun; 143(6):2271-2278.
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  9. Dvorak CC, Haddad E, Buckley RH, Cowan MJ, Logan B, Griffith LM, Kohn DB, Pai SY, Notarangelo L, Shearer W, Prockop S, Kapoor N, Heimall J, Chaudhury S, Shyr D, Chandra S, Cuvelier G, Moore T, Shenoy S, Goldman F, Smith AR, Sunkersett G, Vander Lugt M, Caywood E, Quigg T, Torgerson T, Chandrakasan S, Craddock J, Dávila Saldaña BJ, Gillio A, Shereck E, Aquino V, DeSantes K, Knutsen A, Thakar M, Yu L, Puck JM. The genetic landscape of severe combined immunodeficiency in the United States and Canada in the current era (2010-2018). J Allergy Clin Immunol. 2019 Jan; 143(1):405-407.
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  10. Chinen J, Cowan MJ. Advances and highlights in primary immunodeficiencies in 2017. J Allergy Clin Immunol. 2018 10; 142(4):1041-1051.
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  11. Haddad E, Logan BR, Griffith LM, Buckley RH, Parrott RE, Prockop SE, Small TN, Chaisson J, Dvorak CC, Murnane M, Kapoor N, Abdel-Azim H, Hanson IC, Martinez C, Bleesing JJH, Chandra S, Smith AR, Cavanaugh ME, Jyonouchi S, Sullivan KE, Burroughs L, Skoda-Smith S, Haight AE, Tumlin AG, Quigg TC, Taylor C, Dávila Saldaña BJ, Keller MD, Seroogy CM, Desantes KB, Petrovic A, Leiding JW, Shyr DC, Decaluwe H, Teira P, Gillio AP, Knutsen AP, Moore TB, Kletzel M, Craddock JA, Aquino V, Davis JH, Yu LC, Cuvelier GDE, Bednarski JJ, Goldman FD, Kang EM, Shereck E, Porteus MH, Connelly JA, Fleisher TA, Malech HL, Shearer WT, Szabolcs P, Thakar MS, Vander Lugt MT, Heimall J, Yin Z, Pulsipher MA, Pai SY, Kohn DB, Puck JM, Cowan MJ, O'Reilly RJ, Notarangelo LD. SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery. Blood. 2018 10 25; 132(17):1737-1749.
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  12. Buchbinder D, Smith MJ, Kawahara M, Cowan MJ, Buzby JS, Abraham RS. Application of a radiosensitivity flow assay in a patient with DNA ligase 4 deficiency. Blood Adv. 2018 08 14; 2(15):1828-1832.
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  13. Eder M, Schwarz C, Kammer M, Jacobsen N, Stavroula ML, Cowan MJ, Chongkrairatanakul T, Gaston R, Ravanan R, Ishida H, Bachmann A, Alvarez S, Koch M, Garrouste C, Duffner UA, Cullis B, Schaap N, Medinger M, Sørensen SS, Dauber EM, Böhmig G, Regele H, Berlakovich GA, Wekerle T, Oberbauer R. Allograft and patient survival after sequential HSCT and kidney transplantation from the same donor-A multicenter analysis. Am J Transplant. 2019 Feb; 19(2):475-487.
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  14. Belderbos ME, Gennery AR, Dvorak CC, Blok HJ, Eikema DJ, Silva JMF, Veys P, Neven B, Buckley R, Cole T, Cowan MJ, Goebel WS, Hoenig M, Kuo CY, Stiehm ER, Wynn R, Bierings M. Outcome of domino hematopoietic stem cell transplantation in human subjects: An international case series. J Allergy Clin Immunol. 2018 11; 142(5):1628-1631.e4.
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  15. Miggelbrink AM, Logan BR, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Abdel-Azim H, Prockop SE, Shyr D, Decaluwe H, Hanson IC, Gillio A, Dávila Saldaña BJ, Eibel H, Hopkins G, Walter JE, Whangbo JS, Kohn DB, Puck JM, Cowan MJ, Griffith LM, Haddad E, O'Reilly RJ, Notarangelo LD, Pai SY. B-cell differentiation and IL-21 response in IL2RG/JAK3 SCID patients after hematopoietic stem cell transplantation. Blood. 2018 06 28; 131(26):2967-2977.
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  16. Gilman AL, Leung W, Cowan MJ, Cannon M, Epstein S, Barnhart C, Shah K, Hyland M, Fukes T, Ivanova A. Donor lymphocyte infusion and methotrexate for immune recovery after T-cell depleted haploidentical transplantation. Am J Hematol. 2018 02; 93(2):169-178.
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  17. Heimall J, Logan BR, Cowan MJ, Notarangelo LD, Griffith LM, Puck JM, Kohn DB, Pulsipher MA, Parikh S, Martinez C, Kapoor N, O'Reilly R, Boyer M, Pai SY, Goldman F, Burroughs L, Chandra S, Kletzel M, Thakar M, Connelly J, Cuvelier G, Davila Saldana BJ, Shereck E, Knutsen A, Sullivan KE, DeSantes K, Gillio A, Haddad E, Petrovic A, Quigg T, Smith AR, Stenger E, Yin Z, Shearer WT, Fleisher T, Buckley RH, Dvorak CC. Immune reconstitution and survival of 100 SCID patients post-hematopoietic cell transplant: a PIDTC natural history study. Blood. 2017 12 21; 130(25):2718-2727.
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  18. Dobbs K, Tabellini G, Calzoni E, Patrizi O, Martinez P, Giliani SC, Moratto D, Al-Herz W, Cancrini C, Cowan M, Bleesing J, Booth C, Buchbinder D, Burns SO, Chatila TA, Chou J, Daza-Cajigal V, Ott de Bruin LM, de la Morena MT, Di Matteo G, Finocchi A, Geha R, Goyal RK, Hayward A, Holland S, Huang CH, Kanariou MG, King A, Kaplan B, Kleva A, Kuijpers TW, Lee BW, Lougaris V, Massaad M, Meyts I, Morsheimer M, Neven B, Pai SY, Parvaneh N, Plebani A, Prockop S, Reisli I, Soh JY, Somech R, Torgerson TR, Kim YJ, Walter JE, Gennery AR, Keles S, Manis JP, Marcenaro E, Moretta A, Parolini S, Notarangelo LD. Corrigendum: Natural Killer Cells from Patients with Recombinase-Activating Gene and Non-Homologous End Joining Gene Defects Comprise a Higher Frequency of CD56bright NKG2A+++ Cells, and Yet Display Increased Degranulation and Higher Perforin Content. Front Immunol. 2017; 8:1244.
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  19. Heimall J, Cowan MJ. Long term outcomes of severe combined immunodeficiency: therapy implications. Expert Rev Clin Immunol. 2017 11; 13(11):1029-1040.
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  20. Dvorak CC, Puck JM, Wahlstrom JT, Dorsey M, Melton A, Cowan MJ. Neurologic event-free survival demonstrates a benefit for SCID patients diagnosed by newborn screening. Blood Adv. 2017 Sep 12; 1(20):1694-1698.
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