An Update from the United States National Heart, Lung, and Blood Institute-funded Production Assistance for Cellular Therapies (PACT) Program: A Decade of Cell Therapy

Clinical and Translational Science

Volumn 7, Issue 2, 2014, Pages 93-99

  Wood, Deborah ^a   Wesselschmidt, Robin ^b   Hematti, Peiman ^c   Gee, Adrian P ^d   Rooney, Cliona ^d   Silberstein, Leslie ^e   Armant, Myriam ^e   Couture, Larry ^b   Wagner, John E ^f   Mckenna, David H ^f   Hei, Derek ^c   Mondoro, Traci Heath ^g   Welniak, Lisbeth ^g   Lindblad, Robert ^a  

^a EMMES CORPORATION   (United States)

^b Center for Applied Technology Development   (United States)

^c UNIVERSITY OF WISCONSIN MADISON   (United States)

^d CENTER FOR CELL AND GENE THERAPY   (United States)

^e BOSTON CHILDREN S HOSPITAL   (United States)

^f UNIVERSITY OF MINNESOTA   (United States)

^g NATIONAL HEART LUNG AND BLOOD INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

WISKOTT ALDRICH SYNDROME PROTEIN;

ACUTE LUNG INJURY; ARTICLE; AUTOLOGOUS BONE MARROW DERIVED MONONUCLEAR CELL TRANSPLANTATION; AUTOLOGOUS BONE MARROW TRANSPLANTATION; BONE MARROW DERIVED MESENCHYMAL STROMAL CELL; CELL THERAPY; CLINICAL PROTOCOL; CONFIDENTIALITY; CONSENSUS; CONTINUING EDUCATION; CYTOTOXIC T LYMPHOCYTE; EMBRYONIC STEM CELL; GENE MUTATION; GOOD MANUFACTURING PRACTICE; HUMAN; MESENCHYMAL STROMA CELL; MONONUCLEAR CELL; MULTICENTER STUDY (TOPIC); NATIONAL HEALTH ORGANIZATION; NATURAL KILLER CELL; NONHUMAN; PATENT; PEER REVIEW; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; PRODUCT DEVELOPMENT; PRODUCTION ASSISTANCE FOR CELLULAR THERAPY PROGRAM; PROFESSIONAL STANDARD; TOTAL QUALITY MANAGEMENT; WISKOTT ALDRICH SYNDROME; BIOLOGICAL THERAPY; ECONOMICS; FINANCIAL MANAGEMENT; PROGRAM DEVELOPMENT; TRANSLATIONAL RESEARCH; UNITED STATES;

CELL- AND TISSUE-BASED THERAPY; HUMANS; NATIONAL HEART, LUNG, AND BLOOD INSTITUTE (U.S.); PROGRAM DEVELOPMENT; RESEARCH SUPPORT AS TOPIC; TRANSLATIONAL MEDICAL RESEARCH; UNITED STATES;

EID: 84899624102     PISSN: 17528054     EISSN: 17528062     Source Type: Journal    
DOI: 10.1111/cts.12148     Document Type: Article

References (52)

1. Stroncek D, Harvath L, Barrett J. National Heart, Lung, and Blood Institute of the National Institutes of Health forum on immune reconstitution and cellular therapy following hematopoietic stem-cell transplantation. Cytotherapy. 2002; 4: 415-418.
2. Mondoro TH, Thomas JW, Henslee-Downey PJ, Peterson CM. NHLBI plans for the promise of cell-based therapies. Cytotherapy. 2005; 7: 317-327.
3. Reed W, Noga SJ, Gee AP, Rooney CM, Wagner JE, McCullough J, McKenna DH, Whiteside TL, Donnenberg AD, Baker AK, et al. Production Assistance for Cellular Therapies (PACT): Five-year experience from the United States National Heart Lung and Blood Institute (NHLBI) Contract Research Program in Cell and Tissue Therapies. Transfusion. 2009; 49(4): 786-796.
4. Traverse JH, Henry TD, Pepine CJ, Willerson JT, Zhao D, Ellis SG, Forder JR, Anderson RD, Hatzopoulos AK, Penn MS, et al. Effect of the use and timing of bone marrow mononuclear cell delivery on left ventricular function after acute myocardial infarction: The time randomized trial. JAMA. 2012; 308(22): 2380-2389.
5. Traverse JH, Henry TD, Ellis SG, Pepine CJ, Willerson J, Zhao D, Forder JR, Byrne BJ, Hatzopoulos AK, Penn MS, et al. Effect of intracoronary delivery of autologous bone marrow mononuclear cells 2 to 3 weeks following acute myocardial infarction on left ventricular function. JAMA. 2011; 306(19): 2110-2119.
6. Leen AM, Christin A, Myers GD, Liu H, Cruz CR, Hanley PJ, Kennedy-Nasser AA, Leung KS, Gee AP, Krance RA, et al. Cytotoxic T lymphocyte therapy with donor T cells prevents and treats adenovirus and Epstein-Barr virus infections after haploidentical and matched unrelated stem cell transplantation. Blood. 2009; 114(19): 4283-4292.
7. Leen AM, Myers GD, Sili U, Huls MH, Weiss H, Leung KS, Carrum G, Krance RA, Chang CC, Molldrem JJ, et al. Monoculture-derived T lymphocytes specific for multiple viruses expand and produce clinically relevant effects in immunocompromised individuals. Nat Med. 2006; 12: 1160-1166.
8. Gerdemann U, Vera JF, Rooney CM, Leen AM. Generation of multivirus-specific T cells to prevent/treat viral infections after allogeneic hematopoietic stem cell transplant. J Vis Exp. 2011; (51): e2736.
9. Gerdemann U, Christin AS, Vera JF, Ramos CA, Fujita Y, Liu H, Dilloo D, Heslop HE, Brenner MK, Rooney CM, et al. Nucleofection of DCs to generate Multivirus-specific T cells for prevention or treatment of viral infections in the immunocompromised host. Mol Ther. 2009; 17(9): 1616-1625.
10. Gerdemann U, Keirnan JM, Katari UL, Yanagisawa R, Christin AS, Huye LE, Perna SK, Ennamuri S, Gottschalk S, Brenner MK, et al. Rapidly generated multi-virus specific cytotoxic T lymphocytes for the prophylaxis and treatment of viral infections. Mol Ther. 2012; 20(8): 1622-1632.
11. Gerdemann U, Katari UL, Papadopoulou A, Keirnan JM, Craddock JA, Liu H, Martinez CA, Kennedy-Nasser A, Leung KS, Gottschalk SM, et al. Safety and clinical efficacy of rapidly-generated trivirus-directed T cells as treatment for adenovirus, EBV, and CMV infections after allogeneic hematopoietic stem cell transplant. Mol Ther. 2013; 21: 2113-2121.
12. Shlomchik WD, Couzens MS, Tang CB, McNiff J, Robert ME, Liu J, Shlomchik MJ, Emerson SG. Prevention of graft versus host disease by inactivation of host antigen-presenting cells. Science. 1999; 285(5426): 412-415.
13. Lundqvist A, McCoy JP, Samsel L, Childs R. Reduction of GVHD and enhanced antitumor effects after adoptive infusion of alloreactive Ly49-mismatched NK cells from MHC-matched donors. Blood. 2007; 109(8): 3603-3606.
14. Zaia JA, Sun JY, Gallez-Hawkins GM, Thao L, Oki A, Lacey SF, Dagis A, Palmer J, Diamond DJ, Forman SJ, et al. The effect of single and combined activating killer immunoglobulin-like receptor genotypes on cytomegalovirus infection and immunity after hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2009; 15(3): 315-325.
15. Miller JS, Soignier Y, Panoskaltsis-Mortari A, McNearney SA, Yun GH, Fautsch SK, McKenna D, Le C, Defor TE, Burns LJ, et al. Successful adoptive transfer and in vivo expansion of human haploidentical NK cells in patients with cancer. Blood. 2005; 105: 3051-3057.
16. McKenna DH Jr., Sumstad D, Bostrom N, Kadidlo DM, Fautsch S, McNearney S, Dewaard R, McGlave PB, Weisdorf DJ, Wagner JE, et al. Good manufacturing practices production of natural killer cells for immunotherapy: a six-year single-institution experience. Transfusion. 2007; 47(3): 520-528.
17. Rubenfeld GD, Caldwell E, Peabody E, Weaver J, Martin DP, Neff M, Stern EJ, Hudson LD. Incidence and Outcomes of Acute Lung Injury. N Engl J Med. 2005; 353(16): 1685-1693.
18. Gupta, N, Su X, Popov B, Lee JW, Serikov V, Matthay MA. Intrapulmonary delivery of bone marrow-derived mesenchymal stem cells improves survival and attenuates endotoxin-induced acute lung injury in mice. J Immuno. 2007 Aug 1; 179(3): 1855-1863.
19. Krause DS, Theise ND, Collector MI, Henegariu O, Hwang S, Gardner R, Neutzel S, Sharkis SJ. Multi-organ, multi-lineage engraftment by a single bone marrow-derived stem cell. Cell. 2001; 105(3): 369-377.
20. Lee JW, Krasnodembskaya A, McKenna DH, Song Y, Abbott J, Matthay MA. Therapeutic effects of human mesenchymal stem cells in ex vivo human lungs injured with live bacteria. Am J Respir Crit Care Med. 2013; 187(7): 751-760.
21. Massaad MJ, Ramesh N, Geha RS. Wiskott-Aldrich syndrome: a comprehensive review. Ann N Y Acad Sci. 2013; 1285(1): 26-43.
22. Galy A, Thrasher AJ. Gene therapy for the Wiskott-Aldrich syndrome. Curr Opin Allergy Clin Immunol. 2011; 11(6): 545-550.
23. Hanley PJ, Mei Z, daGraca Cabreira-Hansen M, Klis M, Li W, Zhao Y, Durett AG, Zheng X, Wang Y, Gee AP, et al. Manufacturing mesenchymal stromal cells for phase I clinical trials. Cytotherapy. 2013 Apr 15(4): 416-422.
24. Gee AP, Sumstad D, Stanson J, Watson P, Proctor J, Kadidlo D, Koch E, Sprague J, Wood D, Styers D, et al. A multicenter comparison study between the Endosafe® PTS™ rapid-release testing system and traditional test methods for detecting endotoxin in cell therapy products. Cytotherapy. 2008; 10(4): 427-435.
25. McKenna D Jr, Adams S, Sumstad D, Sumstad T, Kadidlo D, Gee AP, Durett A, Griffin D, Donnenberg A, Amrani D, et al. CD34+ cell selection using small volume marrow aspirates: a platform for novel cell therapies and regenerative medicine. Cytotherapy. 2010; 12: 170-177.
26. Whiteside T, Griffin D, Stanson J, Gooding W, McKenna D Jr., Sumstad D, Kadidlo D, Gee AP, Durett A, Lindblad R, et al. Shipping of therapeutic somatic cell products. Cytotherapy. 2011; 13: 201-213.
27. Stroncek D, Berlyne D, Fox B, Adrian Gee AP, Heimfeld S, Lindblad R, Loper K, McKenna D, Rooney C, Sabatino M, et al. Developments in clinical cell therapy. Cytotherapy 2010; 12: 425-428.
28. Sanchez R, Silberstein L, Lindblad R, Welniak L, Mondoro T, Wagner J. Strategies for more rapid translation of cellular therapies for children: A U.S. perspective. Pediatrics. 2013; 132(2): 351-358.
29. Adrian Gee (editor in chief) et al. Cell Therapy: cGMP Facilities and Manufacturing. Springer Publishing, 2009.
30. Lindblad R, Heath Mondoro T, Wood D. Preclinical Process of Cell-Based Therapies. Silberstein L editor: Hoffman: Hematology 6th edition: Basic Principles and Practice e-book: Chapter 97. Elsevier Inc., 2012.
31. Bollard CM, Gottschalk S, Huls MH, Leen AM, Gee AP Rooney C. Good manufacturing practice-grade cytotoxic T lymphocytes specific for latent membrane proteins (LMP)-1 and LMP2 for patients with Epstein-Barr virus-associated lymphoma. Cytotherapy. 2011; 13: 518-522.
32. Leen AM, Sili U, Savoldo B, Jewell AM, Piedra PA, Brenner MK, Rooney CM. Fiber-modified adenoviruses generate subgroup cross-reactive, adenovirus-specific cytotoxic T lymphocytes for therapeutic applications. Blood. 2004; 103: 1011-1019.
33. Sili U, Huls MH, Davis AR, Gottschalk S, Brenner MK, Heslop HE, Rooney CM. Large-scale expansion of dendritic cell-primed polyclonal human cytotoxic T-lymphocyte lines using lymphoblastoid cell lines for adoptive immunotherapy. J Immunother. 2003; 26: 241-256.
34. Bollard CM, Gottschalk S, Leen AM, Weiss H, Straathof KC, Carrum G, Khalil M, Wu MF, Huls MH, Chang CC, et al. Complete responses of relapsed lymphoma following genetic modification of tumor-antigen presenting cells and T-lymphocyte transfer. Blood. 2007; 110: 2838-2345.
35. Kennedy-Nasser AA, Bollard CM. T cell therapies following hematopoietic stem cell transplantation: surely there must be a better way than DLI? Bone Marrow Transplant. 2007; 40: 93-104.
36. Leen AM, Myers GD, Bollard CM, Huls MH, Sili U, Gee AP, Heslop HE, Rooney CM. T-cell immunotherapy for adenoviral infections of stem-cell transplant recipients. Ann N Y Acad Sci. 2005; 1062: 104-115.
37. Hanley PJ, Shaffer DR, Cruz CR, Ku S, Tzou B, Liu H, Demmler-Harrison G, Heslop HE, Rooney CM, Gottschalk S, et al. Expansion of T cells targeting multiple antigens of cytomegalovirus, Epstein-Barr virus and adenovirus to provide broad antiviral specificity after stem cell transplantation. Cytotherapy. 2011; 13: 976-86.
38. Sili U, Leen AM, Vera JF, Gee AP, Huls H, Heslop HE, Bollard CM, Rooney CM. Production of good manufacturing practice-grade cytotoxic T lymphocytes specific for Epstein-Barr virus, cytomegalovirus and adenovirus to prevent or treat viral infections post-allogeneic hematopoietic stem cell transplant. Cytotherapy. 2012; 14: 7-11.
39. Gerdemann U, Katari U, Christin AS, Cruz CR, Tripic T, Rousseau A, Gottschalk SM, Savoldo B, Vera JF, Heslop HE, et al. Cytotoxic T lymphocytes simultaneously targeting multiple tumor-associated antigens to treat EBV negative lymphoma. Mol Ther. 2011; 19(12): 2258-2268.
40. Harting MT, Baumgartner JE, Worth LL, Ewing-Cobbs L, Gee AP, Day MC, Cox CS Jr. Cell therapies for traumatic brain injuries. Neurosurgical Focus. 2008; 24(3-4): E18.
41. Cox CS Jr., Baumgartner JE, Harting MT, Worth LL, Walker PA, Shah SK, Ewing-Cobbs L, Hasan KM, Day MC, Lee D, et al. Autologous bone marrow mononuclear cell therapy for severe traumatic brain injury in children. Neurosurgery. 2011; 68: 588-600.
42. Traverse JH, Henry TD, Vaughan DE, Ellis SG, Pepine CJ, Willerson JT, Zhao DX, Piller LB, Penn MS, Byrne BJ, et al. Rationale and design for TIME: A phase II, randomized, double-blind, placebo-controlled pilot trial evaluating the safety and effect of timing of administration of bone marrow mononuclear cells after acute myocardial infarction. Am Heart J. 2009; 158: 356-363.
43. Traverse JH, Henry TD, Vaughan DE, Ellis SG, Pepine CJ, Willerson JT, Zhao DX, Simpson LM, Penn MS, Byrne BJ, et al. Late TIME: a phase-II, randomized, double-blinded, placebo-controlled, pilot trial evaluating the safety and effect of administration of bone marrow mononuclear cells 2 to 3 weeks after acute myocardial infarction. Tex Heart Inst J. 2010; 37: 412-420.
44. Blazar BR, Taylor PA. Regulatory T cells. Biol Blood Marrow Transplant. 2005; 11(2 Suppl 2): 46-49.
45. Godfrey WR, Ge YG, Spoden DJ, Levine BL, June CH, Blazar BR, Porter SB. In vitro-expanded human CD4(+)CD25(+) T-regulatory cells can markedly inhibit allogeneic dendritic cell-stimulated MLR cultures. Blood. 2004; 104: 453-461.
46. Godfrey WR, Spoden DJ, Ge YG, Baker SR, Liu B, Levine BL, June CH, Blazar BR, Porter SB. Cord blood CD4(+)CD25(+)-derived T regulatory cell lines express FoxP3 protein and manifest potent suppressor function. Blood. 2005; 105: 750-758.
47. Hippen KL, Merkel SC, Schirm DK, Sieben CM, Sumstad D, Kadidlo DM, McKenna DH, Bromberg JS, Levine BL, Riley JL, et al. Massive ex vivo expansion of human natural regulatory T cells (Tregs) with minimal loss of in vivo functional activity. Sci Transl Med. 2011; 3(83): 83ra41.
48. Brunstein CG, Blazar BR, Miller JS, Cao Q, Hippen K, McKenna DH, Wagner JE. Infusion of ex vivo expanded T regulatory cells in adults transplanted with umbilical cord blood: safety profile and detection kinetics. Blood. 2011 Jan 20; 117(3): 1061-1070.
49. Connolly NC, Riddler SA, Rinaldo CR. Proinflammatory cytokines in HIV disease-a review and rationale for new therapeutic approaches. AIDS Rev. 2005; 7: 168-180.
50. Whiteside TL, Piazza P, Reiter A, Stanson J, Connolly N, Rinaldo CR Jr., Riddler SA. Production of DC-based vaccine containing inactivated autologous virus for therapy of patients with chronic HIV-1 infection. Clin Vaccine Immunol. 2009; 16(2): 233-240.
51. Koepsell SA, Kadidlo DM, Fautsch S, McCullough J, Klingemann H, Wagner JE, Miller JS, -McKenna DH Jr. Successful "in-flight" activation of natural killer cells during long-distance shipping. Transfusion. 2013; 53(2): 398-403.
52. Klingemann H, Grodman C, Cutler E, Duque M, Kadidlo DM, Klein AK, Sprague KA, Miller KB, Comenzo RL, Kewalramani T, et al. Autologous stem cell transplant recipients tolerate haploidentical related-donor natural killer cell-enriched infusions. Transfusion. 2013; 53(2): 412-418.