4.4. Conclusions. References

Principles of stem cell biology and cancer: future applications and therapeutics. Edited by T. Regad, T. J. Sayers and R. C. Rees. John Wiley & Sons (2015)

Part I. Stem Cells


Bone marrow transplantation is an incredible success story in the therapeutic use of stem cells in treating cancer. It has an exciting future, as continued improvements in the procedures will widen the therapeutic applications for which HSCs can be administered routinely. However, for many of these indications, routine clinical use is still far from reality. Continued development and properly designed clinical trials will be needed to bring novel indications to routine and effective clinical use. Care has to be taken with shortcuts, however tempting these may be. Only methodical approaches can delineate definitively useful approaches from ones that aren’t.

References

Aarts, M., Te Riele, H. 2011. Progress and prospects: oligonucleotide-directed gene modification in mouse embryonic stem cells: a route to therapeutic application. Gene Ther. 18, 213 – 219.

Akashi, K., Traver, D., Miyamoto, T., Weissman, I.L. 2000. A clonogenic common myeloid progenitor that gives rise to all myeloid lineages. Nature 404, 193 – 197.

Arber, C., BitMansour, A., Sparer, T.E., Higgins, J.P., Mocarski, E.S., Weissman, I.L., et al., 2003. Common lymphoid progenitors rapidly engraft and protect against lethal murine cytomegalovirus infection after hematopoietic stem cell transplantation. Blood 102, 421 – 428.

Arber, C., Bitmansour, A., Shashidhar, S., Wang, S., Tseng, B., Brown, J.M. 2005. Protection against lethal Aspergillus fumigatus infection in mice by allogeneic myeloid progenitors is not major histocompatibility complex restricted. J. Infect. Dis. 192, 1666 – 1671.

Bar, M., Sandmaier, B.M., Inamoto, Y., Bruno, B., Hari, P., Chauncey, T., et al., 2013. Donor lymphocyte infusion for relapsed hematological malignancies after  allogeneic hematopoietic cell transplantation: prognostic relevance of the initial CD3+ T cell dose. Biol. Blood Marrow Trans. 19, 949 – 957.

BitMansour, A., Burns, S.M., Traver, D., Akashi, K., Contag, C.H., Weissman, I.L., Brown, J.M. 2002. Myeloid progenitors protect against invasive aspergillosis and Pseudomonas aeruginosa infection following hematopoietic stem cell transplantation. Blood 100, 4660 – 4667.

Bluestone, J.A. 2011. Mechanisms of tolerance. Immunol. Rev. 241, 5 – 19.

Boelens, J.J., Rocha, V., Aldenhoven, M., Wynn, R., O’Meara, A., Michel, G., et al., 2009. Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome. Biol. Blood Marrow Trans. 15, 618 – 625.

Cattaneo, D., Perico, N., Gaspari, F., Remuzzi, G. 2004. Nephrotoxic aspects of cyclosporine. Transplant Proc. 36, 234S – 239S.

Cellerant Therapeutics. 2012. Cellerant Therapeutics initiates a Phase I/II clinical trial of CLT-008 for chemotherapy induced neutropenia in acute leukemia patients. Available from: http://www.cellerant.com/pr_032211.html (last accessed 28 November, 2014).

Chambers, S.M., Studer, L. 2011. Cell fate plug and play: direct reprogramming and induced pluripotency. Cell 145, 827 – 830.

Chang, Y.J., Huang, X.J. 2013. Donor lymphocyte infusions for relapse after allogeneic transplantation: when, if and for whom? Blood Rev. 27, 55 – 62.

Chen, B.J., Cui, X., Sempowski, G.D., Liu, C., Chao, N.J. 2004. Transfer of allogeneic CD62L-memory T cells without graft-versus-host disease. Blood 103, 1534 – 1541. Chen, B.J., Deoliveira, D., Cui, X., Le, N.T., Son, J., Whitesides, J.F., Chao, N.J. 2006.

Inability of memory T cells to induce graft-versus-host disease is a result of an abortive alloresponse. Blood 109(7), 3115 – 3123.

Clements, W.K., Traver, D. 2013. Signalling pathways that control vertebrate haematopoietic stem cell specification. Nat. Rev. Immunol. 13, 336 – 348.

Coleman, M.A., Steptoe, R.J. 2012. Induction of antigen-specific tolerance through hematopoietic stem cell-mediated gene therapy: the future for therapy of autoimmune disease? Autoimmun. Rev. 12, 195 – 203.

Czechowicz, A., Kraft, D., Weissman, I.L., Bhattacharya, D. 2007. Efficient transplantation via antibody-based clearance of hematopoietic stem cell niches. Science 318, 1296 – 1299.

Dalal, J., Gandy, K., Domen, J. 2012. Role of mesenchymal stem cell therapy in Crohn’s disease. Pediatr. Res. 71, 445 – 451.

Davila, M.L., Riviere, I., Wang, X., Bartido, S., Park, J., Curran, K., et al., 2014. Efficacy and toxicity management of 19-28z CAR T cell therapy in B cell acute lymphoblastic leukemia. Sci. Transl. Med. 6, 224ra25.

Deol, A., Lum, L.G. 2010. Role of donor lymphocyte infusions in relapsed hematological malignancies after stem cell transplantation revisited. Cancer Treat. Rev. 36, 528 – 538.

Domen, J., Weissman, I.L. 2000. Hematopoietic stem cells need two signals to prevent apoptosis; BCL-2 can provide one of these, Kitl/c-Kit signaling the other. J. Exp. Med. 192, 1707 – 1718.

Domen, J., Sun, L., Trapp, K., Maghami, N., Inagaki, E., Li, Y., et al., 2011. Tolerance induction by hematopoietic cell transplantation: combined use of stem cells and progenitor cells. J. Heart Lung Transplant, 30, 507 – 514.

Domen, J., Gandy, K., Dalal, J. 2012. Emerging uses for pediatric hematopoietic stem cells. Pediatr. Res. 71, 411 – 417.

Fagioli, F., Quarello, P., Zecca, M., Lanino, E., Rognoni, C., Balduzzi, A., et al., 2013. Hematopoietic stem cell transplantation for children with high-risk acute lymphoblastic leukemia in first complete remission: a report from the AIEOP registry. Haematologica 98, 1273 – 1281.

Gandy, K.L., Domen, J., Aguila, H., Weissman, I.L. 1999. CD8+TCR+ and CD8+TCRcells in whole bone marrow facilitate the engraftment of hematopoietic stem cells across allogeneic barriers. Immunity 11, 579 – 590.

Gandy, K., Domen, J., Copeland, J. 2007. Tolerance in heart transplantation: current and future role. In: Watson, R.R., Larson, D.F. (eds.) Immune Dysfunction and Immunotherapy in Heart Disease. Oxford: Blackwell.

Gibson, B.E., Wheatley, K., Hann, I.M., Stevens, R.F., Webb, D., Hills, R.K., et al., 2005. Treatment strategy and long-term results in paediatric patients treated in consecutive UK AML trials. Leukemia 19, 2130 – 2138.

Gillet, J.P., Macadangdang, B., Fathke, R.L., Gottesman, M.M., Kimchi-Sarfaty, C. 2009. The development of gene therapy: from monogenic recessive disorders to complex diseases such as cancer. Methods Mol. Biol. 542, 5 – 54.

Gratwohl, A., Baldomero, H., Aljurf, M., Pasquini, M.C., Bouzas, L.F., Yoshimi, A., et al., 2010. Hematopoietic stem cell transplantation: a global perspective. JAMA 303, 1617 – 1624.

Gross, T.G., Hale, G.A., He, W., Camitta, B.M., Sanders, J.E., Cairo, M.S., et al., 2010. Hematopoietic stem cell transplantation for refractory or recurrent non-Hodgkin lymphoma in children and adolescents. Biol. Blood Marrow Trans. 16, 223 – 230.

Hamidieh, A.A., Ansari, S., Darbandi, B., Soroush, A., Arjmandi Rafsanjani, K., Alimoghaddam, K., et al., 2013. The treatment of children suffering from chronic myelogenous leukemia: a comparison of the result of treatment with imatinib mesylate and allogeneic hematopoietic stem cell transplantation. Pediatr. Trans. 17, 380 – 386.

HHS/HRSA/HSB/DOT. 2009. OPTN/SRTR annual report 1999 – 2008. Available from: http://www.ustransplant.org/annual_reports/current/ (last accessed 28 November 2014).

Kalil, A.C., Dakroub, H., Freifeld, A.G. 2007. Sepsis and solid organ transplantation. Curr. Drug Targets 8, 533 – 541.

Kay, M.A. 2011. State-of-the-art gene-based therapies: the road ahead. Nat. Rev. Genet. 12, 316 – 328.

Kelly, M.J., Horan, J.T., Alonzo, T.A., Eapen, M., Gerbing, R.B., He, W., et al., 2014. Comparable survival for pediatric acute myeloid leukemia with poor-risk cytogenetics following chemotherapy, matched related donor, or unrelated donor transplantation. Pediatr. Blood Cancer 61, 269 – 275.

Leatherman, J. 2013. Stem cells supporting other stem cells. Front Genet. 4, 257. Leen, A.M., Christin, A., Myers, G.D., Liu, H., Cruz, C.R., Hanley, P.J., et al., 2009. 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 114, 4283 – 4292.

Leventhal, J., Abecassis, M., Miller, J., Gallon, L., Ravindra, K., Tollerud, D.J., et al., 2012. Chimerism and tolerance without GVHD or engraftment syndrome in HLA-mismatched combined kidney and hematopoietic stem cell transplantation. Sci. Transl. Med. 4, 124ra28.

Leventhal, J., Abecassis, M., Miller, J., Gallon, L., Tollerud, D., Elliott, M.J., et al., 2013a. Tolerance induction in HLA disparate living donor kidney transplantation by donor stem cell infusion: durable chimerism predicts outcome. Transplantation 95, 169 – 176.

Leventhal, J., Miller, J., Abecassis, M., Tollerud, D.J., Ildstad, S.T. 2013b. Evolving approaches of hematopoietic stem cell-based therapies to induce tolerance to organ transplants: the long road to tolerance. Clin. Pharmacol. Ther. 93, 36 – 45.

Loh, M.L. 2011. Recent advances in the pathogenesis and treatment of juvenile myelomonocytic leukaemia. Br. J. Haematol. 152, 677 – 687.

Louis, C.U., Savoldo, B., Dotti, G., Pule, M., Yvon, E., Myers, G.D., et al., 2011. Antitumor activity and long-term fate of chimeric antigen receptor-positive T cells in patients with neuroblastoma. Blood 118, 6050 – 6056.

Manz, M.G., Miyamoto, T., Akashi, K., Weissman, I.L. 2002. Prospective isolation of human clonogenic common myeloid progenitors. Proc. Nat. Acad. Sci. USA 99, 11 872 – 11 877.

Mathй, G., Jammet, H., Pendic, B., Schwarzenberg, L., Duplan, J.F., Maupin, B., et al., 1959. [Transfusions and grafts of homologous bone marrow in humans after accidental high dosage irradiation.] Rev. Fr. Etud. Clin. Biol. 4, 226 – 238.

Michael, M., Shimoni, A., Nagler, A. 2013. Regulatory T cells in allogeneic stem cell transplantation. Clin. Dev. Immunol. 2013, 608951.

Miura, Y., Yoshioka, S., Yao, H., Takaori-Kondo, A., Maekawa, T., Ichinohe, T. 2013. Chimerism of bone marrow mesenchymal stem/stromal cells in allogeneic hematopoietic cell transplantation: is it clinically relevant? Chimerism, 4, 78 – 83.

Mueller, A.R., Platz, K.P., Schattenfroh, N., Bechstein, W.O., Christe, W., Neuhaus, P. 1994. Neurotoxicity after orthotopic liver transplantation in cyclosporin Aand FK 506-treated patients. Transpl. Int. 7(Suppl. 1), S37 – S42.

Muller, A.M., Kohrt, H.E., Cha, S., Laport, G., Klein, J., Guardino, A.E., et al., 2012. Long-term outcome of patients with metastatic breast cancer treated with high-dose chemotherapy and transplantation of purified autologous hematopoietic stem cells. Biol. Blood Marrow Trans. 18, 125 – 133.

Obokata, H., Wakayama, T., Sasai, Y., Kojima, K., Vacanti, M.P., Niwa, H., et al., 2014. Stimulus-triggered fate conversion of somatic cells into pluripotency. Nature 505, 641 – 7.

Pasquini, M.C., Wang, Z. 2012. Current use and outcome of hematopoietic stem cell transplantation: CIBMTR summary slides. Available from: http://www.cibmtr.org (last accessed 28 November 2014).

Prasad, V.K., Kurtzberg, J. 2010a. Cord blood and bone marrow transplantation in inherited metabolic diseases: scientific basis, current status and future directions. Br. J. Haematol. 148, 356 – 372.

Prasad, V.K., Kurtzberg, J. 2010b. Transplant outcomes in mucopolysaccharidoses. Semin. Hematol. 47, 59 – 69.

Pulsipher, M.A., Peters, C., Pui, C.H. 2011. High-risk pediatric acute lymphoblastic leukemia: to transplant or not to transplant? Biol. Blood Marrow Trans. 17, S137 – S148.

Rabusin, M., Andolina, M., Maximova, N. 2008. Haematopoietic SCT in autoimmune diseases in children: rationale and new perspectives. Bone Marrow Trans. 41(Suppl. 2), S96 – S99.

Reiff, A., Shaham, B., Weinberg, K.I., Crooks, G.M., Parkman, R. 2011. Anti-CD52 antibody-mediated immune ablation with autologous immune recovery for the treatment of refractory juvenile polymyositis. J. Clin. Immunol. 31, 615 – 622.

Sachs, D.H., Sykes, M., Kawai, T., Cosimi, A.B. 2011. Immuno-intervention for the induction of transplantation tolerance through mixed chimerism. Semin. Immunol. 23, 165 – 173.

Scandling, J.D., Busque, S., Dejbakhsh-Jones, S., Benike, C., Sarwal, M., Millan, M.T., et al., 2012. Tolerance and withdrawal of immunosuppressive drugs in patients given kidney and hematopoietic cell transplants. Am. J. Transplant. 12, 1133 – 1145.

Schmitz, N., Pfistner, B., Sextro, M., Sieber, M., Carella, A.M., Haenel, M., et al., 2002. Aggressive conventional chemotherapy compared with high-dose chemotherapy with autologous haemopoietic stem-cell transplantation for relapsed chemosensitive Hodgkin’s disease: a randomised trial. Lancet 359, 2065 – 2071.

Singh, V.K., Christensen, J., Fatanmi, O.O., Gille, D., Ducey, E.J., Wise, S.Y., et al., 2012. Myeloid progenitors: a radiation countermeasure that is effective when initiated days after irradiation. Radiat. Res. 177, 781 – 791.

Spangrude, G.J., Heimfeld, S., Weissman, I.L. 1988. Purification and characterization of mouse hematopoietic stem cells. Science 241, 58 – 62.

Spooncer, E., Lord, B.I., Dexter, T.M. 1985. Defective ability to self-renew in vitro of highly purified primitive haematopoietic cells. Nature 316, 62 – 64.

Strober, S., Spitzer, T.R., Lowsky, R., Sykes, M. 2011. Translational studies in hematopoietic cell transplantation: treatment of hematologic malignancies as a stepping stone to tolerance induction. Semin. Immunol. 23, 273 – 281.

Sullivan, K.M., Muraro, P., Tyndall, A. 2010. Hematopoietic cell transplantation for autoimmune disease: updates from Europe and the United States. Biol. Blood Marrow Trans. 16, S48 – S56.

Sykes, M. 2009. Hematopoietic cell transplantation for tolerance induction: animal models to clinical trials. Transplantation 87, 309 – 316.

Takahashi, K., Tanabe, K., Ohnuki, M., Narita, M., Ichisaka, T., Tomoda, K., Yamanaka, S. 2007. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 131, 861 – 872.

Thomas, E.D., Lochte, H.L. Jr, Lu, W.C., Ferrebee, J.W. 1957. Intravenous infusion of bone marrow in patients receiving radiation and chemotherapy. N. Engl. J. Med. 257, 491 – 496.

Thomson, K.J., Peggs, K.S., Smith, P., Cavet, J., Hunter, A., Parker, A., et al., 2008. Superiority of reduced-intensity allogeneic transplantation over conventional treatment for relapse of Hodgkin’s lymphoma following autologous stem cell transplantation. Bone Marrow Trans. 41, 765 – 770.

Tomblyn, M., Lazarus, H.M. 2008. Donor lymphocyte infusions: the long and winding road: how should it be traveled? Bone Marrow Trans. 42, 569 – 579.

Tyndall, A. 2012. Application of autologous stem cell transplantation in various adult and pediatric rheumatic diseases. Pediatr. Res. 71, 433 – 438.

Vajdic, C.M., Van Leeuwen, M.T. 2009. Cancer incidence and risk factors after solid organ transplantation. Int. J. Cancer 125, 1747 – 1754.

Valayannopoulos, V., Wijburg, F.A. 2011. Therapy for the mucopolysaccharidoses. Rheumatology (Oxford), 50(Suppl. 5), v49 – v59.

Valayannopoulos, V., De Blic, J., Mahlaoui, N., Stos, B., Jaubert, F., Bonnet, D., et al., 2010. Laronidase for cardiopulmonary disease in Hurler syndrome 12 years after bone marrow transplantation. Pediatrics, 126, e1242-e1247.

Wang, H., Yang, H., Shivalila, C.S., Dawlaty, M.M., Cheng, A.W., Zhang, F., Jaenisch, R. 2013. One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering. Cell 153, 910 – 918.

Wei, C., Liu, J., Yu, Z., Zhang, B., Gao, G., Jiao, R. 2013. TALEN or Cas9 – rapid, efficient and specific choices for genome modifications. J. Genet. Genomics 40, 281 – 289.

Yoshida, N., Doisaki, S., Kojima, S. 2012. Current management of juvenile myelomonocytic leukemia and the impact of RAS mutations. Paediatr. Drugs 14, 157 – 163.

Yu, J., Vodyanik, M.A., Smuga-Otto, K., Antosiewicz-Bourget, J., Frane, J.L., Tian, S., et al., 2007. Induced pluripotent stem cell lines derived from human somatic cells. Science 318, 1917 – 1920.

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