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Chapter 12: Novel Treatment Options 245 Summary Gene therapy, stem cell therapy, and stem cell gene therapy are powerful tools that will improve care for Fanconi anemia patients in the future. We have learned from Fanconi anemia gene therapy trials that they will not be successful unless they are changed. The limitations relate to the sensitivity and paucity of hematopoietic stem cells available for correction. We should remain optimistic that the collective knowledge and unique enthusiasm of Fanconi anemia researchers and clinicians will provide a winning combination of ideas and well-designed experiments that will translate into improved care for Fanconi anemia transplant patients in the near future. References 1. Porteus MH, Connelly JP, Pruett SM. A look to future directions in gene therapy research for monogenic diseases. Public Library of Science Genetics 2006; 2: e133. 2. Ivics Z, Kaufman CD, Zayed H, Miskey C, Walisko O, Izsvak Z. The Sleeping Beauty transposable element: evolution, regulation and genetic applications. Current Issues in Molecular Biology 2004; 6: 43-55. 3. Baum C, Dullmann J, Li Z, et al. Side effects of retroviral gene transfer into hematopoietic stem cells. Blood 2003; 101: 2099- 2114. 4. Porteus MH, Baltimore D. Chimeric nucleases stimulate gene targeting in human cells. Science 2003; 300: 763. 5. Fischer A, Abina SH, Thrasher A, von Kalle C, Cavazzana- Calvo M. LMO2 and gene therapy for severe combined immunodeficiency. New England Journal of Medicine 2004; 350: 2526- 2527; author reply 2526-2527.
246 Fanconi Anemia: Guidelines for Diagnosis and Management 6. Hacein-Bey-Abina S, von Kalle C, Schmidt M, et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. New England Journal of Medicine 2003; 348: 255-256. 7. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, et al. LMO2- associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003; 302: 415-419. 8. McCormack MP, Forster A, Drynan L, Pannell R, Rabbitts TH. The LMO2 T-cell oncogene is activated via chromosomal translocations or retroviral insertion during gene therapy but has no mandatory role in normal T-cell development. Molecular and Cellular Biology 2003; 23: 9003-9013. 9. von Kalle C, Fehse B, Layh-Schmitt G, Schmidt M, Kelly P, Baum C. Stem cell clonality and genotoxicity in hematopoietic cells: gene activation side effects should be avoidable. Seminars in Hematology 2004; 41: 303-318. 10. Copelan EA. Hematopoietic stem-cell transplantation. New England Journal of Medicine 2006; 354: 1813-1826. 11. Daley GQ. Missed opportunities in embryonic stem-cell research. New England Journal of Medicine 2004; 351: 627-628. 12. Prockop DJ. “Stemness” does not explain the repair of many tissues by mesenchymal stem/multipotent stromal cells. Clinical Pharmacology and Therapeutics 2007; 82: 241-243. 13. Prockop DJ, Olson SD. Clinical trials with adult stem/progenitor cells for tissue repair: let’s not overlook some essential precautions. Blood 2007; 109: 3147-3151. 14. Le Blanc K, Rasmusson I, Sundberg B, et al. Treatment of severe acute graft-versus-host disease with third party haploidentical mesenchymal stem cells. The Lancet 2004; 363: 1439-1441. 15. Le Blanc K, Ringden O. Immunobiology of human mesenchymal stem cells and future use in hematopoietic stem cell transplantation. Biology of Blood and Marrow Transplantation 2005; 11: 321-334. 16. Le Blanc K, Samuelsson H, Gustafsson B, et al. Transplantation of mesenchymal stem cells to enhance engraftment of hematopoietic stem cells. Leukemia 2007; 21: 1733-1738.
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