Ernst Schering Research Foundation Workshop Supplement 11
Regenerative and Cell Therapy
Ernst Schering Research Foundation Workshop Supplement 11
Regenerative and Cell Therapy
Clinical Advances
A. Keating, K. Dicke, N. Gorin, R. Weber, H. Graf Editors
With 13 Figures
1 2
Series Editors: G. Stock and M. Lessl
Library of Congress Control Number: 2004105981 ISSN 1431-7133
ISBN 3-540-22093-3 Springer Berlin Heidelberg New York
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Preface
Clinical success in hematopoietic stem cell transplantation have paved the way into the new world of regenerative medicine. Open is- sues important for the translation of scientific achievements into therapeutic strategies have been debated on different levels: stem cell biology, manufacturing and clinical use.
In the face of all ongoing laboratory research, investigators have proceeded, some quietly, some with fanfare to conduct clinical trials of cell therapy for a wide variety of human ailments.
This book, inspired by a symposium on regenerative and cell ther- apy held at the Sorbonne in Paris in 2003, attempts to evaluate the clinical advances in this area in the context of earlier or contempora- neous research with in vitro and animal models as well as preclini- cal studies.
We believe that an evaluation of clinical progress in cell therapy is timely, may help to identify issues that require more preclinical investigation before proceeding to clinical trials, or importantly, identify areas that are ripe for further immediate clinical interven- tion. Similarly, bedside to bench issues may become more readily apparent and may foster the investigative loop that occasionally moves clinical development rapidly to the next level. The juxtaposi- tion of regenerative cell therapy, especially for damaged myocar- dium, with the longer established clinical experience in anticancer cell therapy, such as allotransplantation and adoptive immunother- apy, is deliberate ± we hope that mutually instructive insights will arise.
Finally, we are most grateful to all the authors, who are interna- tional leaders in the field, for their thoughtful contributions.
Armand Keating
On behalf of coeditors Karel Dicke, Norbert Gorin, Renate Weber, and Hermann Graf.
VI Preface
Contents
1 Problems and Hopes with Cell Therapy:
The Case of Muscular Dystrophy
G. Cossu . . . . 1 2 Cardiac Myoblasts
P. Menasch . . . . 21 3 Tissue Engineering, Stem Cells,
and Cloning: Current Concepts and Future Trends
C.J. Koh, A. Atala . . . . 35 4 Bone Marrow Cell Therapy
for Genetic Disorders of Bone
E.M. Horwitz, W.W.K. Koo, L.A. Fitzpatrick, M.D. Neel, P.L. Gordon . . . . 69 5Immune-Based Therapy for Acute
and Chronic Neurodegenerative Disorders
M. Schwartz . . . . 81 6 Mesenchymal Stem Cells for the Treatment
of Hematological Malignancies
Role in Hematopoietic Stem Cell Transplantation and Bone Marrow Failure
L. Fouillard . . . . 97
7 Perspectives in Transfusion Medicine for the Ex Vivo Control of Hematopoiesis
L. Douay . . . . 109 8 T-Cell Therapies for EBV-Associated Malignancies
M.K. Brenner, C. Bollard, M.H. Huls, S. Gottschalk,
H. E. Heslop, C.M. Rooney . . . . 121 9 Peptide Vaccination of Myeloid Leukemia
D. Kurbegov, J.J. Molldrem . . . . 131 10 Hematopoietic Cell Transplantation After
Nonmyeloablative Conditioning
M.L. Sorror, R. Storb . . . . 145 Previous Volumes Published in This Series . . . . 177
VIII Contents
List of Editors and Contributors
Editors Keating, A.
Princess Margaret Hospital, Ontario Cancer Institute, Toronto, Ontario, Canada
e-mail: armand.keating@uhn.on.ca
Coeditors Dicke, K.
Arlington Cancer Center, Arlington, TX, USA e-mail: kdicke@acctx.com
Gorin, N.
Hospital Saint-Antoine, Paris, France
e-mail: norbert-claude.gorin@sat.ap-hop-paris.fr Weber, R.
Schering AG, Mçllerstr. 178, 13342 Berlin, Germany e-mail: renate.weber@schering.de
Graf, H.
Schering AG, Mçllerstr. 178, 13342 Berlin, Germany e-mail: hermann.graf@schering.de
Contributors
Atala, A.
Wake Forest University School of Medicine, Department of Urology, Medical Center Boulevard, Winston Salem, NC 27157, USA
e-mail: a.atala@tch.harvard.edu Bollard, C.
Center for Cell and Gene Therapy, Baylor College of Medicine, The Methodist Hospital and Texas Children's Hospital, Houston, TX 77030, USA
e-mail: cmbollar@txccc.org Brenner, M.K.
Section of Hematology-Oncology, Baylor College of Medicine, Center for Cell and Gene Therapy, 6621 Fannin Street, 3-3320, Houston, TX 77030-2303, USA
e-mail: mbrenner@bcm.tmc.edu Cossu, G.
Stem Cell Research Institute, Via Olgettina 58, 20132 Milan, Italy e-mail: cossu.giulio@hsr.it
Douay, L.
HÖpital d'Enfants, Armand-Trousseau, 26 Avenue du Docteur A. Netter, Cedex 12, 75571 Paris, France
e-mail: luc.douay@trs.ap-hop-paris.fr Fitzpatrick, L.A.
Endocrine Research Unit, Department of Endocrinology, Diabetes, Metabolism, Nutrition and Internal Medicine, Mayo Clinic, 200 First Street SW, Rochester, MN 55905, USA
e-mail: fitz@mayo.edu Fouillard, L.
Service des Maladies du Sang, HÖpital Saint Antoine, 184 rue du Faubourg Saint Antoine, 75012 Paris, France e-mail: fouillar@ext.jussieu.fr
X List of Editors and Contributors
Gordon, P.L.
Divisions of Stem Cell Transplantation and Experimental Hematology, St. Jude Children's Research Hospital, 332 N. Lauderdale, Memphis, TN 38105, USA e-mail: patricia.gordon@stjude.org
Heslop, H.E.
Baylor College of Medicine, Center for Cell and Gene Therapy, 6621 Fannin Street, 3-3320, Houston, TX 77030-2303, USA e-mail: hheslop@bcm.tmc.edu
Horwitz, E.M.
Divisions of Stem Cell Transplantation and Experimental Hematology, St. Jude Children's Research Hospital, 332 N. Lauderdale, Memphis, TN 38105, USA e-mail: Edwin.horwitz@stjude.org
Huls, M.H.
Baylor College of Medicine, Center for Cell and Gene Therapy, 6621 Fannin Street, 3-3320, Houston, TX 77030-2303, USA e-mail: hhuls@bcm.tmc.edu
Koh, C.J.
Institute for Regenerative Medicine, Wake Forest University School of Medicine, Medical Center Blvd, Winston Salem, NC 27157, USA e-mail: c.koh@tch.harvard.edu
Koo, W.W.K.
Wayne State University, Detroit, Michigan, USA e-mail: wkoo@wayne.edu
Kurbegov, D.
MD Anderson Cancer Center, 1515 Holcome Boulevard, Houston, TX 77030, USA
e-mail: dkurbego@mail.mdanderson.org Menasch, P.
Service de Chirurgie Cardio Vasculaire, HÖpital Bichat, 46 rue Henri Huchard, 75018 Paris, France
e-mail: philippe.menasche@bch.ap-hop-paris.fr
List of Editors and Contributors XI
Molldrem, J.
MD Anderson Cancer Center, 1515 Holcome Boulevard, Houston, TX 77030, USA
e-mail: jmolldre@mail.mdanderson.org Neel, M.D.
Divisions of Stem Cell Transplantation and Experimental Hematology, St. Jude Children's Research Hospital, 332 N. Lauderdale, Memphis, TN 38105, USA e-mail: mneel@wnm.net
Rooney, C. M.
Baylor College of Medicine, Center for Cell and Gene Therapy, 6621 Fannin Street, 3-3320, Houston, TX 77030-2303, USA e-mail: crooney@bcm.tmc.edu
Schwartz, M.
Department of Neurobiology, Building for Brain Research,
Weizmann Institute of Science, P.O. Box 26, Rehovot 76100, Israel e-mail: michal.Schwartz@weizmann.ac.il
Storb, R.
Fred Hutchinson Cancer Research Center, 1100 Fairview Avenue North, D1-100, P.O. Box 19024, Seattle, WA 98109-1024, USA e-mail: rstorb@fhcrc.org
XII List of Editors and Contributors
