Miguel-Angel Perales - Cell and Gene Therapies

Advances and Controversies in Hematopoietic Transplantation and Cell Therapy

Series Editors

Syed A. Abutalib

Deerfield, Illinois, USA

James O. Armitage

Omaha, Nebraska, USA

Each volume will focus on different aspects of blood and marrow transplantation or cellular therapy and presents up-to-date data and concepts as well as controversial aspects.

More information about this series at http://www.springer.com/series/13907

Editors

Miguel-Angel Perales , Syed A. Abutalib and Catherine Bollard

Cell and Gene Therapies

Editors

Miguel-Angel Perales

Adult Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY, USA

Syed A. Abutalib

Cancer Treatment Centers of America, Deerfield, IL, USA

Catherine Bollard

Center for Cancer and Immunology Research Childrens National and The George Washington University, Division Allergy and Immunology, Washington, DC, USA

ISSN 2569-1376 e-ISSN 2569-135X

Advances and Controversies in Hematopoietic Transplantation and Cell Therapy

ISBN 978-3-319-54367-3 e-ISBN 978-3-319-54368-0

https://doi.org/10.1007/978-3-319-54368-0

Library of Congress Control Number: 2018957701

Springer Nature Switzerland AG 2019

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Contents

Miguel-Angel Perales and Catherine Bollard

Syed A. Abutalib and Saar I. Gill

Part IGenetically Modified T Cells (Chimeric Antigen Receptors and T Cell Receptors)

Januario E. Castro and Thomas J. Kipps

Mala K. Talekar and Stephan A. Grupp

Mark B. Geyer , Jae H. Park and Renier J. Brentjens

Yakup Batlevi and Craig S. Sauter

Aaron P. Rapoport and Jean A. Yared

Part IIBeyond Chimeric Antigen Receptor and T-Cell Receptors: Data, Consensus and Challenges

Maria A. V. Marzolini and Karl S. Peggs

Franziska Maria Uhl and Robert Zeiser

Part IIIBeyond T-Cells

Dhifaf Sarhan and Jeffery S. Miller

David J. Chung

John Barrett and Jacques Galipeau

Part IVGene Therapy

Januario E. Castro and Thomas J. Kipps

Xiuyan Wang and Isabelle Rivire

In the beginning days of blood banking, surgeons would call imperiously for fresh whole blood recognizing its superior restorative properties over banked blood. Since then technological advances have made it possible to break down the therapeutic elements of fresh blood into their constituent platelets, red cells, plasma, and clotting factors, and through apheresis, blood bankers can even provide granulocytes, lymphocytes, progenitors, and stem cells. The component therapy concept is so widely accepted that we cease to think it as being unusual. Curiously, and in contrast, transplant physicians have been slower to apply a component therapy approach to their practice. Even today the majority of hematopoietic cell transplantation (HCT), whether from the bone marrow, peripheral blood, or cord blood, is as unmanipulated as the fresh whole blood beloved of our surgeons of the past. Nevertheless, the attractions of a component therapy approach to HCT are many including but not limited to (1) T-cell depletion by selection of CD34+ cells, which can reduce GvHD, and (2) infused donor lymphocytes which can improve engraftment and treat leukemic relapse. Careful studies in the 1990s determined the doses of CD34+ cells and lymphocytes in the graft that led to the best outcomes, and donor lymphocyte infusion doses were calibrated to achieve graft-versus-leukemia effects with minimal graft-versus-host disease.

These initial graft manipulations contributed to steady progress to improving HCT outcome, extending the upper age limit of HCT recipients and paving the way for successful transplants from HLA-haploidentical mismatch donors. However, we now see these advances as merely a prelude to the full realization of the component therapy approach through modern cell and gene therapies. Advances in technology in translational research have opened up exciting and powerful new cell-based treatments which promise to dramatically transform the way we perform allogeneic HCT and eliminate the obstacles of GvHD, relapse, and transplant-related mortality (TRM).

In this volume, we review the exciting developments in cell and gene therapy as it relates to HCT. From blood or marrow, a diverse repertoire of cell products are now manufactured including mesenchymal stromal cells (Chap. ).

With the rapid advances in treatments of neoplastic disease and the prospect of continuing breakthroughs in treatments, as we have seen with the introduction of tyrosine kinase inhibitors and recently checkpoint inhibitors, we should be wary about predicting where HCT will be by the next decade. However, the rapid advances in cell therapy show a growing ability to render HCT safer and more effective. The progress documented with cell and gene therapy ensures that HCT will continue to remain central to the treatment of neoplastic and nonmalignant disorders for the foreseeable future.