Advanced targeted, cell and gene-therapy approaches for pediatric hematological malignancies: results and future perspectives
- Department of Pediatrics, Centro di Ricerca Matilde Tettamanti, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy
Despite the survival of pediatric patients affected by hematological malignancies being improved in the last 20 years by chemotherapy and hematopoietic stem cell transplantation, a significant amount of patients still relapses. Treatment intensification is limited by toxic side effects and is constrained by the plateau of efficacy, while the pipeline of new chemotherapeutic drugs is running short. Therefore, novel therapeutic strategies are essential and researchers around the world are testing in clinical trials immune and gene-therapy approaches as second-line treatments. The aim of this review is to give a glance at these novel promising strategies of advanced medicine in the field of pediatric leukemias. Results from clinical protocols using new targeted “smart” drugs, immunotherapy, and gene therapy are summarized, and important considerations regarding the combination of these novel approaches with standard treatments to promote safe and long-term cure are discussed.
Keywords: advanced therapy, pediatric leukemia, novel drugs, adoptive immunotherapy, chimeric antigen receptor, gene therapy
Citation: Magnani CF, Tettamanti S, Maltese F, Turazzi N, Biondi A and Biagi E (2013) Advanced targeted, cell and gene-therapy approaches for pediatric hematological malignancies: results and future perspectives. Front. Oncol. 3:106. doi: 10.3389/fonc.2013.00106
Received: 30 January 2013; Accepted: 15 April 2013;
Published online: 30 April 2013.
, University Hospital for Childhood and Adolescence Medicine, Germany
Copyright: © 2013 Magnani, Tettamanti, Maltese, Turazzi, Biondi and Biagi. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits use, distribution and reproduction in other forums, provided the original authors and source are credited and subject to any copyright notices concerning any third-party graphics etc.
*Correspondence: Ettore Biagi, Department of Pediatrics, Centro di Ricerca Matilde Tettamanti, San Gerardo Hospital, University Milano-Bicocca, via Pergolesi 33, Monza 20052, Italy. e-mail: email@example.com