title: Advanced targeted, cell and gene-therapy approaches for pediatric hematological malignancies: results and future perspectives.
abstract: 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.
authors: Department of Pediatrics, Centro di Ricerca Matilde Tettamanti, San Gerardo Hospital, University of Milano-Bicocca Monza, Italy.
authors: Chiara F
authors: CF
authors: Magnani
authors: Sarah
authors: S
authors: Tettamanti
authors: Francesca
authors: F
authors: Maltese
authors: Nice
authors: N
authors: Turazzi
authors: Andrea
authors: A
authors: Biondi
authors: Ettore
authors: E
authors: Biagi
keywords: adoptive immunotherapy; advanced therapy; chimeric antigen receptor; gene therapy; novel drugs; pediatric leukemia
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