Acute Myeloid Leukemia Targeting by Chimeric Antigen Receptor T Cells: Bridging the Gap from Preclinical Modeling to Human Studies

MC Rotiroti, S Arcangeli, M Casucci, V Perriello, A Bondanza, Andrea Biondi, S Tettamanti, Ettore Biagi

Research output: Contribution to journalArticle

11 Citations (Scopus)

Abstract

Acute myeloid leukemia (AML) still represents an unmet clinical need for adult and pediatric high-risk patients, thus demanding advanced and personalized therapies. In this regard, different targeted immunotherapeutic approaches are available, ranging from naked monoclonal antibodies (mAb) to conjugated and multifunctional mAbs (i.e., BiTEs and DARTs). Recently, researchers have focused their attention on novel techniques of genetic manipulation specifically to redirect cytotoxic T cells endowed with chimeric antigen receptors (CARs) toward selected tumor associated antigens. So far, CAR T cells targeting the CD19 antigen expressed by B-cell origin hematological cancers have gained impressive clinical results, leading to the possibility of translating the CAR platform to treat other hematological malignancies such as AML. However, one of the main concerns in the field of AML CAR immunotherapy is the identification of an ideal target cell surface antigen, being highly expressed on tumor cells but minimally present on healthy tissues, together with the design of an anti-AML CAR appropriately balancing efficacy and safety profiles. The current review focuses mainly on AML target antigens and the related immunotherapeutic approaches developed so far, deeply dissecting methods of CAR T cell safety improvements, when designing novel CARs approaching human studies. © 2017, Mary Ann Liebert, Inc.
Original languageEnglish
Pages (from-to)231-241
Number of pages11
JournalHuman Gene Therapy
Volume28
Issue number3
DOIs
Publication statusPublished - 2017

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Antigen Receptors
T-Cell Antigen Receptor
Acute Myeloid Leukemia
CD19 Antigens
Safety
Genetic Techniques
Neoplasm Antigens
Hematologic Neoplasms
Surface Antigens
Immunotherapy
Neoplasms
Monoclonal Antibodies
Research Personnel
Pediatrics
T-Lymphocytes
Antigens

Cite this

Rotiroti, MC., Arcangeli, S., Casucci, M., Perriello, V., Bondanza, A., Biondi, A., ... Biagi, E. (2017). Acute Myeloid Leukemia Targeting by Chimeric Antigen Receptor T Cells: Bridging the Gap from Preclinical Modeling to Human Studies. Human Gene Therapy, 28(3), 231-241. https://doi.org/10.1089/hum.2016.092

Acute Myeloid Leukemia Targeting by Chimeric Antigen Receptor T Cells: Bridging the Gap from Preclinical Modeling to Human Studies. / Rotiroti, MC; Arcangeli, S; Casucci, M; Perriello, V; Bondanza, A; Biondi, Andrea; Tettamanti, S; Biagi, Ettore.

In: Human Gene Therapy, Vol. 28, No. 3, 2017, p. 231-241.

Research output: Contribution to journalArticle

Rotiroti, MC, Arcangeli, S, Casucci, M, Perriello, V, Bondanza, A, Biondi, A, Tettamanti, S & Biagi, E 2017, 'Acute Myeloid Leukemia Targeting by Chimeric Antigen Receptor T Cells: Bridging the Gap from Preclinical Modeling to Human Studies', Human Gene Therapy, vol. 28, no. 3, pp. 231-241. https://doi.org/10.1089/hum.2016.092
Rotiroti, MC ; Arcangeli, S ; Casucci, M ; Perriello, V ; Bondanza, A ; Biondi, Andrea ; Tettamanti, S ; Biagi, Ettore. / Acute Myeloid Leukemia Targeting by Chimeric Antigen Receptor T Cells: Bridging the Gap from Preclinical Modeling to Human Studies. In: Human Gene Therapy. 2017 ; Vol. 28, No. 3. pp. 231-241.
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