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Pediatric Acute Lymphoblastic Leukemia Emerging Therapies—From Pathway to Target

Over the past 40 years, the 5-years-overall survival rate of pediatric cancer reached 75–80%, and for acute lymphoblastic leukemia (ALL), exceeded 90%. Leukemia continues to be a major cause of mortality and morbidity for specific patient populations, including infants, adolescents, and patients wit...

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Autores principales: Ivanov, Anca Viorica, Alecsa, Mirabela Smaranda, Popescu, Roxana, Starcea, Magdalena Iuliana, Mocanu, Adriana Maria, Rusu, Cristina, Miron, Ingrith Crenguta
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10003692/
https://www.ncbi.nlm.nih.gov/pubmed/36902091
http://dx.doi.org/10.3390/ijms24054661
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author Ivanov, Anca Viorica
Alecsa, Mirabela Smaranda
Popescu, Roxana
Starcea, Magdalena Iuliana
Mocanu, Adriana Maria
Rusu, Cristina
Miron, Ingrith Crenguta
author_facet Ivanov, Anca Viorica
Alecsa, Mirabela Smaranda
Popescu, Roxana
Starcea, Magdalena Iuliana
Mocanu, Adriana Maria
Rusu, Cristina
Miron, Ingrith Crenguta
author_sort Ivanov, Anca Viorica
collection PubMed
description Over the past 40 years, the 5-years-overall survival rate of pediatric cancer reached 75–80%, and for acute lymphoblastic leukemia (ALL), exceeded 90%. Leukemia continues to be a major cause of mortality and morbidity for specific patient populations, including infants, adolescents, and patients with high-risk genetic abnormalities. The future of leukemia treatment needs to count better on molecular therapies as well as immune and cellular therapy. Advances in the scientific interface have led naturally to advances in the treatment of childhood cancer. These discoveries have involved the recognition of the importance of chromosomal abnormalities, the amplification of the oncogenes, the aberration of tumor suppressor genes, as well as the dysregulation of cellular signaling and cell cycle control. Lately, novel therapies that have already proven efficient on relapsed/refractory ALL in adults are being evaluated in clinical trials for young patients. Tirosine kinase inhibitors are, by now, part of the standardized treatment of Ph+ALL pediatric patients, and Blinatumomab, with promising results in clinical trials, received both FDA and EMA approval for use in children. Moreover, other targeted therapies such as aurora-kinase inhibitors, MEK-inhibitors, and proteasome-inhibitors are involved in clinical trials that include pediatric patients. This is an overview of the novel leukemia therapies that have been developed starting from the molecular discoveries and those that have been applied in pediatric populations.
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spelling pubmed-100036922023-03-11 Pediatric Acute Lymphoblastic Leukemia Emerging Therapies—From Pathway to Target Ivanov, Anca Viorica Alecsa, Mirabela Smaranda Popescu, Roxana Starcea, Magdalena Iuliana Mocanu, Adriana Maria Rusu, Cristina Miron, Ingrith Crenguta Int J Mol Sci Review Over the past 40 years, the 5-years-overall survival rate of pediatric cancer reached 75–80%, and for acute lymphoblastic leukemia (ALL), exceeded 90%. Leukemia continues to be a major cause of mortality and morbidity for specific patient populations, including infants, adolescents, and patients with high-risk genetic abnormalities. The future of leukemia treatment needs to count better on molecular therapies as well as immune and cellular therapy. Advances in the scientific interface have led naturally to advances in the treatment of childhood cancer. These discoveries have involved the recognition of the importance of chromosomal abnormalities, the amplification of the oncogenes, the aberration of tumor suppressor genes, as well as the dysregulation of cellular signaling and cell cycle control. Lately, novel therapies that have already proven efficient on relapsed/refractory ALL in adults are being evaluated in clinical trials for young patients. Tirosine kinase inhibitors are, by now, part of the standardized treatment of Ph+ALL pediatric patients, and Blinatumomab, with promising results in clinical trials, received both FDA and EMA approval for use in children. Moreover, other targeted therapies such as aurora-kinase inhibitors, MEK-inhibitors, and proteasome-inhibitors are involved in clinical trials that include pediatric patients. This is an overview of the novel leukemia therapies that have been developed starting from the molecular discoveries and those that have been applied in pediatric populations. MDPI 2023-02-28 /pmc/articles/PMC10003692/ /pubmed/36902091 http://dx.doi.org/10.3390/ijms24054661 Text en © 2023 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Ivanov, Anca Viorica
Alecsa, Mirabela Smaranda
Popescu, Roxana
Starcea, Magdalena Iuliana
Mocanu, Adriana Maria
Rusu, Cristina
Miron, Ingrith Crenguta
Pediatric Acute Lymphoblastic Leukemia Emerging Therapies—From Pathway to Target
title Pediatric Acute Lymphoblastic Leukemia Emerging Therapies—From Pathway to Target
title_full Pediatric Acute Lymphoblastic Leukemia Emerging Therapies—From Pathway to Target
title_fullStr Pediatric Acute Lymphoblastic Leukemia Emerging Therapies—From Pathway to Target
title_full_unstemmed Pediatric Acute Lymphoblastic Leukemia Emerging Therapies—From Pathway to Target
title_short Pediatric Acute Lymphoblastic Leukemia Emerging Therapies—From Pathway to Target
title_sort pediatric acute lymphoblastic leukemia emerging therapies—from pathway to target
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10003692/
https://www.ncbi.nlm.nih.gov/pubmed/36902091
http://dx.doi.org/10.3390/ijms24054661
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