Review

Targeted therapies in pediatric cancers

Volume: 40 Number: 3 July 30, 2026

Targeted therapies in pediatric cancers

Abstract

Abstract Childhood cancers remain one of the leading causes of disease-related mortality in the pediatric population. Although multi-agent chemotherapy protocols have significantly improved survival rates, outcomes remain suboptimal in a substantial proportion of patients with high-risk, relapsed, or refractory disease. In addition, the cumulative toxicity of conventional cytotoxic agents is associated with serious long-term morbidities. Advances in genomic profiling and molecular characterization have enhanced our understanding of the biology of leukemias and solid tumors, enabling the development of more targeted therapeutic strategies with reduced systemic toxicity. In recent years, immunotherapeutic agents such as blinatumomab and inotuzumab ozogamicin, along with chimeric antigen receptor-T cell (CAR-T) cell therapy, have fundamentally transformed the treatment paradigm for relapsed and refractory acute lymphoblastic leukemia (ALL). Furthermore, targeted therapies, including tyrosine kinase inhibitors and FLT3 inhibitors, have significantly improved survival when incorporated into conventional chemotherapy regimens in both acute myeloid leukemia and high-risk ALL. In solid tumors, immune checkpoint inhibitors, targeted small molecules, and antibody-based therapies have demonstrated promising results. This review aims to summarize the current evidence on targeted therapies in pediatric leukemias, lymphomas and solid tumors, highlight ongoing clinical trials, and discuss future perspectives regarding the integration of these agents into frontline treatment strategies.

Keywords

References

  1. 1. Farber, S., and Diamond, L.K. Temporary remissions in acute leukemia in children produced by folic acid antagonist, 4-aminopteroyl-glutamic acid. N Engl J Med. 1948 Jun 3;238(23):787-93.
  2. 2. Druker BJ, Talpaz M, Resta DJ, Peng B, Buchdunger E, Ford JM, et al. Efficacy and safety of a specific inhibitor of the BCR-ABL tyrosine kinase in chronic myeloid leukemia. N Engl J Med. 2001 Apr 5;344(14):1031-7.
  3. 3. Jabbour E, Short NJ, Jain N, Haddad FG, Welch MA, Ravandi F, et al. The evolution of acute lymphoblastic leukemia research and therapy at MD Anderson over four decades. J Hematol Oncol. 2023 Mar 16;16(1):22.
  4. 4. Kantarjian H, Pui CH, Jabbour E. Acute lymphocytic leukaemia. Lancet. 2025 Aug 30;406(10506):950-962.
  5. 5. Malczewska M, Kośmider K, Bednarz K, Ostapińska K, Lejman M, Zawitkowska J. Recent Advances in Treatment Options for Childhood Acute Lymphoblastic Leukemia. Cancers (Basel). 2022 Apr 16;14(8):2021.
  6. 6. Cohen MH, Williams G, Johnson JR, Duan J, Gobburu J, Rahman A, et al. Approval summary for Imatinib mesylate capsules in the treatment of chronic myelogenous leukemia. Clin Cancer Res. 2002;8(5):935–42.
  7. 7. Towatari M, Yanada M, Usui N, Takeuchi J, Sugiura I, Takeuchi M, et al. Japan Adult Leukemia Study Group. Combination of intensive chemotherapy and imatinib can rapidly induce high-quality complete remission for a majority of patients with newly diagnosed BCR-ABL-positive acute lymphoblastic leukemia. Blood. 2004 Dec 1;104(12):3507-12.
  8. 8. Jain N, Roberts KG, Jabbour E, Patel K, Eterovic AK, Chen K, et al. Ph-like acute lymphoblastic leukemia: a high-risk subtype in adults. Blood. 2017 Feb 2;129(5):572-581.

Details

Primary Language

English

Subjects

Clinical Oncology

Journal Section

Review

Publication Date

July 30, 2026

Submission Date

May 31, 2026

Acceptance Date

June 18, 2026

Published in Issue

Year 2026 Volume: 40 Number: 3

APA
Okur Acar, S., Kızmazoğlu, D., & Tüfekçi Gürocak, Ö. (2026). Targeted therapies in pediatric cancers. Developments and Experiments in Health and Medicine, 40(3), 301-316. https://doi.org/10.18614/dehm.1955635
AMA
1.Okur Acar S, Kızmazoğlu D, Tüfekçi Gürocak Ö. Targeted therapies in pediatric cancers. Dev Exp Health Med. 2026;40(3):301-316. doi:10.18614/dehm.1955635
Chicago
Okur Acar, Sultan, Deniz Kızmazoğlu, and Özlem Tüfekçi Gürocak. 2026. “Targeted Therapies in Pediatric Cancers”. Developments and Experiments in Health and Medicine 40 (3): 301-16. https://doi.org/10.18614/dehm.1955635.
EndNote
Okur Acar S, Kızmazoğlu D, Tüfekçi Gürocak Ö (July 1, 2026) Targeted therapies in pediatric cancers. Developments and Experiments in Health and Medicine 40 3 301–316.
IEEE
[1]S. Okur Acar, D. Kızmazoğlu, and Ö. Tüfekçi Gürocak, “Targeted therapies in pediatric cancers”, Dev Exp Health Med, vol. 40, no. 3, pp. 301–316, July 2026, doi: 10.18614/dehm.1955635.
ISNAD
Okur Acar, Sultan - Kızmazoğlu, Deniz - Tüfekçi Gürocak, Özlem. “Targeted Therapies in Pediatric Cancers”. Developments and Experiments in Health and Medicine 40/3 (July 1, 2026): 301-316. https://doi.org/10.18614/dehm.1955635.
JAMA
1.Okur Acar S, Kızmazoğlu D, Tüfekçi Gürocak Ö. Targeted therapies in pediatric cancers. Dev Exp Health Med. 2026;40:301–316.
MLA
Okur Acar, Sultan, et al. “Targeted Therapies in Pediatric Cancers”. Developments and Experiments in Health and Medicine, vol. 40, no. 3, July 2026, pp. 301-16, doi:10.18614/dehm.1955635.
Vancouver
1.Sultan Okur Acar, Deniz Kızmazoğlu, Özlem Tüfekçi Gürocak. Targeted therapies in pediatric cancers. Dev Exp Health Med. 2026 Jul. 1;40(3):301-16. doi:10.18614/dehm.1955635