Neo-antigen specific cancer vaccines for acute lymphoblastic leukemia—challenges, opportunities, and future directions

Abstract Acute lymphoblastic leukemia (ALL) is the most common pediatric malignancy, with standard treatment consisting of intensive chemotherapy and corticosteroids. While curative in most cases, this regimen leads to significant toxicity and long-term sequelae. Recent advancements in cancer immuno...

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Main Authors: Victoria Bloch Blytt Sandstad, Signe Modvig, Morten Orebo Holmström
Format: Article
Language:English
Published: Springer 2025-06-01
Series:Cancer Immunology, Immunotherapy
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Online Access:https://doi.org/10.1007/s00262-025-04107-y
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author Victoria Bloch Blytt Sandstad
Signe Modvig
Morten Orebo Holmström
author_facet Victoria Bloch Blytt Sandstad
Signe Modvig
Morten Orebo Holmström
author_sort Victoria Bloch Blytt Sandstad
collection DOAJ
description Abstract Acute lymphoblastic leukemia (ALL) is the most common pediatric malignancy, with standard treatment consisting of intensive chemotherapy and corticosteroids. While curative in most cases, this regimen leads to significant toxicity and long-term sequelae. Recent advancements in cancer immunotherapy, including chimeric antigen receptor T cells and bispecific T cell engagers, have improved outcomes, yet are limited by toxicity and immune escape by target downregulation. Thus, novel less toxic treatment modalities are highly warranted. The tumor mutational burden in ALL is low, which results in a low number of potentially immunogenic neo-antigens that could be used as targets for neo-epitope-specific therapeutic cancer vaccines. However, recent findings in solid cancer demonstrate that it is not the quantity but the quality of neo-antigens in the tumor that determine the tumor-specific immune response. Furthermore, novel sequencing techniques such as long-read sequencing and optical genome mapping can identify unknown genetic aberrations that may be targeted by neo-antigen vaccines. In ALL, both the ETV6-RUNX1 and BCR-ABL1 fusion genes, and the RAS-isoform mutations are frequent, and these genomic alterations generate immunogenic neo-epitopes. Additionally, therapeutic cancer vaccinations are well suited for ALL as the tumor burden is extremely low at time of a potential post-induction vaccination therapy, and patients are relatively young and are therefore less affected by immunosenescence. Thus, we envisage that neo-antigen specific therapeutic cancer vaccines could pose an important modality in future treatment algorithms for ALL.
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spelling doaj-art-e27f788d15084bbd8bf111d5fda0439a2025-08-20T03:45:56ZengSpringerCancer Immunology, Immunotherapy1432-08512025-06-0174811310.1007/s00262-025-04107-yNeo-antigen specific cancer vaccines for acute lymphoblastic leukemia—challenges, opportunities, and future directionsVictoria Bloch Blytt Sandstad0Signe Modvig1Morten Orebo Holmström2Department of Clinical Immunology, Copenhagen University Hospital RigshospitaletDepartment of Clinical Immunology, Copenhagen University Hospital RigshospitaletNational Center for Cancer Immune Therapy, Department of Oncology, Copenhagen University Hospital Herlev-GentofteAbstract Acute lymphoblastic leukemia (ALL) is the most common pediatric malignancy, with standard treatment consisting of intensive chemotherapy and corticosteroids. While curative in most cases, this regimen leads to significant toxicity and long-term sequelae. Recent advancements in cancer immunotherapy, including chimeric antigen receptor T cells and bispecific T cell engagers, have improved outcomes, yet are limited by toxicity and immune escape by target downregulation. Thus, novel less toxic treatment modalities are highly warranted. The tumor mutational burden in ALL is low, which results in a low number of potentially immunogenic neo-antigens that could be used as targets for neo-epitope-specific therapeutic cancer vaccines. However, recent findings in solid cancer demonstrate that it is not the quantity but the quality of neo-antigens in the tumor that determine the tumor-specific immune response. Furthermore, novel sequencing techniques such as long-read sequencing and optical genome mapping can identify unknown genetic aberrations that may be targeted by neo-antigen vaccines. In ALL, both the ETV6-RUNX1 and BCR-ABL1 fusion genes, and the RAS-isoform mutations are frequent, and these genomic alterations generate immunogenic neo-epitopes. Additionally, therapeutic cancer vaccinations are well suited for ALL as the tumor burden is extremely low at time of a potential post-induction vaccination therapy, and patients are relatively young and are therefore less affected by immunosenescence. Thus, we envisage that neo-antigen specific therapeutic cancer vaccines could pose an important modality in future treatment algorithms for ALL.https://doi.org/10.1007/s00262-025-04107-yAcute lymphoblastic leukemiaNeo-antigensCancer vaccinesPersonalized vaccinesImmune escape
spellingShingle Victoria Bloch Blytt Sandstad
Signe Modvig
Morten Orebo Holmström
Neo-antigen specific cancer vaccines for acute lymphoblastic leukemia—challenges, opportunities, and future directions
Cancer Immunology, Immunotherapy
Acute lymphoblastic leukemia
Neo-antigens
Cancer vaccines
Personalized vaccines
Immune escape
title Neo-antigen specific cancer vaccines for acute lymphoblastic leukemia—challenges, opportunities, and future directions
title_full Neo-antigen specific cancer vaccines for acute lymphoblastic leukemia—challenges, opportunities, and future directions
title_fullStr Neo-antigen specific cancer vaccines for acute lymphoblastic leukemia—challenges, opportunities, and future directions
title_full_unstemmed Neo-antigen specific cancer vaccines for acute lymphoblastic leukemia—challenges, opportunities, and future directions
title_short Neo-antigen specific cancer vaccines for acute lymphoblastic leukemia—challenges, opportunities, and future directions
title_sort neo antigen specific cancer vaccines for acute lymphoblastic leukemia challenges opportunities and future directions
topic Acute lymphoblastic leukemia
Neo-antigens
Cancer vaccines
Personalized vaccines
Immune escape
url https://doi.org/10.1007/s00262-025-04107-y
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