{"title":"CAR-T cell therapy for treatment of acute myeloid leukemia, advances and outcomes.","authors":"Malak Khalifeh, Emily Hopewell, Huda Salman","doi":"10.1016/j.ymthe.2025.03.052","DOIUrl":null,"url":null,"abstract":"<p><p>Despite recent U.S. Food and Drug Administration (FDA) approval of multiple therapies for patients with acute myeloid leukemia (AML), clinical outcomes for those patients continue to remain poor. There are very few effective immunotherapeutic modalities such as allogeneic stem cell transplant for AML, and this is, in part, due to a lack of known antigens that are unique to AML and not present on vital normal hematopoietic precursors. Additionally, AML is supported by a hostile marrow tumor microenvironment that has a notable role in dampening T cell effector function. Myeloid-derived suppressor cells and regulatory T cells play a pivotal role in AML microenvironment immune hostility toward endogenous T cells as well as adoptively transferred T cells. There are many clinical trials that are designed to test the feasibility and efficacy of adoptively transferred T cells, including chimeric antigen receptor T cell therapies in AML, yet none is FDA approved for this fatal disease. In this review, we dissect these trials, their contribution to this therapeutic direction, and their success.</p>","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":" ","pages":"2441-2453"},"PeriodicalIF":12.1000,"publicationDate":"2025-06-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Molecular Therapy","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1016/j.ymthe.2025.03.052","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/4/2 0:00:00","PubModel":"Epub","JCR":"Q1","JCRName":"BIOTECHNOLOGY & APPLIED MICROBIOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Despite recent U.S. Food and Drug Administration (FDA) approval of multiple therapies for patients with acute myeloid leukemia (AML), clinical outcomes for those patients continue to remain poor. There are very few effective immunotherapeutic modalities such as allogeneic stem cell transplant for AML, and this is, in part, due to a lack of known antigens that are unique to AML and not present on vital normal hematopoietic precursors. Additionally, AML is supported by a hostile marrow tumor microenvironment that has a notable role in dampening T cell effector function. Myeloid-derived suppressor cells and regulatory T cells play a pivotal role in AML microenvironment immune hostility toward endogenous T cells as well as adoptively transferred T cells. There are many clinical trials that are designed to test the feasibility and efficacy of adoptively transferred T cells, including chimeric antigen receptor T cell therapies in AML, yet none is FDA approved for this fatal disease. In this review, we dissect these trials, their contribution to this therapeutic direction, and their success.
期刊介绍:
Molecular Therapy is the leading journal for research in gene transfer, vector development, stem cell manipulation, and therapeutic interventions. It covers a broad spectrum of topics including genetic and acquired disease correction, vaccine development, pre-clinical validation, safety/efficacy studies, and clinical trials. With a focus on advancing genetics, medicine, and biotechnology, Molecular Therapy publishes peer-reviewed research, reviews, and commentaries to showcase the latest advancements in the field. With an impressive impact factor of 12.4 in 2022, it continues to attract top-tier contributions.