A variety of cellular therapies including hematopoietic cell transplantation (HCT) hold the promise to treat medical conditions and diseases that currently have limited or no effective therapeutic options. A number of cellular therapies other than HCT, such as CAR T-cell therapy, are currently in preclinical and clinical development and the field is rapidly growing. The current activity of cellular therapies, including HCT, in the clinical setting are summarized in this article. Collaborative efforts from all relevant professionals and organizations will be of great importance to overcome substantial challenges in clinical development and post-launch evidence collection of cellular therapies. Harmonization among decision-makers also plays a critical role in reinforcing consistency and improving efficiencies of the regulatory and health technology assessment process. For the long-term safety follow-up of patients undergoing cellular therapies, registries for HCT are able to manage the complexity of data and in the best position to introduce and monitor future innovative cellular therapies for a variety of hematological disorders.
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http://dx.doi.org/10.31547/bct-2022-013 | DOI Listing |
Blood Adv
January 2025
Mayo Clinic, Rochester, Minnesota, United States.
In this study, we first analyzed data from 147 patients with solitary plasmacytomas treated at the Mayo Clinic between 2005 and 2022 and then expanded our investigation through a systematic review and meta-analysis of 62 studies, encompassing 3,487 patients from the years 1960 to 2022. Our findings reveal that patients with up to 10% clonal plasma cells in their bone marrow (BM), denoted as plasmacytoma +, had a significantly reduced median disease-free survival (DFS) of 15.7 months vs.
View Article and Find Full Text PDFAm Soc Clin Oncol Educ Book
January 2025
Division of Oncology, Department of Medicine, University of Washington, Seattle, WA.
The growing sophistication of tumor molecular profiling has helped to slowly transition oncologic care toward a more personalized approach in different tumor types, including in bladder cancer. The National Comprehensive Cancer Network recommends that all patients with stage IVA and stage IVB urothelial carcinoma have molecular analysis that integrates at least testing to help facilitate the selection of future therapeutic options. Sequencing of tumor-derived tissue is the mainstay to obtain this genomic testing, but as in other cancers, there has been extensive research into the integration of liquid biopsies in longitudinal management.
View Article and Find Full Text PDFJ Clin Oncol
January 2025
Center for Cell Engineering, Sloan Kettering Institute, New York, NY.
Purpose: We designed a CD19-targeted chimeric antigen receptor (CAR) comprising a calibrated signaling module, termed 1XX, that differs from that of conventional CD28/CD3ζ and 4-1BB/CD3ζ CARs. Preclinical data demonstrated that 1XX CARs generated potent effector function without undermining T-cell persistence. We hypothesized that 1XX CAR T cells may be effective at low doses and elicit minimal toxicities.
View Article and Find Full Text PDFProc Natl Acad Sci U S A
February 2025
State Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Sun Yat-sen University Cancer Center, Guangzhou 510050, China.
Chromobox 2 (CBX2), a crucial component of the polycomb repressive complex (PRC), has been implicated in the development of various human cancers. However, its role in the regulation of tumor immunogenicity and immune evasion remains inadequately understood. In this study, we found that ablation of CBX2 led to tumor growth inhibition, activation of the tumor immune microenvironment, and enhanced therapeutic efficacy of anti-PD1 or adoptive T cell therapies by using murine syngeneic tumor models.
View Article and Find Full Text PDFProc Natl Acad Sci U S A
February 2025
State Key Laboratory of Genetic Engineering, School of Life Sciences, Liver Cancer Institute of Zhongshan Hospital, Fudan University, Shanghai 200438, China.
Aging is a complex process that affects multiple organs, and the discovery of a pharmacological approach to ameliorate aging is considered the Holy Grail of medicine. Here, we performed an N-ethyl-N-nitrosourea forward genetic screening in zebrafish and identified an accelerated aging mutant named (), harboring a mutation in the - () gene. Loss of leads to a short lifespan and age-related characteristics in the intestine of zebrafish embryos, such as cellular senescence, genomic instability, and epigenetic alteration.
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