Current landscape of translational and clinical research in myelodysplastic syndromes/neoplasms (MDS): Proceedings from the 1st International Workshop on MDS (iwMDS) of the International Consortium for MDS (icMDS) Review

  

Authors:	Bewersdorf, J. P.; Xie, Z.; Bejar, R.; Borate, U.; Boultwood, J.; Brunner, A. M.; Buckstein, R.; Carraway, H. E.; Churpek, J. E.; Daver, N. G.; Porta, M. G. D.; DeZern, A. E.; Fenaux, P.; Figueroa, M. E.; Gore, S. D.; Griffiths, E. A.; Halene, S.; Hasserjian, R. P.; Hourigan, C. S.; Kim, T. K.; Komrokji, R.; Kuchroo, V. K.; List, A. F.; Loghavi, S.; Majeti, R.; Odenike, O.; Patnaik, M. M.; Platzbecker, U.; Roboz, G. J.; Sallman, D. A.; Santini, V.; Sanz, G.; Sekeres, M. A.; Stahl, M.; Starczynowski, D. T.; Steensma, D. P.; Taylor, J.; Abdel-Wahab, O.; Xu, M. L.; Savona, M. R.; Wei, A. H.; Zeidan, A. M.
Review Title:	Current landscape of translational and clinical research in myelodysplastic syndromes/neoplasms (MDS): Proceedings from the 1st International Workshop on MDS (iwMDS) of the International Consortium for MDS (icMDS)
Abstract:	Biological events that contribute to the pathogenesis of myelodysplastic syndromes/neoplasms (MDS) are becoming increasingly characterized and are being translated into rationally designed therapeutic strategies. Herein, we provide updates from the first International Workshop on MDS (iwMDS) of the International Consortium for MDS (icMDS) detailing recent advances in understanding the genetic landscape of MDS, including germline predisposition, epigenetic and immune dysregulation, the complexities of clonal hematopoiesis progression to MDS, as well as novel animal models of the disease. Connected to this progress is the development of novel therapies targeting specific molecular alterations, the innate immune system, and immune checkpoint inhibitors. While some of these agents have entered clinical trials (e.g., splicing modulators, IRAK1/4 inhibitors, anti-CD47 and anti-TIM3 antibodies, and cellular therapies), none have been approved for MDS. Additional preclinical and clinical work is needed to develop a truly individualized approach to the care of MDS patients. © 2023 Elsevier Ltd
Keywords:	unclassified drug; genetics; pathogenesis; review; placebo; nonhuman; drug approval; drug targeting; neoplasm; neoplasms; animal; animals; novel therapies; disease model; immunology; myelodysplastic syndrome; protein processing; protein processing, post-translational; epigenetics; clinical research; innate immunity; biological therapy; disease predisposition; azacitidine; myelodysplastic syndromes; translational research; protein antibody; personalized medicine; animal models; mds; cd47 antigen; splicing defect; germline mutation; epigenomics; immune dysregulation; clonal hematopoiesis; immune checkpoint inhibitor; preclinical study; tamibarotene; telomere homeostasis; humans; human; cell- and tissue-based therapy; venetoclax; pevonedistat; magrolimab; sabatolimab; eprenetapopt; hepatitis a virus cellular receptor 2 antibody
Journal Title:	Blood Reviews
Volume:	60
ISSN:	0268-960X
Publisher:	Churchill Livingstone
Date Published:	2023-07-01
Start Page:	101072
Language:	English
DOI:	10.1016/j.blre.2023.101072
PUBMED:	36934059
PROVIDER:	scopus
DOI/URL:	https://www.scopus.com/inward/record.uri?eid=2-s2.0-85150256821&doi=10.1016%2fj.blre.2023.101072&partnerID=40&md5=75e4549c588433effb2fe3eebdee715d
Notes:	Review -- Source: Scopus

https://synapse.mskcc.org/synapse/works/has_related_data_chk/208222