Preclinical models of Waldenström's macroglobulinemia and drug resistance

Research output: Contribution to journalReview article

2 Citations (Scopus)

Abstract

Newer therapeutic strategies are emerging in Waldenström's Macroglobulinemia (WM), which has traditionally been an orphan disease diagnosis. Ibrutinib, a Bruton's tyrosine kinase (BTK) inhibitor was FDA-approved in 2015 as the first ever drug for the treatment of WM. This being a targeted therapy, has given rise to increased research into novel agents and pathways that can be exploited for clinical benefit in WM. In order to understand the underlying mechanisms of disease behavior as well as to test the benefit of various drugs, appropriate preclinical models are required. Historically there had been a lack of representative preclinical models in WM, but in recent years this has dramatically changed. This review highlights the currently available preclinical models and data regarding drug resistance pathways in WM. Knowledge from these will certainly help in paving the future course of treatment in this rare disorder which is indolent and yet, so far incurable.

Original languageEnglish (US)
Pages (from-to)169-178
Number of pages10
JournalBest Practice and Research: Clinical Haematology
Volume29
Issue number2
DOIs
StatePublished - Jun 1 2016

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Waldenstrom Macroglobulinemia
Drug Resistance
Pharmaceutical Preparations
Rare Diseases
Therapeutics
Research

Keywords

  • Cell line
  • Preclinical
  • Resistance
  • Waldenström's macroglobulinemia

ASJC Scopus subject areas

  • Medicine(all)
  • Oncology
  • Clinical Biochemistry

Cite this

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title = "Preclinical models of Waldenstr{\"o}m's macroglobulinemia and drug resistance",
abstract = "Newer therapeutic strategies are emerging in Waldenstr{\"o}m's Macroglobulinemia (WM), which has traditionally been an orphan disease diagnosis. Ibrutinib, a Bruton's tyrosine kinase (BTK) inhibitor was FDA-approved in 2015 as the first ever drug for the treatment of WM. This being a targeted therapy, has given rise to increased research into novel agents and pathways that can be exploited for clinical benefit in WM. In order to understand the underlying mechanisms of disease behavior as well as to test the benefit of various drugs, appropriate preclinical models are required. Historically there had been a lack of representative preclinical models in WM, but in recent years this has dramatically changed. This review highlights the currently available preclinical models and data regarding drug resistance pathways in WM. Knowledge from these will certainly help in paving the future course of treatment in this rare disorder which is indolent and yet, so far incurable.",
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N2 - Newer therapeutic strategies are emerging in Waldenström's Macroglobulinemia (WM), which has traditionally been an orphan disease diagnosis. Ibrutinib, a Bruton's tyrosine kinase (BTK) inhibitor was FDA-approved in 2015 as the first ever drug for the treatment of WM. This being a targeted therapy, has given rise to increased research into novel agents and pathways that can be exploited for clinical benefit in WM. In order to understand the underlying mechanisms of disease behavior as well as to test the benefit of various drugs, appropriate preclinical models are required. Historically there had been a lack of representative preclinical models in WM, but in recent years this has dramatically changed. This review highlights the currently available preclinical models and data regarding drug resistance pathways in WM. Knowledge from these will certainly help in paving the future course of treatment in this rare disorder which is indolent and yet, so far incurable.

AB - Newer therapeutic strategies are emerging in Waldenström's Macroglobulinemia (WM), which has traditionally been an orphan disease diagnosis. Ibrutinib, a Bruton's tyrosine kinase (BTK) inhibitor was FDA-approved in 2015 as the first ever drug for the treatment of WM. This being a targeted therapy, has given rise to increased research into novel agents and pathways that can be exploited for clinical benefit in WM. In order to understand the underlying mechanisms of disease behavior as well as to test the benefit of various drugs, appropriate preclinical models are required. Historically there had been a lack of representative preclinical models in WM, but in recent years this has dramatically changed. This review highlights the currently available preclinical models and data regarding drug resistance pathways in WM. Knowledge from these will certainly help in paving the future course of treatment in this rare disorder which is indolent and yet, so far incurable.

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