Preclinical human models and emerging therapeutics for advanced systemic mastocytosis

Arock, Michel and Wedeh, Ghaith and Hoermann, Gregor and Bibi, Siham and Akin, Cem and Peter, Barbara and Gleixner, Karoline V. and Hartmann, Karin and Butterfield, Joseph H. and Metcalfe, Dean D. and Valent, Peter. (2018) Preclinical human models and emerging therapeutics for advanced systemic mastocytosis. Haematologica, 103 (11). pp. 1760-1771.

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Official URL: https://edoc.unibas.ch/70873/

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Mastocytosis is a term used to denote a group of rare diseases characterized by an abnormal accumulation of neoplastic mast cells in various tissues and organs. In most patients with systemic mastocytosis, the neoplastic cells carry activating mutations in; KIT; Progress in mastocytosis research has long been hindered by the lack of suitable; in vitro; models, such as permanent human mast cell lines. In fact, only a few human mast cell lines are available to date: HMC-1, LAD1/2, LUVA, ROSA and MCPV-1. The HMC-1 and LAD1/2 cell lines were derived from patients with mast cell leukemia. By contrast, the more recently established LUVA, ROSA and MCPV-1 cell lines were derived from CD34; +; cells of non-mastocytosis donors. While some of these cell lines (LAD1/2, LUVA, ROSA; KIT WT; and MCPV-1) do not harbor; KIT; mutations, HMC-1 and ROSA; KIT D816V; cells exhibit activating; KIT; mutations found in mastocytosis and have thus been used to study disease pathogenesis. In addition, these cell lines are increasingly employed to validate new therapeutic targets and to screen for effects of new targeted drugs. Recently, the ROSA; KIT D816V; subclone has been successfully used to generate a unique; in vivo; model of advanced mastocytosis by injection into immunocompromised mice. Such a model may allow; in vivo; validation of data obtained; in vitro; with targeted drugs directed against mastocytosis. In this review, we discuss the major characteristics of all available human mast cell lines, with particular emphasis on the use of HMC-1 and ROSA; KIT D816V; cells in preclinical therapeutic research in mastocytosis.
Faculties and Departments:03 Faculty of Medicine > Departement Biomedizin > Department of Biomedicine, University Hospital Basel > Allergy and Immunity (Hartmann)
UniBasel Contributors:Hartmann, Karin
Item Type:Article, refereed
Article Subtype:Research Article
Note:Publication type according to Uni Basel Research Database: Journal article
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Last Modified:09 Apr 2020 15:08
Deposited On:09 Apr 2020 15:08

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