Artigo Acesso aberto Revisado por pares

TG101209, a small molecule JAK2-selective kinase inhibitor potently inhibits myeloproliferative disorder-associated JAK2V617F and MPLW515L/K mutations

2007; Springer Nature; Volume: 21; Issue: 8 Linguagem: Inglês

10.1038/sj.leu.2404750

ISSN

1476-5551

Autores

Animesh Pardanani, John Hood, Terra L. Lasho, Ross L. Levine, Michael Martin, Glenn Noronha, Christy Finke, Chi Ching Mak, Ruben A. Mesa, Huanzhang Zhu, Richard Söll, D. Gary Gilliland, Ayalew Tefferi,

Tópico(s)

Eosinophilic Disorders and Syndromes

Resumo

JAK2V617F and MPLW515L/K represent recently identified mutations in myeloproliferative disorders (MPD) that cause dysregulated JAK-STAT signaling, which is implicated in MPD pathogenesis. We developed TG101209, an orally bioavailable small molecule that potently inhibits JAK2 (IC50=6 nM), FLT3 (IC50=25 nM) and RET (IC50=17 nM) kinases, with significantly less activity against other tyrosine kinases including JAK3 (IC50=169 nM). TG101209 inhibited growth of Ba/F3 cells expressing JAK2V617F or MPLW515L mutations with an IC50 of ∼200 nM. In a human JAK2V617F-expressing acute myeloid leukemia cell line, TG101209-induced cell cycle arrest and apoptosis, and inhibited phosphorylation of JAK2V617F, STAT5 and STAT3. Therapeutic efficacy of TG101209 was demonstrated in a nude mouse model. Furthermore, TG101209 suppressed growth of hematopoietic colonies from primary progenitor cells harboring JAK2V617F or MPL515 mutations.

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