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Disease models

Xenograft mouse model for pediatric acute megakaryoblastic leukemia (AMKL)

A mouse xenograft model for pediatric AMKL could help identify new treatments for the cancer. Blast cells from the blood or bone marrow of patients with pediatric AMKL were injected into immunocompromised mice, with six of eight samples leading to successful engraftment. The models recapitulated aspects of the human disease, including low blast counts in the blood. In the model, the aurora kinase A (AURKA; aurora-A) inhibitor alisertib decreased leukemic blast levels and the incidence of paralysis and increased survival compared with placebo. Next steps include using this model to assess the efficacy of additional therapeutic candidates.
Takeda Pharmaceutical Co. Ltd.'s alisertib (MLN8237) is in Phase III testing to treat peripheral T cell lymphoma (PTCL). The compound also is in Phase II testing or earlier to treat other cancers.

SciBX 5(44); doi:10.1038/scibx.2012.1173
Published online Nov. 8, 2012

Patent and licensing status undisclosed

Thiollier, C. et al. J. Exp. Med.;
published online Oct. 8, 2012;
Contact: Thomas Mercher, Institut National de la Santé et de la Recherche Médicale (INSERM), Vellejuif, France