Soft tissue sarcomas are difficult to treat using chemotherapy owing to a current
deficiency in candidate drugs for specific targets. Screening candidate compounds and
analyzing therapeutic targets in sarcomas is insufficient, given the lack of an
appropriate human sarcoma animal model to accurately evaluate their efficacy, as well as
the lack of an adequate technical protocol for efficient transplantation and engraftment
of sarcoma specimens in patient-derived xenograft (PDX) models. Accordingly, in this
study, we sought to identify the optimal type of sarcoma and develop a protocol for
generating a PDX model. We characterized a PDX mouse model using histopathological and
immunohistochemical analyses to determine whether it would show pathological
characteristics similar to those of human sarcomas. We achieved engraftment of one of the
10 transplanted sarcoma specimens, the xenografted tumor of which exhibited massive
proliferation. Histologically, the engrafted sarcoma foci resembled a primary tumor of
pleomorphic leiomyosarcoma and maintained their histological structure in all passages.
Moreover, immunohistochemical analysis revealed the expression of specific markers of
differentiation to smooth muscle, which is consistent with the features of leiomyosarcoma.
We thus demonstrated that our pleomorphic leiomyosarcoma PDX mouse model mimics at least
one aspect of human sarcomas, and we believe that this model will facilitate the
development of novel therapies for sarcomas.