Drug prioritization identifies panobinostat as a tailored treatment element for patients with metastatic hepatoblastoma.

ABSTRACT:

Background

Patients with metastatic hepatoblastoma are treated with severely toxic first-line chemotherapies in combination with surgery. Yet, inadequate response of lung metastases to neo-adjuvant chemotherapy still compromises patient outcomes making new treatment strategies, tailored to more efficient lung clearance, mandatory.

Methods

We harnessed a comprehensive patient-derived xenograft platform and a variety of in vitro and in vivo assays to establish the preclinical and biological rationale for a new drug for patients with metastatic hepatoblastoma.

Results

The testing of a library of established drugs on patient-derived xenografts identified histone deacetylase inhibitors, most notably panobinostat, to be highly efficacious on hepatoblastoma cells, as compare

SUBMITTER: Demir S 

PROVIDER: S-EPMC11556140 | biostudies-literature | 2024 Nov

REPOSITORIES: biostudies-literature