Recombinant human acid sphingomyelinase as an adjuvant to sorafenib treatment of experimental liver cancer.

ABSTRACT:

Background

Hepatocellular carcinoma (HCC) is the most common form of liver cancer and the third leading cause of cancer death worldwide. The only approved systemic treatment for unresectable HCC is the oral kinase inhibitor, sorafenib. Recombinant human acid sphingomyelinase (rhASM), which hydrolyzes sphingomyelin to ceramide, is an orphan drug under development for the treatment of Type B Niemann-Pick disease (NPD). Due to the hepatotropic nature of rhASM and its ability to generate pro-apoptotic ceramide, this study evaluated the use of rhASM as an adjuvant treatment with sorafenib in experimental models of HCC.

Methodology/principal findings

In vitro, rhASM/sorafenib treatment reduced the viability of Huh7 liver cancer cells more than sorafenib. In vivo, using a subcutane

SUBMITTER: Savic R 

PROVIDER: S-EPMC3665770 | biostudies-literature | 2013

REPOSITORIES: biostudies-literature