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Preclinical Evaluation of UAB30 in Pediatric Renal and Hepatic Malignancies
Alicia M Waters1, Jerry E Stewart1, Venkatram R Atigadda2
1Department of Surgery, University of Alabama, Birmingham, Birmingham, Alabama.
Abstract:
Rare tumors of solid organs remain some of the most difficult pediatric cancers to cure. These difficult tumors include rare pediatric renal malignancies, such as malignant rhabdoid kidney tumors (MRKT) and non-osseous renal Ewing sarcoma, and hepatoblastoma, a pediatric liver tumor that arises from immature liver cells. There are data in adult renal and hepatic malignancies demonstrating the efficacy of retinoid therapy. The investigation of retinoic acid therapy in cancer is not a new strategy, but the widespread adoption of this therapy has been hindered by toxicities. Our laboratory has been investigating a novel synthetic rexinoid, UAB30, which exhibits a more favorable side-effect profile. In this study, we hypothesized that UAB30 would diminish the growth of tumor cells from both rare renal and liver tumors in vitro and in vivo We successfully demonstrated decreased cellular proliferation, invasion and migration, cell-cycle arrest, and increased apoptosis after treatment with UAB30. Additionally, in in vivo murine models of human hepatoblastoma or rare human renal tumors, there were significantly decreased tumor xenograft growth and increased animal survival after UAB30 treatment. UAB30 should be further investigated as a developing therapeutic in these rare and difficult-to-treat pediatric solid organ tumors. Mol Cancer Ther; 15(5); 911-21. ©2016 AACR.
Insights
A novel synthetic rexinoid, UAB30, effectively reduced the growth of rare pediatric kidney and liver tumors in laboratory and animal studies. This agent shows promise for treating difficult pediatric solid organ cancers with fewer side effects.
Area of Science:
- Pediatric Oncology
- Molecular Therapeutics
- Cancer Biology
Background:
- Rare pediatric solid organ tumors, including malignant rhabdoid kidney tumors (MRKT), renal Ewing sarcoma, and hepatoblastoma, are challenging to treat.
- Retinoid therapy has shown efficacy in adult renal and hepatic malignancies, but toxicities limit its use.
- A novel synthetic rexinoid, UAB30, has been developed with a potentially improved side-effect profile.
Purpose of the Study:
- To investigate the efficacy of the novel synthetic rexinoid UAB30 against rare pediatric renal and liver tumors.
- To evaluate UAB30's effects on tumor cell proliferation, invasion, migration, cell-cycle progression, and apoptosis.
- To assess UAB30's therapeutic potential in vivo using murine models of human pediatric kidney and liver tumors.
Main Methods:
- In vitro studies on pediatric renal and liver tumor cell lines treated with UAB30.
- Assessment of cellular proliferation, invasion, migration, cell-cycle arrest, and apoptosis.
- In vivo studies using xenograft models of human hepatoblastoma and rare renal tumors in mice.
- Evaluation of tumor xenograft growth and animal survival rates post-UAB30 treatment.
Main Results:
- UAB30 treatment significantly decreased proliferation, invasion, and migration of tumor cells.
- UAB30 induced cell-cycle arrest and increased apoptosis in vitro.
- In vivo studies showed significantly reduced tumor xenograft growth and increased survival in UAB30-treated mice.
- UAB30 demonstrated a favorable side-effect profile compared to traditional retinoids.
Conclusions:
- The novel synthetic rexinoid UAB30 demonstrates significant anti-tumor activity against rare pediatric renal and liver tumors.
- UAB30 effectively inhibits tumor cell growth and induces cell death through multiple mechanisms.
- UAB30 warrants further investigation as a promising therapeutic agent for rare and difficult-to-treat pediatric solid organ cancers.
