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Ref Type Journal Article
PMID (26873726)
Authors Waters AM, Stewart JE, Atigadda VR, Mroczek-Musulman E, Muccio DD, Grubbs CJ, Beierle EA
Title Preclinical Evaluation of UAB30 in Pediatric Renal and Hepatic Malignancies.
Journal Molecular cancer therapeutics
Vol 15
Issue 5
Date 2016 May
URL
Abstract Text 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.

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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
UAB30 UAB30 is a synthetic analogue of 9-cis-retinoic acid that activates the retinoid X receptor and may lead to a decrease in cell proliferation and tumor growth (PMID: 26873726, PMID: 31362265).
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
Unknown unknown Ewing sarcoma not applicable UAB30 Preclinical Actionable In a preclinical study, a renal Ewing sarcoma cell line was sensitive to UAB30 in culture, demonstrating cell-cycle arrest, decreased cell proliferation, and apoptosis (PMID: 26873726). 26873726
Unknown unknown hepatoblastoma not applicable UAB30 Preclinical - Cell line xenograft Actionable In a preclinical study, a human hepatoblastoma cell line was sensitive to UAB30 in both culture and in xenograft models, demonstrating cell-cycle arrest, decreased cell proliferation, and reduced tumor growth (PMID: 26873726). 26873726
Unknown unknown kidney rhabdoid cancer not applicable UAB30 Preclinical - Cell line xenograft Actionable In a preclinical study, a human malignant rhabdoid kidney tumor cell line was sensitive to UAB30 in both culture and in xenograft models, demonstrating cell-cycle arrest, decreased cell proliferation, apoptosis, and reduced tumor growth (PMID: 26873726). 26873726