FCCC LOGO Faculty Publications
Medium throughput biochemical compound screening identifies novel agents for pharmacotherapy of neurofibromatosis type I
Biochimie. 2017 Jan 05;135 :1-5
PMID: 28065690    PMCID: PMC5405558    URL: https://www.ncbi.nlm.nih.gov/pubmed/28065690
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The variable manifestation of phenotypes that occur in patients with neurofibromatosis type 1 (NF1) includes benign and malignant neurocutaneous tumors for which no adequate treatment exists. Cell-based screening of known bioactive compounds library identified the protein phosphatase 2A (PP2A) inhibitor Cantharidin and the L-type calcium channel blocker Nifedipine as potential candidates for NF1 pharmacotherapy. Validation of screening results using human NF1-associated malignant peripheral nerve sheath tumor (MPNST) cells showed that Cantharidin effectively impeded MPNST cell growth, while Nifedipine treatment significantly decreased local tumor growth in an MPNST xenograft animal model. These data suggest that inhibitors of PP2A, as well as calcium channel blockers, might be used in broader MPNST preclinical studies as single agents or in combinatorial therapeutic strategies.
1638-6183 Semenova, Galina Stepanova, Dina Deyev, Sergey M Chernoff, Jonathan Journal Article France Biochimie. 2017 Jan 5. pii: S0300-9084(17)30001-9. doi: 10.1016/j.biochi.2017.01.001.