Medium throughput biochemical compound screening identifies novel agents for pharmacotherapy of neurofibromatosis type 1

Galina Semenova, Dina S. Stepanova, Sergey M. Deyev, Jonathan Chernoff

Research output: Contribution to journalArticle

4 Citations (Scopus)


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.

Original languageEnglish
Pages (from-to)1-5
Number of pages5
Publication statusPublished - 1 Apr 2017
Externally publishedYes



  • Cantharidin
  • Chemical screening
  • Neurofibromatosis type 1
  • Nifedipine

ASJC Scopus subject areas

  • Biochemistry

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