Tyrosine Kinase Inhibitor Imatinib Mesylate as Anticancer Agent for Advanced Ocular Melanoma Expressing Immuno-Istochemical C-KIT (CD 117): Preliminary Results of a Compassionate Use Clinical Trial

Giammaria Fiorentini, S. Rossi, G. Lanzanova, M. Biancalani, A. Palomba, P. Bernardeschi, P. Dentico, U. De Giorgi

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Imatinib mesylate (IM), is a selective and competitive inhibitor of tyrosine kinases, including BCR-ABL, ABL, KIT, and the platelet-derived growth factor receptors (PDGF-R). It binds to the ATP-binding site of the target kinase and prevents the transfer of phosphate from ATP to the tyrosine residues of various substrates. At oral doses of 200-600 mg, the majority of patients with chronic myeloid leukaemia, Philadelphia chromosome-positive acute lymphoblastic leukemia expressing the BCR-ABL fusion protein and gastrointestinal stromal tumours (GIST) achieve a bio-molecular and clinical response, frequently complete, associated with limited toxicity. Several other human cancers, as small-cell lung carcinoma, melanoma, seminoma, some sarcomas, and adenoid cystic carcinomas may over-express KIT or PDGF-R, and clinical trials to evaluate the role of IM in the treatment of such cancers are currently ongoing. We determined c-KIT with Dako CD 117 antibody in 5 cases of advanced ocular melanoma (OM) and we found positive immuno-reactivity for CD 117 in three patients. We treated all patients with palliative-use IM at the oral dose of 400 mgr daily. We obtained in expressing positive immuno-reactivity for CD 117 patients: a reduction of malignant ascites in one, a partial remission in the neck nodes in another, and progression of liver metastases in the third. Evidences of progression has been reported in the other two patients expressing negative immuno-reactivity for CD 117. We conclude that the effect of IM should be assessed only in OM with positive immuno-histochemical c-kit (CD 117) expression. IM might be a potential therapeutic strategy for these patients.

Original languageEnglish
Pages (from-to)17-20
Number of pages4
JournalJournal of Experimental and Clinical Cancer Research
Issue number4 SUPPL.
Publication statusPublished - Dec 2003



  • Imatinib mesylate
  • Immuno-istochemical C-KIT (CD 117)
  • Ocular melanoma
  • Platelet-derived growth factor receptors
  • Tyrosine kinases

ASJC Scopus subject areas

  • Cancer Research
  • Oncology

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