Conjunctival Melanoma Targeted Therapy: MAPK and PI3K/mTOR Pathways Inhibition

Invest Ophthalmol Vis Sci. 2019 Jun 3;60(7):2764-2772. doi: 10.1167/iovs.18-26508.

Abstract

Purpose: To analyze the activity of mitogen-activated protein kinase (MAPK) and phosphoinositide 3-kinases/mechanistic target of rapamycin (PI3K/mTOR) pathways in benign and malignant conjunctival melanocytic proliferations and explore whether specific inhibitors can suppress growth of conjunctival melanoma (CJM) cells.

Methods: The presence of a BRAF V600E mutation and activation of ERK, MEK, S6, and AKT were assessed with immunohistochemistry in 35 conjunctival nevi and 31 melanomas. Three CJM cell lines were used: CRMM1, carrying the BRAF V600E mutation; CRMM2, harboring the NRAS Q61L mutation; and T1527A, with a BRAF G466E mutation. WST-1 assays were performed with a BRAF inhibitor (vemurafenib), two MEK inhibitors (trametinib, selumetinib), a PI3K inhibitor (pictilisib), and a dual PI3K/mTOR inhibitor (dactolisib). The phosphorylation of ERK, MEK, and S6 were tested with western blots and apoptosis with cleaved caspase-3 immunostaining.

Results: A BRAF V600E mutation was detected in 42.6% of nevi and in 35.5% of CJM. MEK and ERK activation were higher in CJM, occurring in 62.9% and 45.7% of the nevi and 90.3% and 96.8% of the CJM, respectively. There was also a significant increase in S6 activation in CJM (90.3%) compared with the nevi (20%). CRMM1 was sensitive to trametinib and the PI3K inhibitors but only marginally to vemurafenib. CRMM2 was moderately sensitive to pictilisib, whereas T1527A was resistant to all drugs tested.

Conclusions: The MAPK pathway activity in CJM is increased, not only as a consequence of the BRAF V600E mutation. Targeted therapy may be useful for patients with CJM, especially those with activating BRAF mutations, whereas NRAS-mutated melanomas are relatively resistant.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Antineoplastic Agents / therapeutic use*
  • Benzimidazoles / therapeutic use
  • Blotting, Western
  • Conjunctival Neoplasms / drug therapy*
  • Conjunctival Neoplasms / enzymology
  • Conjunctival Neoplasms / pathology
  • Female
  • Fluorescent Antibody Technique, Indirect
  • Humans
  • Imidazoles / therapeutic use
  • Indazoles / therapeutic use
  • Male
  • Melanoma / drug therapy*
  • Melanoma / enzymology
  • Melanoma / pathology
  • Middle Aged
  • Mitogen-Activated Protein Kinases / antagonists & inhibitors*
  • Molecular Targeted Therapy*
  • Phosphatidylinositol 3-Kinases / drug effects*
  • Protein Kinase Inhibitors / therapeutic use*
  • Proto-Oncogene Proteins B-raf / genetics
  • Pyridones / therapeutic use
  • Pyrimidinones / therapeutic use
  • Quinolines / therapeutic use
  • Sulfonamides / therapeutic use
  • TOR Serine-Threonine Kinases / antagonists & inhibitors*
  • Tumor Cells, Cultured

Substances

  • 2-(1H-indazol-4-yl)-6-(4-methanesulfonylpiperazin-1-ylmethyl)-4-morpholin-4-ylthieno(3,2-d)pyrimidine
  • AZD 6244
  • Antineoplastic Agents
  • Benzimidazoles
  • Imidazoles
  • Indazoles
  • Protein Kinase Inhibitors
  • Pyridones
  • Pyrimidinones
  • Quinolines
  • Sulfonamides
  • trametinib
  • BRAF protein, human
  • Proto-Oncogene Proteins B-raf
  • TOR Serine-Threonine Kinases
  • Mitogen-Activated Protein Kinases
  • dactolisib