June 2021
Volume 62, Issue 8
Open Access
ARVO Annual Meeting Abstract  |   June 2021
Identification of Drug Candidates using a Patient-Derived Model of Proliferative Vitreoretinopathy
Author Affiliations & Notes
  • Dhanesh Amarnani
    Schepens Eye Research Institute of Massachusetts Eye and Ear, Boston, Massachusetts, United States
  • Santiago Delgado-Tirado
    Schepens Eye Research Institute of Massachusetts Eye and Ear, Boston, Massachusetts, United States
  • Lucia Gonzalez-Buendia
    Schepens Eye Research Institute of Massachusetts Eye and Ear, Boston, Massachusetts, United States
  • Michael O'Hare
    Schepens Eye Research Institute of Massachusetts Eye and Ear, Boston, Massachusetts, United States
  • Whitney A Greene
    Sensory Trauma Task Area, United States Army Institute of Surgical Research, Texas, United States
  • Heuy-Ching Hetty Wang
    Sensory Trauma Task Area, United States Army Institute of Surgical Research, Texas, United States
  • Joseph Arboleda-Velasquez
    Schepens Eye Research Institute of Massachusetts Eye and Ear, Boston, Massachusetts, United States
  • Leo A Kim
    Schepens Eye Research Institute of Massachusetts Eye and Ear, Boston, Massachusetts, United States
  • Footnotes
    Commercial Relationships   Dhanesh Amarnani, None; Santiago Delgado-Tirado, None; Lucia Gonzalez-Buendia, None; Michael O'Hare, None; Whitney Greene, None; Heuy-Ching Wang, None; Joseph Arboleda-Velasquez, None; Leo Kim, None
  • Footnotes
    Support  The Office of the Assistant Secretary of Defense for Health Affairs, through the Vision Research Program Technology/Therapeutic Development Award under Award No. W81XWH-17-2-0006.
Investigative Ophthalmology & Visual Science June 2021, Vol.62, 3643. doi:
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      Dhanesh Amarnani, Santiago Delgado-Tirado, Lucia Gonzalez-Buendia, Michael O'Hare, Whitney A Greene, Heuy-Ching Hetty Wang, Joseph Arboleda-Velasquez, Leo A Kim; Identification of Drug Candidates using a Patient-Derived Model of Proliferative Vitreoretinopathy. Invest. Ophthalmol. Vis. Sci. 2021;62(8):3643.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : Proliferative vitreoretinopathy (PVR) is a sight-threatening complication of retinal detachment and its surgical repair with no effective treatment. The aim of this study is to identify promising drug combinations with a potential synergistic effect by carrying out a screen in our primary culture model and an explant model of PVR.

Methods : Twelve candidate drugs were tested at 2 concentrations at 72 hours for their effects on proliferation and cytotoxicity in C-PVR cells. The most significant hits were tested in combination with other compounds to study combinatorial effects on proliferation. This effect was further evaluated in an ex vivo explant model, and total branch lengths were measured.

Results : At 5 μM concentration 6 out of 12 drugs, bendamustine, semagacestat, daclatsvir, marbofloxacin, stavudine, and resveratrol significantly inhibited cell proliferation (20%, 30%, 16%, 30%, 19%, and 28% inhibition respectively). At 1 μM concentration, 2 out of 12 drugs, bendamustine (25%) and semagacestat (28%) significantly inhibited proliferation. 30 μM melphalan induced 74% inhibition, and a 60 μM concentration reduced cell proliferation by 83%. Topotecan at 40 nM induced a 54% decrease in proliferation, and a 53% decrease was noted with 80 nM. Daunorubicin, 15 nM treatment resulted in a 50% reduction, while the 30 nM treatment resulted in a 53% reduction in proliferation. Likewise, with lenalidomide, 24% inhibition of proliferation was observed. The combination of melphalan or topotecan with other drugs showed a dramatic effect on the proliferation of C-PVR cells. Melphalan showed synergistic effect with all 10 drugs. In comparison, with topotecan, 6 out of 10 showed a synergistic effect in combination. No significant cell death was observed. Melphalan at 30 μM (27.5 mm total branch length) and topotecan at 40 nM (34 mm total branch length) alone reduced the distance covered by outgrowths compared to vehicle (167 mm total branch length) in the explants. Explants treated with combination therapy showed an almost complete inhibition of any growth suggesting synergistic effect.

Conclusions : The majority of our candidate drugs showed dose dependent effects on C-PVR cell proliferation. Melphalan and topotecan in combination with other drugs successfully inhibited proliferation in vitro, and in an explant model of PVR suggesting synergistic effects of these agents when used as combination therapy.

This is a 2021 ARVO Annual Meeting abstract.

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