July 2019
Volume 60, Issue 9
Open Access
ARVO Annual Meeting Abstract  |   July 2019
Phase I/IIa Clinical Trial of Human Embryonic Stem Cell (hESC)-Derived Retinal Pigmented Epithelium (RPE, OpRegen) Transplantation in Advanced Dry Form Age-Related Macular Degeneration (AMD): Interim Results
Author Affiliations & Notes
  • Eyal Banin
    Department of Ophthalmology, Hadassah-Hebrew Univ Med Ctr, Jerusalem, Israel
  • Adiel Barak
    Department of Ophthalmology, Sourasky Medical Center, Tel Aviv, Israel
  • David S Boyer
    Retina Vitreous Associates Medical Group, Los Angeles, California, United States
  • Diana V Do
    Department of Ophthalmology, Byers Eye Institute, Stanford University School of Medicine, Palo Alto, California, United States
  • Rita Ehrlich
    Department of Ophthalmology, Rabin Medical Center, Petah Tikva, Israel
  • Tareq Jaouni
    Department of Ophthalmology, Hadassah-Hebrew Univ Med Ctr, Jerusalem, Israel
  • Richard McDonald
    West Coast Retina Group, San Francisco, California, United States
  • David G. Tealander
    Retinal Consultants Medical Group, Sacramento, California, United States
  • Maria Gurevich
    BioTime subsidiary, Cell Cure Neurosciences, Jerusalem, Israel
  • Ohad Cohen
    BioTime subsidiary, Cell Cure Neurosciences, Jerusalem, Israel
  • Ghesal Razag
    BioTime, Inc., Alameda, California, United States
  • Gary S Hogge
    BioTime, Inc., Alameda, California, United States
  • Benjamin Reubinoff
    Center for Embryonic Stem Cells and the Department of Gynecology and Obstetrics, Hadassah-Hebrew University Medical Center, Jerusalem, Israel
    BioTime subsidiary, Cell Cure Neurosciences, Jerusalem, Israel
  • Footnotes
    Commercial Relationships   Eyal Banin, Cell Cure Neurosciences (P), Cell Cure Neurosciences (C); Adiel Barak, Cell Cure Neurosciences (C), Cell Cure Neurosciences (F); David Boyer, BioTime, Inc. (C), BioTime, Inc. (F); Diana Do, BioTime, Inc. (C), BioTime, Inc. (F); Rita Ehrlich, Cell Cure Neurosciences (F); Tareq Jaouni, Cell Cure Neurosciences (F); Richard McDonald, BioTime, Inc. (F); David Tealander, BioTime, Inc. (F); Maria Gurevich, Cell Cure Neurosciences (E); Ohad Cohen, Cell Cure Neurosciences (E); Ghesal Razag, BioTime, Inc. (E); Gary Hogge, BioTime, Inc. (E); Benjamin Reubinoff, Cell Cure Neurosciences Ltd (P), Cell Cure Neurosciences Ltd (C)
  • Footnotes
    Support  CellCure Neurosciences, a subsidiary of BioTime
Investigative Ophthalmology & Visual Science July 2019, Vol.60, 6402. doi:https://doi.org/
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      Eyal Banin, Adiel Barak, David S Boyer, Diana V Do, Rita Ehrlich, Tareq Jaouni, Richard McDonald, David G. Tealander, Maria Gurevich, Ohad Cohen, Ghesal Razag, Gary S Hogge, Benjamin Reubinoff; Phase I/IIa Clinical Trial of Human Embryonic Stem Cell (hESC)-Derived Retinal Pigmented Epithelium (RPE, OpRegen) Transplantation in Advanced Dry Form Age-Related Macular Degeneration (AMD): Interim Results. Invest. Ophthalmol. Vis. Sci. 2019;60(9):6402. doi: https://doi.org/.

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

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Abstract

Purpose : Studies using autologous RPE cells in AMD patients suggest that introducing healthy RPE cells may be of therapeutic benefit. We developed technology to derive RPEs from hESCs using GMP directed differentiation. Safety and tolerability of this cell product is being evaluated in a Phase I/IIa clinical study in patients with advanced dry AMD and geographic atrophy (GA) (NCT02286089). We report accumulated safety and imaging data from all subjects in the fully-enrolled first 3 cohorts (n=12) and ongoing 4th cohort.

Methods : Transplantation is performed by subretinal injection of 50-200k OpRegen cells in suspension to the worse vision eye following conventional 23G vitrectomy under local anesthesia. Systemic immunosuppression is administered prior to transplantation until 3 months after implantation. Systemic and ocular safety is closely monitored. Retinal function and structure are monitored using various imaging modalities including BCVA, color fundus, SD-OCT, and FAF.

Results : Following completion of the first 3 cohorts, now under long term follow-up, dosing of cohort 4 is ongoing. Overall, treatment has been well tolerated and there have been no unexpected adverse events (AEs) or treatment-related systemic serious adverse events reported. The most common ocular AEs were the formation of predominately mild epiretinal membranes (ERM), though one severe ERM was successfully peeled 2 months following dosing. Additionally, one patient experienced a retinal detachment. Causality of the event was not able to be determined and the patient continues in the study following successful surgical repair. Within the area of the RPE cell transplant, signs of changes in drusen as well as improvements of the ellipsoid zone and RPE layers at the border of GA were seen in some subjects. Persistent changes observed following treatment, including subretinal pigmentation and hyper-reflective areas on OCT, are suggestive of the continued presence of transplanted RPE cells.

Conclusions : Subretinal transplantation of hESC-derived RPE cells in patients with advanced dry AMD and GA appears well tolerated to date. Imaging findings suggest presence of transplanted cells in the subretinal space. Potentially positive structural and clinical changes observed in some patients will require additional follow-up over time.

This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.

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