Long-term follow-up of patients with retinitis pigmentosa receiving intraocular ciliary neurotrophic factor implants
Open accessAbstract
Purpose To evaluate the long-term efficacy of ciliary neurotrophic factor delivered via an intraocular encapsulated cell implant for the treatment of retinitis pigmentosa.Design Long-term follow-up of a multicenter, sham-controlled study.Methods Thirty-six patients at 3 CNTF4 sites were randomly assigned to receive a high- or low-dose implant in 1 eye and sham surgery in the fellow eye. The primary endpoint (change in visual field sensitivity at 12 months) had been reported previously. Here we measure long-term visual acuity, visual field, and optical coherence tomography (OCT) outcomes in 24 patients either retaining or explanting the device at 24 months relative to sham-treated eyes.Results Eyes retaining the implant showed significantly greater visual field loss from baseline than either explanted eyes or sham eyes through 42 months. By 60 months and continuing through 96 months, visual field loss was comparable among sham-treated eyes, eyes retaining the implant, and explanted eyes, as was visual acuity and OCT macular volume.Conclusions Over the short term, ciliary neurotrophic factor released continuously from an intravitreal implant led to loss of total visual field sensitivity that was greater than the natural progression in the sham-treated eye. This additional loss of sensitivity related to the active implant was reversible when the implant was removed. Over the long term (60-96 months), there was no evidence of efficacy for visual acuity, visual field sensitivity, or OCT measures of retinal structure.
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Neuroprotective factors such as GDNF, CNTF, BDNF, basic fibroblast growth factor, and PEDF have shown promise in mouse models of retinal degeneration.
“Neuroprotective factors that support the survival of retinal cells, such as glial cell-derived neurotrophic factor (GDNF), ciliary neurotrophic factor (CNTF), brain-derived neurotrophic factor (BDNF), basic fibroblast growth factor, and pigment epithelium-derived factor (PEDF) have shown promise in mouse models of retinal degeneration.67,103–105”
From Gene Therapy for Retinal Degenerative Diseases: Progress, Challenges, and Future Directions (2023) · cites this paper as [103]
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- Authors
- Birch, D. G., Bennett, L. D., Duncan, J. L., Weleber, R. G., & Pennesi, M. E.
- Affiliations
- Retina Foundation of the Southwest (US)
- University of California, San Francisco (US)
- Oregon Health & Science University (US)
- Corresponding author
- Birch, David G.
- Venue
- American Journal of Ophthalmology
- Publisher
- Elsevier BV
- Published
- 2016-10
- Volume
- 170
- Pages
- 10-14
- ISSN
- 0002-9394 (ISSN-L)
- PMC
- PMC5056139
- OpenAlex
- W2501579558
- Cited by
- 112
- References
- 22
- Citations by year
- 2026: 8 · 2025: 9 · 2024: 4 · 2023: 10 · 2022: 12 · 2021: 15 · 2020: 15 · 2019: 11 · 2018: 19 · 2017: 8 · 2016: 1
- FWCI
- 6.75
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- American Journal of Ophthalmology (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- eScholarship (California Digital Library) (submittedVersion)
- Funders
- National Institutes of Health (5k08ey021186-03)
- U.S. Food and Drug Administration (R01 FD004100)
- National Eye Institute (R01EY009076, K08-EY021186)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Retinal Development and Disorders
- Health Sciences › Medicine › Ophthalmology › Retinal Diseases and Treatments
- Health Sciences › Medicine › Ophthalmology › Glaucoma and retinal disorders
- Keywords
- retinitis pigmentosa, ciliary neurotrophic factor, visual field sensitivity, visual acuity, macular volume, implant removal, long-term efficacy, neurotrophic therapy
- MeSH terms
- Adult, Aged, Electroretinography, Female, Follow-Up Studies, Humans, Male, Retina, Retinitis Pigmentosa, Visual Acuity, Visual Fields, Drug Delivery Systems, Ciliary Neurotrophic Factor, Tomography, Optical Coherence, Visual Field Tests
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