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• W2924915481 abstract "Retinal diseases are among the most challenging to develop therapies for in ophthalmology. For diseases that cause progressive death of retinal neurons, treatments to interrupt the pathogenic mechanism by correcting the genetic defect may provide the greatest potential to stop disease progression, or in some cases, improve vision.1Russell S. Bennett J. Wellman J.A. et al.Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.Lancet. 2017; 390: 849-860Abstract Full Text Full Text PDF PubMed Scopus (898) Google Scholar However, for many patients with retinal diseases, including idiopathic macular telangiectasia type 2, a single genetic defect has not been identified that can explain vision loss, and there are limited or no animal models in which to study the disease mechanism or potential treatment efficacy. Studies indicate the primary site of degeneration in idiopathic macular telangiectasia type 2 is Müller cells,2Powner M.B. Gillies M.C. Tretiach M. et al.Perifoveal Muller cell depletion in a case of macular telangiectasia type 2.Ophthalmology. 2010; 117: 2407-2416Abstract Full Text Full Text PDF PubMed Scopus (180) Google Scholar and research into the genetic cause of this inherited degenerative disease is ongoing. In diseases like idiopathic macular telangiectasia type 2, nonspecific neuroprotective therapies may hold potential for slowing the rate of vision loss. Prior studies of ciliary neurotrophic factor (CNTF) showed efficacy in slowing photoreceptor degeneration in 13 different preclinical models of photoreceptor degeneration, and the mechanism of photoreceptor protection requires activation of the gp130 receptor in Müller cells,3Rhee K.D. Nusinowitz S. Chao K. et al.CNTF-mediated protection of photoreceptors requires initial activation of the cytokine receptor gp130 in Muller glial cells.Proc Natl Acad Sci U S A. 2013; 110: E4520-E4529Crossref PubMed Scopus (63) Google Scholar suggesting it could be a nonspecific neuroprotective treatment for diseases associated with Müller cell or photoreceptor loss. Randomized, multicenter trials of CNTF in eyes with geographic atrophy resulting from age-related macular degeneration demonstrated no improvement in visual acuity over 12 to 24 months,4Zhang K. Hopkins J.J. Heier J.S. et al.Ciliary neurotrophic factor delivered by encapsulated cell intraocular implants for treatment of geographic atrophy in age-related macular degeneration.Proc Natl Acad Sci U S A. 2011; 108: 6241-6245Crossref PubMed Scopus (242) Google Scholar and studies of retinitis pigmentosa showed reduced peripheral visual field in CNTF-treated eyes that was reversible on removal of the treatment.5Birch D.G. Bennett L.D. Duncan J.L. et al.Long-term follow-up of patients with retinitis pigmentosa receiving intraocular ciliary neurotrophic factor implants.Am J Ophthalmol. 2016; 170: 10-14Abstract Full Text Full Text PDF PubMed Scopus (65) Google Scholar However, CNTF-treated eyes showed increased retinal thickness on OCT scans,5Birch D.G. Bennett L.D. Duncan J.L. et al.Long-term follow-up of patients with retinitis pigmentosa receiving intraocular ciliary neurotrophic factor implants.Am J Ophthalmol. 2016; 170: 10-14Abstract Full Text Full Text PDF PubMed Scopus (65) Google Scholar suggesting that standard measures of visual acuity and visual field sensitivity do not always correspond to changes in retinal structure and may not be sensitive measures of disease progression or response to therapy. The Macular Telangiectasia Type 2-Phase 2 CNTF study (page 540) used an objective, sensitive measure of photoreceptor structure, disruption of the ellipsoid zone band on OCT images, and correlated OCT images of retinal structure with measures of retinal sensitivity in the macula at comparable locations. The investigators demonstrated significant correlations between retinal sensitivity and photoreceptor loss as demonstrated by ellipsoid zone disruption. Unlike studies in primary photoreceptor degeneration such as retinitis pigmentosa, the Macular Telangiectasia Type 2-Phase 2 CNTF study reported 45% less retinal sensitivity loss and 31% less progression of photoreceptor loss on OCT scans in CNTF-treated eyes compared with eyes randomized to receive sham treatment.6Chew E.Y. Clemons T.E. Jaffe G.J. et al.Effect of ciliary neurotropic factor on retinal neurodegeneration in patients with macular telangiectasia type 2: a randomized clinical trial.Ophthalmology. 2019; 126: 540-549Abstract Full Text Full Text PDF PubMed Scopus (77) Google Scholar The study reported a strong correlation between retinal sensitivity, measured using microperimetry, and area of photoreceptor loss (r = 0.86; P < 0.001).6Chew E.Y. Clemons T.E. Jaffe G.J. et al.Effect of ciliary neurotropic factor on retinal neurodegeneration in patients with macular telangiectasia type 2: a randomized clinical trial.Ophthalmology. 2019; 126: 540-549Abstract Full Text Full Text PDF PubMed Scopus (77) Google Scholar Furthermore, reading speed declined significantly in the sham-treated eyes by almost 14 words per minute on average, whereas CNTF-treated eyes showed no loss of reading speed.6Chew E.Y. Clemons T.E. Jaffe G.J. et al.Effect of ciliary neurotropic factor on retinal neurodegeneration in patients with macular telangiectasia type 2: a randomized clinical trial.Ophthalmology. 2019; 126: 540-549Abstract Full Text Full Text PDF PubMed Scopus (77) Google Scholar Ciliary neurotrophic factor was well tolerated, with similar rates of adverse ocular events occurring in both sham- and CNTF-treated eyes.6Chew E.Y. Clemons T.E. Jaffe G.J. et al.Effect of ciliary neurotropic factor on retinal neurodegeneration in patients with macular telangiectasia type 2: a randomized clinical trial.Ophthalmology. 2019; 126: 540-549Abstract Full Text Full Text PDF PubMed Scopus (77) Google Scholar The current work did not identify safety concerns, and there seems to be efficacy for the outcomes assessed in response to CNTF for patients with idiopathic macular telangiectasia type 2. The well-designed, randomized Macular Telangiectasia Type 2-Phase 2 CNTF study provides evidence to support the use of objective measures of retinal structure as primary outcome measures for clinical trials. Because it represents one of the first reports to correlate OCT measures of retinal structure with retinal function measures using microperimetry, the study provides data to validate the use of OCT images of retinal structure that could provide objective, sensitive outcome measures of photoreceptor survival. Regulatory agencies such as the Food and Drug Administration and the European Medicines Agency consider validity, reliability, sensitivity to change, and clinical significance when considering new outcome measures that could support approval of new therapies.7Csaky K. Ferris 3rd, F. Chew E.Y. et al.Report from the NEI/FDA Endpoints Workshop on Age-Related Macular Degeneration and Inherited Retinal Diseases.Invest Ophthalmol Vis Sci. 2017; 58: 3456-3463Crossref PubMed Scopus (93) Google Scholar The Macular Telangiectasia Type 2-Phase 2 CNTF study not only suggests that CNTF is worthy of further study as a potential treatment for eyes with idiopathic macular telangiectasia type 2, but it also provides additional evidence that images of retinal structure can provide objective, reliable outcome measures for clinical trials that correlate strongly with retinal sensitivity and visual function. The current study sets a strong precedent and paves the way for future therapies to demonstrate improved photoreceptor survival using objective measures of retinal structure, which may permit completion of clinical trials in shorter periods than would be possible using traditional measures of visual acuity or visual field sensitivity. Effect of Ciliary Neurotrophic Factor on Retinal Neurodegeneration in Patients with Macular Telangiectasia Type 2: A Randomized Clinical TrialOphthalmologyVol. 126Issue 4PreviewTo test the effects of an encapsulated cell-based delivery of a neuroprotective agent, ciliary neurotrophic factor (CNTF), on progression of macular telangiectasia type 2, a neurodegenerative disease with no proven effective therapy. Full-Text PDF" @default.
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• W2924915481 title "Ciliary Neurotrophic Factor Treatment Improves Retinal Structure and Function in Macular Telangiectasia Type 2" @default.
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