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Kauper K. et al, IOVS 2012
NT-501 ECT consists of encapsulated human cells genetically modified to secrete therapeutic doses of ciliary neurotrophic factor (CNTF) into the back of the eye for the treatment of retinal degenerative diseases. Neurotech is conducting multiple clinical studies with NT-501 ECT, including trials in subjects with macular telangectasia (MacTel) and glaucoma. Neurotech also collaborates with research ophthalmologists in investigator-initiated clinical trials for other neurotrophic ocular diseases.
A total of 184 subjects have been enrolled in three separate Phase 2 studies in the US, with some subjects reaching past 50 months post-implantation.
Ciliary neurotrophic factor (CNTF), one of several neurotrophic factors produced endogenously by neurons or Mueller cells, has been well-studied since its identification over three decades ago 1. CNTF is expressed in the retina under stressful conditions such as experimental ocular hypertension 2 and optic nerve trauma 3, and acts as an injury-activated signal to protect neural tissues, including the retina. CNTF has been shown to have a neuroprotective effect on photoreceptors, the cells responsible for detecting light in the retina, and animal models of retinal degeneration have suggested that this neuroprotective effect slows vision loss due to photoreceptor death.
Phase 2 Macular Telangiectasia (MacTel) Trials
Neurotech is conducting a phase 2 study (NCT01949324) using NT-501 ECT for the treatment of subjects with Macular Telangiectasia, an idiopathic condition that includes a vascular disorder of the macula, where juxtafoveal angiogenesis leads to loss of the central vision. MacTel is a slow progressing disease, manifesting over a period of 10-20 years. The MacTel Project has identified CNTF as a potential modulator of vision loss in MacTel disease and is collaborating with Neurotech in investigating potential benefit of long term treatment with NT-501.
Neurotech has received Orphan Drug designation for NT-501 ECT for the treatment of Macular Telangiectasia from the U.S. Food and Drug Administration.
1. Adler R, Landa KB, Manthorpe M, Varon S. Cholinergic neuronotrophic factors: intraocular distribution of soluble trophic activity for ciliary neurons. Science. 1979; 204:1434-1436..
2. Yu S, Tanabe T, Yoshimura N. A rat model of glaucoma induced by episcleral vein ligation. Exp Eye Res. 2006;83(4):758-770.
3. Valter K, Bisti S, Gargini C, et al. Time course of neurotrophic factor upregulation and retinal protection against light-induced damage after optic nerve section. Invest Ophthalmol Vis Sci. 2005;46(5):1748-1754.