February 28, 2006.
New York - An intraocular implant containing a protein called CNTF, which has been shown to slow retinal degeneration in animals, may improve vision in patients with retinal disease, findings from a study suggest.
A variety of genetic mechanisms underlie degenerative retinal diseases, but regardless of the cause the end result is the death of photoreceptor cells. This observation has led researchers to look for therapeutic modalities that would work against these diseases, regardless of the genetic etiology involved.
Dr Paul A Sieving and colleagues delivered CNTF by incorporating the CNTF gene into cells placed in a semi-permeable capsule. This capsule was then implanted into a disease-affected eye in 10 subjects.
At six months the capsules were removed and, in all cases, viable cells were observed producing quantities of CNTF that have been shown to be "therapeutic" in animals.
Three of seven "evaluable" eyes demonstrated clinically relevant improvements in visual acuity.
The implantation procedure and the presence of the cell capsule appeared to be safe and well tolerated.
Sieving, from the National Institutes of Health in Bethesda, Maryland, and colleagues report their work in the Proceedings of the National Academy of Science.
"This phase I trial indicated that CNTF is safe for the human retina even with severely compromised photoreceptors," the authors conclude. "The approach to delivering therapeutic proteins to degenerating retinas using encapsulated cell implants may have application beyond disease caused by genetic mutations."
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