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    Trophic factor replacement targets most debilitating effects of RP

    Though not a cure, approach may maintain cone vitality; prevent loss of central vision

    Take-Home Message: Degeneration of rods in eyes with RP leads to night vision problems and loss of rod-derived cone viability factor (RdCVF) that is necessary for cone survival. Replacing RdCVF may prevent cone degeneration and loss of central vision.

    (Figure 1) Immunohistochemical analysis of the expression of basigin-1, the rod-derived cone viability factor cell surface receptor, in the macaque retina. Basigin-1 expression localized to inner segments and outer segments of cones, identified by their unique morphology. Brown color on the top: Retinal pigmented epithelium. (Image courtesy of Thierry Léveillard, PhD)

    Paris—Replacement of rod-derived cone viability factor (RdCVF) is being looked to as a potential strategy for maintaining cone-mediated central vision in patients with retinitis pigmentosa (RP), according to Thierry Léveillard, PhD.

    “As RdCVF is a trophic factor that promotes the survival of cones, its replacement could maintain cone viability and prevent the loss of central vision that is the major handicap occurring with RP,” said Dr. Léveillard, director of research, Institut National de Santé et de Recherche Médicale (INSERM), The Vision Institute, Pierre and Marie Curie University, Paris, France.

    “This approach is not a cure for RP since it does not correct a genetic defect that causes the disease,” Dr. Léveillard explained.

    “However, genetic approaches to RP treatment are limited by the fact that RP is a genetically heterogeneous disease with 54 identified causative genes,” Dr. Léveillard said. “Replacement of RdCVF is an attractive strategy because it addresses a common pathway leading to cone degeneration, regardless of the causative mutation.”

    Dr. Léveillard and José Sahel, MD, professor of ophthalmology, Pierre and Marie Curie University, reported the first fundamental discovery of RdCVF in mice in the journal Nature Genetics in 2004. Since then, they have published several papers describing their research findings and presented some of their most recent studies at the 2015 meeting of the Association for Research in Vision and Ophthalmology.

    Supporting evidence

    Evidence to support RdCVF replacement as a useful treatment in RP is available from preclinical studies demonstrating that it protected cone function in two genetically distinct animal models of RP.

    Cheryl Guttman Krader
    Cheryl Guttman is a medical writer based in Deerfield, Ill.

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