New progress in therapeutic research on degenerative diseases of the retina

March 16 2009

During recent years, the team of Thierry Léveillard, Inserm Research Director, Saddek Mohand-Saïd and Professor José-Alain Sahel at the Paris Vision Institute have devoted their research to the study of Retinitis Pigmentosa, a group of inherited retinal diseases. In new studies published in the review Molecular Therapy, they demonstrated the protective role of RdCVF protein on visual function. The ultimate objective is to provide a treatment for these incurable diseases affecting 40,000 people in France.

Retinitis Pigmentosa is a group of genetic disorders that gradually attack the cells of the eye (photoreceptors) which convert the light signal into a signal interpretable by the brain, progressively leading to blindness. 40,000 people are affected in France. Although new approaches by gene therapy are under development, these diseases are still incurable today.

In patients with this disease, the rod photoreceptors involved in night vision are first affected. Degeneration of the cone photoreceptors essential for central vision and daytime visual acuity then occurs.

In 2004, researchers identified a protein with a strong potential for the treatment of inherited retinal degenerative diseases: RdCVF protein or rod-derived cone viability factor. This protein secreted by rods serves as a "nutrient" for cone photoreceptors. During the progression of the disease, the disappearance of rods causes the loss of RdCVF secretion followed by the degeneration of the cones and the progressive deterioration of associated visual function.

Today, Ying Yang, a researcher in the team directed by Thierry Léveillard, reports that the injection of RdCVF protein increases the number of cones present in the retina of an animal model, and importantly, that the vision of treated animals is twice as good as that of untreated animals. These results indicate that the protein not only ensures the survival of the cones but significantly preserves their function and therefore slows vision loss.

In parallel, Ram Fridlich, a student in the team, showed that another form of RdCVF protein made it possible to combat light-induced oxidative stress.

These research milestones should lead to the treatment of patients suffering from these diseases by an approach developed in partnership with the company Fovea-Pharmaceuticals.

Reinforced by this recent progress we are currently working with this partner to develop and validate a synthetic formulation of RdCVF protein that may be used in man. "Our goal after this last stage is to start the first clinical trials as rapidly as possible at the Quinze-Vingts Hospital Clinical Investigation Centre" explained Professor José-Alain Sahel.

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Functional Cone Rescue by RdCVF Protein in a Dominant Model of Retinitis Pigmentosa
Ying Yang1,2, Saddek Mohand-Said1-3, Aude Danan3, Manuel Simonutti1,2, Valérie Fontaine1,2, Emmanuelle Clerin1,2, Serge Picaud1,2, Thierry Léveillard1,2 and José-Alain Sahel1-5
1 Université Pierre et Marie Curie-Paris6, UMR-S 592, Paris, France;
2 Institut de la Vision, UMRS 968 Inserm - UPMC; Paris, France;
3 Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts, Department of Professor Sahel, Paris, France;
4 Fondation Ophtalmologique Adolphe de Rothschild, Department of Professor Sahel Paris, France
5 Institute of Ophthalmology, University College of London, London, UK;
Molecular Therapy: 10 March 2009

The thioredoxin-like protein RdCVFL interacts with Tau and inhibits its phosphorylation in the retina Ram
Fridlich1,2, Francois Delalande3, Céline Jaillard1,2, Jun Lu4, Laetitia Poidevin5, Thérèse Cronin1,2, Ludivine Perrocheau1,2, Géraldine Millet-Puel1,2, Marie-Laure Niepon1,2, Olivier Poch1,2,5, Arne Holmgren4, Alain Van Dorsselaer3, Jose-Alain Sahel1,2, Thierry Léveillard1,2
1 Pierre et Marie Curie-Paris 6 University, UMR-S 592, Paris, France;
2 Institut de la Vision, UMRS 968 Inserm - UPMC; Paris, France;
3 BioOrganic Mass Spectrometry Laboratory , IPHC-DSA, ULP, CNRS, Strasbourg, France ;
4 Medical Nobel Institute for Biochemistry, Karolinska Institute, Stockholm, Sweden;
5 Laboratory of Bio data processing and Integrative Genomics, IGBMC, Illkirch France
Mol Cell Proteomics. 11 March 2009

Scientists' contact
Thierry Léveillard
Phone: 01 53 46 25 48
José-Alain Sahel

Press contact:
Priscille Rivière
Phone: 01 44 23 60 97

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