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In 2005, French researchers José Sahel, M.D., and Thierry Léveillard, Ph.D., won the Foundation’s Annual Trustee Award for the most promising advance of the year: Identifying a protein that shows great potential for preserving vision in people affected by a variety of retinal degenerative diseases. Sahel and Léveillard, both with the Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts in Paris, are now part of a new FFB-funded research center chartered with moving this promising protein into clinical trials.
The protein, known as rod-derived cone-viability factor (RdCVF), is produced by rods, which give us night and peripheral vision. However, the investigators found it to be essential for survival of cones, which give us central and detailed vision.
Cones are most critical for tasks of daily living. Studies have shown that even if someone has lost 95 percent of their cones, they still have enough vision to navigate without assistance. Therefore, saving cones is a good strategy for saving usable vision, and RdCVF may be just what the cones need for long-term survival.
Sahel and Léveillard are developing a gene therapy to provide long-term, sustained delivery of RdCVF to an affected person’s retina. If animal studies of the treatment are successful, they hope to launch a human clinical trial in about three years.
In addition to the work on RdCVF, the Foundation’s grant to the Paris group is funding evaluation and monitoring technologies and instruments used in clinical trials. Saddek Mohand-Said, M.D., Ph.D., also of the Centre Hospitalier, will be leading the implementation of these tools to evaluate the efficacy of therapies in clinical trials. This facility is a leading center in Europe for human studies of emerging treatments for retinal degenerative diseases.
Measuring a given treatment’s benefit goes well beyond what a person sees on a standard eye chart. At the Paris Center, clinicians will be using sophisticated tools to look at changes in patients’ retinas in response to a therapy. In addition to routine tests such as visual acuity, visual field, and standard dark adaptation and contrast sensitivity evaluations, the clinicians will use innovative equipment to obtain retinal images and measure the retina’s sensitivity. These evaluation methods provide the most reliable, accurate, and objective data.
“Many people with a retinal condition can have reasonable visual acuity, even close to 20/20 vision, but have an especially difficult time seeing in the dark or seeing objects that aren’t directly in front of them,” says Stephen Rose, Ph.D., Chief Research Officer, Foundation Fighting Blindness. “For us to accurately determine the benefit of a given treatment, it is essential that we have places like the Paris Center where sophisticated imaging and monitoring tools are implemented to look at changes in the retina itself.”
Also as part of the Paris Center Grant, Christine Petit, M.D., Ph.D., Professor at College de France and at Institut Pasteur, a world-renowned Usher syndrome researcher, will be conducting preclinical investigations of disease mechanisms in Usher syndrome type 1. Of the three Usher syndrome types, type 1 is particularly severe, causing profound hearing impairment at birth, progressive vision loss before adolescence, and balance problems. Petit is focusing on the earliest stages of the disease process, which impact hearing at birth, with the goal of identifying treatment targets that can minimize vision loss, which occurs later in childhood.
The Foundation established the Paris Center, coordinated by Sahel, because of its strength and scope in both preclinical and clinical research, as well as the promising treatments they already have in the pipeline. “The Paris Center Grant provides a good balance of clinical and preclinical resources and expertise. That’s critical for the development of treatments and cures, and moving them into human studies,” says Dr. Rose. “Furthermore, Dr. Sahel and his colleagues have made key advancements in the development of treatments, and we are excited about helping them get those into the clinic and to the people who need them.
DISCLAIMER: Physicians differ in their approach to incorporating research results into their clinical practice. You should always consult with and be guided by your Physician’s advice when considering treatment based on research results.
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