With stem cells the focus of much recent attention in the media, this statement from Dr. David Gamm clarifies both the promise and the misconceptions about stem cell use for vision therapies.
Approximately 150 people heard Dr. Jose-Alain Sahel deliver the 5th Annual McPherson Endowed Lecture on Thursday, April 27th. Dr. Sahel's talked ranged widely over his work in recent decades, with particular emphasis on maintaining cone function in retinal degenerations.
Cone functional loss is the key event leading to blindness in rod-cone dystrophies. Dr. Sahel's lab embarked on a "fishing expedition" aimed at identifying the mechanisms underlying this loss, turning up potential clues for therapies aiming at preserving/restoring light-adapted and central vision in these patients. With collaborators Thierry Leveillard and Saddek Mohand-Said, Dr. Sahel discovered Rod-derived Cone Viability Factor, identified its receptor, and demonstrated its potential therapeutic benefit in several animal models, paving the way to upcoming clinical trials. This strategy has been extended to attempt to restore cone or inner retinal function by optogenetics. In depth phenotyping will guide the selection of target populations of patients that might benefit from these strategies; the development of novel, real life, functional outcome measurements will be needed to demonstrate its therapeutic value.
Dr. José-Alain Sahel, MD, PhD, is Professor and Chairman of the Department of Ophthalmology at the University of Pittsburgh Medical Center. He holds The Eye and Ear Foundation Endowed Chair of Ophthalmology in the University of Pittsburgh Medical School. Dr. Sahel is also the Director of Institut de la Vision at the Pierre et Marie Curie Medical School, Sorbonne Universites/Inserm/CNRS, Centre Hospitalier National d'Ophtalmologie, Paris.