Thanks to a $1 million gift to the Foundation Fighting Blindness, researchers are one step closer to curing a severe, vision-robbing retinal disease known as X-linked retinitis pigmentosa (XLRP).
The gift, provided by an anonymous donor, will be used to accelerate the advancement of potential XLRP treatments into and through clinical trials. The Foundation has established a consortium of the world’s leading XLRP investigators to identify treatment targets and preclinical disease models for potential therapies. The Foundation Fighting Blindness is currently working to raise a total of $3 million for the first phase of the effort, which will last three years. The second phase of the project will require substantially more revenue to fund clinical trials.
Initial funding will support XLRP gene therapy research at the University of Pennsylvania and the University of Florida, and transplantation and molecular research at the University of Michigan.
“This generous gift is a big boost in our efforts to overcome a particularly severe condition,” says Stephen Rose, Ph.D., Chief Research Officer, Foundation Fighting Blindness. “The development of our XLRP Consortium is an aggressive move to get potential treatments into clinical trials. We have the best researchers working collaboratively to find the answers as quickly as possible.”
Potential therapies for XLRP include: gene therapy to replace the unhealthy genes that cause vision loss; stem cells to restore and/or replace retinal tissue; and pharmaceutical treatments to slow or halt disease progress. “We are attacking XLRP on multiple fronts” says Rose. “Each approach has its advantages depending on the course and specific form of disease. We will leave no stone unturned.”
XLRP is a condition that primarily affects males. Females are carriers of XLRP, and usually do not develop symptoms. Experts estimate that at least 15 percent of all cases of retinitis pigmentosa are X-linked.
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