University Of MassachusettsRetinitis pigmentosa (RP), an adverse neurodegenerative disease of the retina is believed to be caused by the death of photoreceptor cells. This ailment which affects 1 in every 4,000 individuals in the United States may eventually result in blindness. Researchers from the University of Massachusetts Medical School (UMMS) have ascertained a novel treatment for RP. Probable beneficial association between valproic acid and RP has been revealed.

Exhibited in young adulthood as night blindness or a loss of peripheral vision, the disease may ultimately progress to legal blindness by the age of 40. The FDA has supposedly approved valproic acid to decrease seizures, treat migraines and manage bipolar disorder. Usage of valproic acid may terminate vision loss in patients with RP. Also implementation of the acid has probably reported improvement in vision.

“Inflammation and cell death are key components of RP. It appears the valproic acid protects photoreceptor cells from this. If our observations can be further substantiated by randomized clinical trials then low dose valproic acid could have tremendous potential to help the thousands of people suffering from RP,” remarked Shalesh Kaushal, MD, PhD, chair of ophthalmology and associate professor of ophthalmology and cell biology at UMMS.

Artificial insemination displayed valproic acid to be an efficient treatment for photoreceptor loss correlated with RP. The present research findings are based on a three-year clinical trial undertaken to ascertain the capacity of valproic acid as a treatment for RP. Just like the retrospective analysis, the latest investigation also treated patients’ off-label with doses of valproic acid ranging from 500mg to 750mg daily over the course of two to six months.

Kaushal commented, “Traditionally, moving a new scientific discovery from the bench to the patient requires a significant investment of time and resources. Repurposing drugs already approved by the FDA and which have been shown to be safe, such as valproic acid, is an economical and time-efficient way to quickly bring new treatments to patients.”

Improvement in vision was registered in five of the seven patients in the research who were treated when normally due to RP patients suffer rapid vision loss. The experts mentioned that with more than 40 different genes, determining a potential treatment for RP is not easy. Therefore approximately 100,000 patients in the U.S. remain untreated with this ailment.

Steve Bramer, Ph.D., chief drug development officer, National Neurovision Research Institute, a clinical support arm of the Foundation Fighting Blindness added, “The Foundation Fighting Blindness is delighted to be moving Dr. Kaushal’s outstanding work with valproic acid into our clinical trial network, because the drug has the potential to preserve vision for thousands of people affected by retinal diseases. It’s an exciting research collaboration for us, because of the drug’s potential, and the knowledge and expertise Dr. Kaushal and the University of Massachusetts Medical School bring to the clinical research.”

Therapies ascertaining nutritional supplementation, vitamin A supplementation, light reduction or gene therapy are being conducted. The qualities of a potent inhibitor of the inflammatory response pathway and cell death, valproic acid was known to the researchers. This enabled them to determine the acid to be a treatment for retinal disease.

The research is published in the July 20 online edition of the British Journal of Ophthalmology.