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FDA-Approved Drug Moving into Clinical Trial for Treatment of RP

Valproic acid, a drug which has shown promise for preserving vision in people affected by autosomal dominant forms of retinitis pigmentosa (adRP), is moving into a Foundation-funded multicenter clinical trial this fall. 

The three-year, 90-participant clinical study will be conducted at three sites — the University of Massachusetts Medical School, the University of Utah, and the Retina Foundation of the Southwest — under the auspices of the National Eye Evaluation Research Network. The Foundation Fighting Blindness established the network to launch clinical trials of promising treatments and cures for retinal degenerative diseases. Valproic acid is the first treatment to be evaluated in the network.

Valproic acid appears to work by masking certain protein defects that can cause vision loss in some people with retinal degenerative diseases. Investigators also believe that the drug may have anti-oxidative and anti-inflammatory properties, which may help preserve vision, as well.

Shalesh Kaushal, M.D., Ph.D., the chair of ophthalmology at the University of Massachusetts Medical School, has played the lead role in investigating valproic acid and other related agents for the treatment of retinal degenerative diseases. In a paper published in the British Journal of Ophthalmology on July 20, 2010, Dr. Kaushal reports on the encouraging results he obtained while evaluating valproic acid in his patients with adRP.

“Dr. Kaushal has performed groundbreaking work in identifying how valproic acid and related compounds may benefit people with retinal diseases,” says Steve Bramer, Ph.D., chief drug development officer of the National Neurovision Research Institute, the clinical arm of the Foundation Fighting Blindness. “We are excited about working with him and other clinical experts to move the treatment into a formal human study and getting a clearer picture of the drug’s application and benefits.”

Valproic acid has already received FDA approval for the treatment of epilepsy, so the drug can potentially go through a quicker and less costly clinical trial process than would be possible if it were an unapproved, untested compound.

“Traditionally, moving a new scientific discovery from the bench to the patient requires a significant investment of time and resources,” said Kaushal. “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 potential new treatments to patients.”

While the clinical trial will be initially recruiting participants with adRP, the Foundation is currently funding preclinical studies of valproic acid for the treatment of other retinal diseases to see if the study may be expanded at a later date to include people with other forms of retinitis pigmentosa.

Dr. Bramer says that thorough clinical training and testing standardization will make certain that the trial delivers accurate results. “We are ensuring that all clinicians and staff are well-versed on the testing equipment, documentation requirements, and clinical protocols. This is a critical role for the Foundation,” he says. “By having everyone well-informed and on the same page, we’ll know with better certainty if the drug can preserve vision, and which people will benefit most from it.”

Participant recruitment information for the clinical trial will be posted at in the near future.

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