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Oral medication for Leber's congenital amaurosis demonstrates biological signal in early study
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VANCOUVER, British Columbia — Three patients enrolled in a phase 1b proof of concept study for an oral medication therapy for Leber's congenital amaurosis had vision gains and reported improvements in functional vision after 7 days of treatment.
According to a press release from QLT announcing the interim study results, the three patients, age 10, 12 and 38 years, had rapid improvement in best corrected visual acuity, Goldmann visual field testing and/or retinal sensitivity after initiating therapy with QLT091001, a synthetic retinoid replacement therapy. Improvements were most pronounced in younger patients, and results continued to improve over time, up to 4 months, the release said.
All three patients share a common genetic mutation in lecithin:retinol acyltransferase. Future enrollment will include patients with genetic mutations at RPE65, according to the release. The study drug is a synthetic retinoid replacement for 11-cis-retinal.
"These preliminary results are very exciting, are better than expected, and provide a measure of hope that a treatment might be developed for this devastating disease," Robert K. Koenekoop, MD, PhD, the trial's principal investigator, said in the release.
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