Canadian Trial To Enrol Retinitis Pigmentosa Patients
Oct 28, 2010 - Canadian researchers have announced that they are expanding their clinical trial of a vision therapy for people with Leber congenital amaurosis (LCA) to include some individuals with retinitis pigmentosa. The therapy, currently known as QLT091001, is being developed by the Canadian biotechnology company QLT Inc. The trial is being led by Dr. Robert Koenekoop of Montreal Children's Hospital - a leading Canadian scientist who has long received support from the Foundation Fighting Blindness.
QLT091001 is an oral medication specifically designed to treat patients with genetic mutations in either retinal pigment epithelium protein 65 (RPE65) or lecithin:retinol acyltransferase (LRAT). Mutations in these genes may be present in people with either LCA or retinitis pigmentosa. The treatment aims to replace a biochemical substance, 11-cis-retinal, which is key to healthy vision. This substance is absent in people with these gene defects.
There are at least 300,000 patients with retinitis pigmentosa worldwide, of which less than 5% carry inherited deficiencies in either RPE65 or LRAT. Only these individuals might potential benefit from this trial. Appropriate trial participants will be identified using an innovative tool for genetic screening, first developed by Dr. Koenekoop and his colleagues, with funds from the Foundation Fighting Blindness.
The trial will recruit up to 24 patients with LCA or retinitis pigmentosa who carry the appropriate RPE65 or LRAT mutations. All participants will receive daily oral treatments of one of two doses of QLT091001 for seven days at the Montreal Children's Hospital at the McGill University Health Centre, Montreal, Canada under the supervision Dr. Koenekoop. The primary purpose of this early trial is to ensure that this treatment is safe and to establish an appropriate dosage for a larger trial.
"We continue to enroll LCA patients in the trial," said Bob Butchofsky, President and Chief Executive Officer of QLT, "with a total of 7 patients currently treated or under treatment. We expect to treat the first retinitis pigmentosa patient in the coming weeks."
Earlier this year, Dr. Robert Koenekoop, shared information about the outcomes for the first three LCA patients enrolled in this study at the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO). After seven days of treatment with QLT091001, each of these patients had experienced clinically relevant improvements in one or more of the tests used to assess their vision. The onset of visual changes was rapid and there was progressive improvement beyond the treatment period. Some of these improvements persisted for up to four months after only seven days of treatment. See our previous news story for more details.
For more information about enrolling in this study, including detailed study criteria, please contact the study's Research Coordinator, Eunice Esteban, at (514) 758-7724.