Promising Findings for Leber Congenital Amaurosis Treatment Trial
Report from the annual ARVO (Association for Research in Vision and Ophthalmology) meeting
May 3, 2011 - Findings from a phase 1 trial of a new treatment drug for some types of Leber congenital amaurosis (LCA) and retinitis pigmentosa (RP) were presented at the 2011 ARVO meeting. This trial of an oral medication called QLT091001 is taking place in Montreal under the direction of Dr. Robert Koenekoop. Although he is working with QLT Inc on this clinical trial, Dr. Koenekoop has received funds from your donations for many years to identify the genes associated with LCA and other retinal degenerative diseases. QLT091001 is designed to treat people with mutations in one of two specific genes: LRAT or RPE65. It is only intended to benefit people whose vision loss is due to one of these mutations.
People with LCA were the first to enter the trial, so researchers were able to present preliminary information about 12 people with LCA who were enrolled early in the study. Six of these people had mutations in the LRAT gene; six in the RPE65 gene. The people involved in the trial were at different stages of vision loss from near normal to one participant who had no remaining visual field.
On the 12 people treated in the study, eight had improvement in their visual field (how much of the area in front of them they could see), their visual acuity (how clearly they could see), or both. Improvements in visual field size ranged from 22-217%, with the most dramatic improvements occurring in people who had smaller visual fields at the start of the trial. Improvements in visual acuity were also seen in four of the participants, including one who was unable to read any letters on the eye chart at the start of the study, but could read a number of letters nine days after treatment.
Although short-term headaches and light-sensitivity were reported by some participants, there were no serious ill effects. Some patients had small increases in triglyceride levels, but these levels normalized during the two-week testing period.
If this medication proves useful, people will likely take it for an extended period of time; however in this study people took the drug once a day for seven days and were then followed on a monthly basis. Despite this short period of treatment, in some people the improvements in vision lasted for more than 14 months.
”Clearly, additional studies are required to fully assess QLT091001 in these patients but we believe that these data are very promising,” said Bob Butchofsky, President and Chief Executive Officer of QLT. “We have obtained important clinical signals in this broad ranging study and plan to work diligently with the regulators towards a more pivotal evaluation of this molecule.”
“I am very encouraged by these results, which suggest that QLT091001 may have the potential to improve the lives of patients,” said Dr. Koenekoop. “The study results have clinical relevance for these LCA patients, and I look forward to continuing my work with QLT on this compound.”