Our friend Fran attended the 16th conference organized by Retina International, which was held in Italy last weekend. It wasn't specifically on LCA but most LCA experts were there! It was very interesting. Here are her highlights:
Dr. Jean Bennett presented the astounding results of the RPE65 trials. Children who were nearly blind can now play baseball and walk without a cane! We all had read that, but to hear it from her was exciting.
Dr. Koenekoop showed the incredible results of the QLT drug for LRAT LCA. After only nine days of oral administration, vision improved dramatically, and the visual field kept increasing until it tripled! It produced improvements even in an adult patient. He said they will now test it on people with RPE65. To the question about future application of similar drugs to different LCA genes, he said that it may be possible, but didn't go into details.
Many researchers said that soon gene therapy trials for other genes will be planned. Jean Bennett said that she cannot say which will come next, but different labs are working on diferent animal models. Some of the candidates for clinical trials that were mentioned were AIPL1, LCA5, RPGRIP, Gucy2d, and CEP290. CEP290 is being studied a lot, the problem with it is that it is a large gene, but many labs are working on building the right vector to get it into the retina; once it is found, things should go fast from mice to cats to humans.
A talk was given about the retina prosthesis. It's promising, though the visual field it allows is still pretty small. We were shown a video where a blind person receiving the implant was able to read a few letters on a chart and recognize some objects. For now they will not try it on people blind from birth.
Unfortunately I missed the lecture on stem cells by Robert Ali. Comments I heard were that research is progressing, but application is a bit further away.
An exciting new development I had never heard about before is photoswitch, which is a strategy that aims to activate ganglian cells of the retina to interpret light and send signals to the brain, bypassing photoreceptors. It isn't working yet, but Dr. Chader was confident that it might work soon, and make it to human trials. This is wonderful as it would work even in people with no living photoreceptors.
A lot was said about drugs like nerve growth factors which can prolong the life of photoreceptors, but I didn't follow those too closely, sorry.
If anyone has any questions of course feel free to ask, and I'll see if I can remember more.
Thanks Fran! You can read more about Fran and her visit to the conference on her blog: LCA Blog.
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