Have you heard of the company QLT? They are a Canadian-based technology company dedicated to creating products designed to help visually impaired people and the doctors that treat them. They have been doing quite a bit of research into a drug that can help treat diseases like Leber’s Congenital Amaurosis (LCA) and Retinitis Pigmentosa (RP).
I must admit that when I first heard about this pill I thought it sounded far fetched. An oral medication that can cure blindness? You’ve got to be kidding! But the concept behind it actually makes sense, even to a layperson like myself.
In LCA and RP mutations in LRAT and RPE65 genes the problem is that an important molecule essential for vision is missing. In other words, if you have an LRAT or RPE65 mutation, your vision loss is due to an absence of this molecule (11-cis-retinal). What if we could replace that molecule (or one very similar to it) and get the visual cycle working again? What if we could create this molecule in the form of a pill?
This is working in dog and rat models and human trials have been looking good so far. More research is needed to make sure the drug is really safe and effective, but it is promising.
You may have some questions at this point. First of all, this would only work for patients with the LRAT or RPE65 gene mutations and it is estimated that this would be less than 10% of LCA and RP patients. Secondly, the study is ongoing and yes, they do recruit new patients. Check out ClinicalTrials.gov for more information on the research.
QLT has just released documents that will help answer more of your questions. Click the links below to access pdf versions of these documents that you can read in your browser or download to your computer:
- Rationale and Background for the development of QLT091001: General information about LCA, RP, and QLT091001.
- EURETINA 2011 Abstract: Information about the QLT091001 trials including results and conclusions.
- Oral Synthetic cis-Retinoid Therapy in Subjects with Leber Congenital Amaurosis: Information about the QLT091001 trials including results and conclusions.