Share The advent of anti-VEGF injections changed the face of treatment for macular degeneration and saved the vision of thousands of people around the world. But, the burden of monthly treatment and the cost have been a downside to this breakthrough.  Some patients do well with less frequent injections, but wouldn't it be nice to skip the shot in the eye entirely?

That day is not here yet, but it is getting closer.  An eye drop for wet AMD is under development and two recent animal studies are promising.

Squalamine is a small molecule that is anti-angiogenic (interfers with the factors that cause blood vessels to grow).  In this, it is similar to Lucentis, Macugen and Avastin.  But, while those treatments only block VEGF (vascular endothelial growth factor), Squalamine also blocks PDGF (platelet derived growth factor), which is newly seen as another target for treating wet AMD.

Previous human clinical trials used Squalamine as an intravenous infusion and was shown to be safe and effective in wet AMD.  But, over an extended period of time, there was a sharp decline in drug concentrations between weekly treatments.

In animal studies, the Squalamine eye drop did not have this problem.  The drug reached the retina well over the dose needed to stop blood vessel growth and the dose could be maintained by daily treatment.  Importantly, the drug did not seem to penetrate other structures, like the cornea, or reach the lens of the eye.  Safety data was excellent.

These eye drops would be self-administered by patients.  They might be used in combination with current injection therapy to hopefully reduce the number of injections needed - or possibly replace them.

We need large clinical trials before the safety and effectiveness of Squalimine eye drops is shown in humans.  It certainly would be a significant improvement over monthly injections.

July 2010