Grant: Proof of Principle Program - Phase I

Age related macular degeneration (AMD) is the commonest cause of blindness in the industrialized world. There are 2 types of AMD, and treatments are available for only one, which affects 10% of all cases, and which does not always work. Moreover, there is an obvious and urgent need to develop therapies for the other 90% of patients. Research has shown that a system, known as the complement system, is overactive in the eyes of patients with AMD, and this leads to damage to some of the cells in the retina (the part of the eye that is responsible for sight). Additional factors that occur during aging, exacerbate the damage to the cells in the retina. One way to prevent damage is to reduce activation of the complement system. For many years, our lab has been working on ways to achieve that. We have recently found that a natural product in the blood called polyphosphate, reduces activation of the complement system. Through studies in blood, we have figured out how it works. We have also shown in early studies that polyphosphate reduces AMD in a rat model, and that it also protects the retinal cells from injury. Polyphosphate is not likely to have side effects, since it occurs naturally in the body. We propose to further develop the use of polyphosphate for commercial purposes. We intend to further test the protective properties of polyphosphate in rodent models of both forms of AMD. We will confirm that the polyphosphate does not have any side effects. We will also develop longer-last forms of the polyphosphate so that it won't have to be injected so frequently. Overall, polyphosphate holds promise for a new therapeutic agent that will provide huge benefit for the millions of people worldwide who are and will suffer from this major cause of reduced vision and blindness.