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Hope For Patients With Macular Degeneration From Manmade Protein

ouple it with a four-amino-acid peptide tail,' he says. 'The tail essentially handcuffs the manmade molecule within the protein-making machinery of the cell so that it stays there and anything that binds with it, namely VEGF, stays there too. So it's a very specific way of down-regulating a target protein.'

In May 2005, Dr. Ambati and his colleagues published work in Investigative Ophthalmology & Visual Science showing the intraceptor helped reduce blood vessel development in the test tube and animal models for corneal injury and melanoma.

'Now we are talking about making them go away,' says Dr. Ambati. While the work is still in the laboratory, it provides further evidence of the intraceptor’s potential clinical application, he says.

The work shows the intraceptor prompts regression of blood vessels by inducing programmed cell death, or apoptosis, in the vascular endothelial cells that line the vessels.

'The biology of all this is showing this molecule interrupts the proper folding of proteins involved in existing blood vessels, which makes them die. It's a nice result,' says Dr. Ambati.

Some existing anti-angiogenesis treatments target VEGF outside cells. 'It is important to bind it within cells because certain cells, such as cancer and blood vessel cells, have the capability to produce their own VEGF and their own receptors,' Dr. Ambati says. 'Imagine trying to block from the outside a factory that has everything it needs inside. You have to throw a monkey wrench inside the factory and that is what we managed to do.'

For the study, the manmade protein was injected directly into the cornea with a microneedle. 'Ideally we would like to develop a topical eye drop with a long-term delivery system,' says Dr. Ambati.

His research team is pursuing its work of the intraceptor's potential role in destroying blood vessels that help sustain cancers. They also are looking at a biodegradable pol
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