This is a fantastic opportunity and this is really what academic research
is all about.
So then what would be the processes that copper would be important for?
That's something to think about. And so
Wilson's disease comes in at this point
Mutation that occur in Wilson's disease lead to accumulation of copper in the liver.
The liver fills up with copper and then when the liver is full it spills over into the
brain and it causes neurological complications. About 1 in 30,000 people
is thought to suffer from Wilson's disease and it may be even higher than that.
What we were able to show in this mouse model was that we could treat the
mice with our compound and you could see that the levels of copper in the liver
were reduced dramatically the levels of copper in the brain were reduced dramatically.
That was quite striking. So, we have a molecule that is orally bioavailable,
very specific for copper, crosses the blood-brain barrier,
and is rapidly excreted from the animal as a copper complex through the feces.
This shows a number of properties that are very exciting as a potential therapeutic
to treat Wilson's disease.
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