Scientists at University College Cork (UCC) have come up with an innovative strategy to deliver a therapy into the brain to treat the neurogenerative disease, Huntington’s disease (HD).
The strategy, which involves using modified sugars to carry gene therapies into the brain, is being hailed as an exciting development which could be applied to many brain disorders, especially those with a genetic basis.
Brain disorders affect about 30 per cent of the Irish population annually with immense social and economic cost. However, there are two major challenges in developing therapeutic strategies for the brain – the blood brain barrier keeps most medicines out of the brain, and brain cells (neurons) themselves are very difficult to penetrate in a safe manner.
Prof Caitríona O’Driscoll from the School of Pharmacy at UCC and her long-standing collaborator, Dr Raphael Darcy of University College Dublin, have developed sugar-based carriers called cyclodextrins which are able to encapsulate and transport molecules which silence the expression of toxic genes. They collaborated with neuroscientist Prof John F. Cryan of the Department of Anatomy and Neuroscience, UCC to apply this novel technology to brain disorders.
The team has delivered a therapy that successfully silences a gene responsible for Huntington’s disease, a rare disease estimated to affect 10 people in 10,000. Their research findings have just been published online in the prestigious journal Molecular Pharmaceutics.
Prof Cryan noted, “Whilst these experiments in cells and model systems are encouraging, we are still a long way from transferring this technology to human use.”
