An experimental new treatment has been shown to reduce the activity of the gene responsible for Huntington’s disease for several months on end.
The EU-funded FINGERS4CURE project was led by researchers at Imperial College London and involved injecting mice with a special protein called a ‘zinc finger’.
Huntington’s disease is a genetic disorder that affects around 1 in every 10,000 people by damaging nerve cells in the brain. There is currently no cure and no way to slow the progression of the disease.
The disease is caused by a mutant form of a single gene called Huntingtin. What the ‘zinc finger’ does is target that mutant gene and then repress its ability to create the harmful proteins which then kill of nerve cells.
In the study, the team found that when they injected the mice with a single treatment of the ‘zinc finger’ they were able to repress the gene’s activities over a period of months with no side-effects.
Project lead Dr Mark Isalan from the Department of Life Sciences at Imperial said: “We are extremely excited by our latest results, which show a lot of promise for treating Huntington’s disease.”
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“However, while these encouraging results in mice mean that the zinc finger looks like a good candidate to take forward to human trials, we still need to do a lot of work first to answer important questions around the safety of the intervention, whether repeat treatments are effective, whether there might be longer-term side effects, and whether we can extend and increase the benefits beyond six months.”
One of the hurdles in combating the disease is making sure that any treatment doesn’t target both the mutant gene and the non-mutant copy of the gene.
As Dr Isalan points out, that’s just the start: “We don’t know exactly how the mutant Huntingtin gene causes the disease, so the idea is that targeting the gene expression cuts off the problem at its source - preventing it from ever having the potential to act,”
The team are hoping that they can start human trials within five years and are already working to see if they can increase the repression effects even longer.