A Ray of Hope
Scientists from Imperial College London, UK started testing a new treatment for Huntington's disease on mice. Researchers are seeing an effectiveness even up to six months after the initial treatment.
Huntington's disease is a hereditary, progressive brain disorder that affects movement, speech, and cognition, among other symptoms. As for now, the disease is incurable and patients usually only live 15-20 years after the first symptoms appear.
Huntington's occurs when a mutation produces a longer than normal Huntingtin gene. The gene is toxic to some cell types causing the brain damage which then causes the symptoms.
Scientists aren't sure how the disease damages the brain, but the Imperial team has a brilliant plan. "We don’t know exactly how the mutant Huntington 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," says Mark Isalan, the lead researcher on the project. The research team came up with a new treatment using a modified protein called a 'zinc finger.' The proteins cling to the faulty Huntington genes and prevents them from releasing proteins that are harmful to the brain.
Trials Seeing Success
In their most recent trials, the team injected 12 mice with the blocking protein. After three weeks, 77% of Huntington was repressed. Percentage of repression declined in the next weeks, but even after 6 months, gene expression was still being curbed. "In this study we weren’t looking at how repressing the gene activity affected the symptoms of the disease, and this is obviously a critical question as well. However, we have reason to be confident from our previous studies that repressing the gene does in fact significantly reduce symptoms," explains Isalan. The results were published in Molecular Neurodegeneration.
As with any animal trials of human treatments, there are a few things to keep in mind. According to ScienceAlert, there is no way to ensure the treatment will also translate to humans. Also, there is no concrete proof that protein build-up from the mutation is to blame. Finally, while these trials show that the gene expression can be blocked, there hasn't been any testing if this also stops symptoms.
If results continue to be promising, human trials can begin within the next 5 years.