London: A single injection of a new treatment has been found to reduce the activity of the gene responsible for Huntington’s disease for six months in a trial in an animal model.
Huntington’s disease is a genetic disorder that causes neurological symptoms affecting movement, cognition and behaviour.
“We are extremely excited by our latest results, which show a lot of promise for treating Huntington’s disease,” said project lead Mark Isalan from Imperial College London.
Huntington’s usually only begins to show symptoms in adulthood. There is currently no cure and no way to slow the progression of the disease. Symptoms typically progress over 10-25 years until the person eventually dies, a university statement said.
In this study, the researchers tested the efficacy of a therapeutic protein called a “zinc finger”.
Huntington’s disease is caused by a mutant form of a single gene called Huntingtin. The zinc finger protein works by targeting the mutant copies of the Huntingtin gene, repressing its ability to express and create harmful proteins.
In the new study involving mice, published in the journal Molecular Neurodegeneration, the injection of zinc finger repressed the mutant copies of the gene for at least six months.
In a previous study in mice, the team had curbed the mutant gene’s activity for just a couple of weeks.
By tweaking the ingredients of the zinc finger in the new study they were able to extend its effects to several months, repressing the disease gene over that period without seeing any harmful side effects.
This involved making the zinc finger as invisible to the immune system as possible.
“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,” Isalan said.
“If all goes well and we have further positive results, we would aim to start clinical trials within five years to see whether the treatment could be safe and effective in humans,” Isalan pointed out.