Huntington's disease sees first-ever successful treatment in medical breakthrough

Scientists have achieved first-ever successful treatment for one of the most devastating genetic diseases, Huntington’s in a recent medical breakthrough.

In the pivotal research study, the team of doctors has shown 75 percent efficacy of treatment against Huntington’s disease.

While talking to BBC News, Professor Sarah Tabrizi said, the deterioration in health condition that patients usually expect after one year would be delayed to a span of four years after this revolutionary treatment, giving the patients a good quality life.

The new treatment consists of a gene therapy that is given to patients during 12 to 18 hours of sensitive brain surgery.

Being a genetic disease, Huntington’s usually runs through families, killing brain cells inexorably. The disease shows diverse symptoms, combining Parkinson’s, dementia, and motor neuron disease.

Prof Tabrizi, director of the University College London Huntington's Disease Centre, called the results "spectacular".

"We never in our wildest dreams would have expected a 75% slowing of clinical progression," she said.

Although the gene therapy treatment is very expensive, the promising results offer real hope against the life-threatening disease.

Huntington’s disease is caused by a mutation of a gene called the huntingtin gene, turning the normal protein into a killer of neurons.

The proposed gene therapy utilizes cutting-edge gene silencing technology and genetic medicine combining gene therapy.

The procedure starts with the altered virus that contains a specially designed sequence of DNA, which is infused into the brain using MRI.

The virus delivers the new piece of DNA inside brain cells and activates it. As a result, the DNA turns neurons into a factory, helping them to prevent their own death.

The cells also produce microRNA that helps in disabling instructions being sent from cells’ DNA for producing mutant huntingtin, thereby leading to reduced levels of mutant gene.