Genetic therapy slows Huntington by 75 % in the “leading” clinical experiment

Doctors in the United Kingdom claim that they had made a huge medical breakthrough. Using a form of genetic therapy, successfully treated – for the first time – Huntington’s nervous degeneration disorder.

On Wednesday, the company UNIQURE Declare The results of the first/second stage experience in the treatment test, amt-130 called symbol. The experiment, which was conducted with the help of researchers from the University of College London, found that AMT-130 has invalidated 75 % of people’s symptoms. It also appears to reduce the death of brain cells, which is the advantage of signing the disease. The first days are still, but the results can soon preach the arrival of a great treatment for the current life.

“I think this leading data is the most convincing in this field and affects statement From ucl.

A genetic curse

the illness It is run by a mutation in the Huntttin gene, which causes the body to produce a flawed version of the Huntingtin protein. Then this protein gradually destroys the cells of the brain, especially those in areas that govern movement and perception. The genetic mutation that causes Huntington is dominant, which means that people only need to obtain one copy of one of the parents for the occurrence of the disease; As such, the person has a 50 % chance of his inheritance if one of his parents carries the mutation.

People usually live in the thirties and forties of their age before the symptoms appear, such as the troubles of moving. But the disease gradually exacerbates from there, and people will eventually develop dementia and other serious health problems. Most people with Huntington live only 15 to 20 years as soon as the disease appears. Although there are treatments that can help people manage their symptoms, there is no treatment that will get out of the destruction of the inevitable brain – so far, perhaps.

Huntington short circle

Amt-130 aims to a short circle of the operation that Huntington is slowly killing the brain.

Using a neutral virus to connect the DNA to a person’s brain cells, the treatment guides these cells to produce a little genetic substance called microrna. This microrna should hinder the ability of cells to produce a hunter protein (both natural and transformed versions), and we in turn hope to reduce the damage caused by the disease. Since brain cells are not constantly recycled like other parts of the body, genetic therapy should be a one -time treatment.

The basic part of the experiment included 29 patients with early Huntington who were treated with AMT-130 (some patients in the control group will receive treatment later). In 36 months, the treatment achieved its main goal, and it appears to slow the symptoms of people by 75 %, as ruled by a common scale used to assess the disease. Biological signs tests also indicated that treatment for more expected brain cell death. Moreover, the results were more impressive in people with the highest dose, indicating a real therapeutic response (the effect of the dose response).

Although none of the patients have been determined, some still walk although it is expected to need a wheelchair by this point, BBC I mentioned Wednesday, while one person returned to work after medical retirement at first. Although it is still very early to know this with certainty, it can provide an improvement with AMT-1330 years, and even contracts of extended life and good health for patients.

Treatment appears to be safe and endured, with most negative events associated with general anesthesia and surgery needed to provide treatment to the brain.

AMT-130 future

More importantly, the company’s data has not been reviewed and evaluated by external researchers, an important part of the scientific process. So these results remain preliminary at the present time.

But the company is definitely spent on its data to determine the audit. It plans to meet the Food and Drug Administration later this year, and the assumption that everything is fine, will formally submit a request for treatment approval early next year.

Even if this treatment is approved, there will be important questions about how easy it is to reach it at reasonable prices for the people they need. Genetics treatments are generally expensive, and Amt-130 is unlikely to be exceptions. But for the first time ever, patients who suffer from this unfinished condition may be real hope.