“The Best News” for the Huntington disease community comes with unanswered questions

Experimental genetic therapy seems to be the possibility of slowing the exhausted progress of Huntington, a European biotechnology company that has developed the new treatment.

Huntington’s disease is a disease that destroys the nerves that work in captivity and harms movement, steals memory and changes the personality while people are in their lives. Neurons specialists say they are always fatal within 10 to 20 years of diagnosis.

Now, researchers feel excited about the results of a preliminary clinical study of the company for a treatment targeting the genetic error that causes the disease.

“I have failed optimism,” Ed Wilde, the main investigator at the Center for Diseases at the University of Huntington at the University of Huntington who participated in the trial, in an interview with CBC’s As it happens.

“This is the best news that we have ever made in the Huntington community.”

The Netherlands -based UNIQURE announced preliminary results on Wednesday of experimental genetic therapy, which indicates the treatment, which is managed during brain surgery, with a slowdown of the development of the disease by 75 percent over a period of three years for patients with the highest dose. The experiment was small, where about 30 patients were joined, and treatment was not treated with the disease.

So what does it mean?

It is a promising development that is likely to have a few of them since the detection of the wrong gene behind the disease in 1993, but experts say that there are still questions that have not been answered and more work.

The slowingness of the disease, although it is not a treatment, can improve the quality of patients ’life.

“People will be able to stay working for a longer period, and they will be able to work for a longer period, and they will be able to maintain their independence,” said Dr. Sarah Tabrizi, Director of the University’s Disease Center at the University of Huntington, who led part of the trial, during a phone call at UNIQURE on Wednesday.

But what might mean the slowdown in the development of the disease for people who are not ideal candidates for brain surgery to receive potential treatment is unclear. This includes whether people with an early or late stage may qualify.

The full data of the public or the peers has not been published by experts independent of the company. These processes can help determine defects in experimental design or shed light on important factors such as the best dose or timing.

In a media statement, the company said that its highest results showed “a statistically slowing down in the development of the disease.”

It is difficult to explain and evaluate the real impact of the company’s announcement, given the absence of data available to the public.

“It might be very exciting. It may also be like many other press releases, that is, it may be a great and selective representation of what they noticed.”

Kimmelman also said that the available information indicates that the treatment appears to slow the disease, rather than treat it or prevent it completely exacerbated.

Others are more optimistic.

“Something that seems optimistic about clinical trials.

However, Harding also admitted restrictions, such as the small sample size in the experiment-only 12 patients have been followed for 36 months at the highest dose-and no long-term data.

“Will the effects of the drug be affected, decrease, or, as you know, do not work as well as the progress of people’s disease?” Harding said. “We do not know.”

Treatment surgery and considerations

Wilde said that brain surgery takes at least 12 hours, and genetic therapy should be delivered to two areas of the brain while wiping MRI in real time.

Treatment aims to correct the transformed fishing gene. Surgeons use an average virus to provide potential genetic therapy, called AMT-1330.

Possible treatment aims to lower levels of transformed hunter protein that disrupts the normal function of neurons in the brain. Treatments of genes preceding a single dose Assign $ 2 million.

The company said it is holding a meeting with the US Food and Drug Administration later this year to exchange data with the agency, before it submitted 2026 planned to the Food and Drug Administration to agree to marketing.