`Best news’ for the Huntington Disease Community is delivered with unanswered questions

Experimental gene therapy seems to show a potential to slow down the debilitating progression of Huntington’s disease, a European biotechnology company that has developed the new treatment.

Huntington’s disease is a nervous destructive disease that takes place in families and damages movement, deprives memory and changes personality while people are in their lives. It is almost always deadly within 10 to 20 years of diagnosis, say neurologists.

Now, researchers are enthusiastic about the results of the small preliminary clinical study of a business of a treatment that targets the genetic error that causes the disease.

“I have a frantic optimism,” said Ed Wild, principal investigator of the Huntington’s Disease Center of the University College, who was involved in the trial, in an interview with CBC’s In this case.

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

The Dutch company, Unique, announced on Wednesday the preliminary results of experimental gene therapy, suggesting that therapy, administered during brain surgery, has slowed the increase in the disease of 75% over three years for patients with the highest dose. The trial was small, with around 30 patients registered and the treatment did not cure them of the disease.

So what does that mean?

It is a potentially promising development for a condition that has had little since the discovery of the defective gene behind the disease in 1993, but the experts say that there are still unanswered questions and work to do.

The slowdown in the progression of the disease, although it is not a remedy, could improve the quality of life of patients.

“People will be able to stay at work longer, they will be able to operate longer, they will be able to maintain their independence,” said Dr. Sarah Tabrizi, director of the University College Huntington’s Disease Center on Wednesday, who directed part of the trial, during the Unique call conference.

But what this slowdown in the progression of the disease could mean for people who are not candidates perfect for brain surgery to receive potential treatment is not clear. This includes if people with previous or later qualify stages.

Complete data has not been made public or evaluated by peers by company’s independent experts. These processes can help identify the defects in experimental design or shed light on important factors such as the best dose or the best time.

In a press release, the company said its higher results have demonstrated “a statistically significant slowdown in the progression of the disease”.

Dr. Jonathan Kimmelman, professor of bioethics at McGill University in Montreal, said that it was difficult to interpret and assess the real impact of the company’s announcement, given the lack of data accessible to the public.

“It could be very exciting. It could also be like many other press releases, that is to say that it could be a very exuberant and selective representation of what they have observed.”

Kimmelman also said that the information available suggests that the processing seems to slow down the disease, rather than cure or prevent it completely.

Others are more optimistic.

Rachel Harding, assistant professor at the Faculty of Pharmacy of Leslie Dan of the University of Toronto and editor -in -chief of HDBUZZ, a website focused on information on Huntington’s disease, said that it was a good feeling to finally see “something that seems full of hope in clinical trials”.

Harding, however, also recognized the limits, such as the small size of the trial sample – only 12 patients were followed for 36 months in the highest dose – and the lack of long -term data.

“Will the effects of the drug decline or deposit or, will you know, will it not work as well as people’s disease progress?” Said Harding. “We don’t know.”

Surgery and treatment considerations

Brain surgery lasts at least 12 hours, Wild said, and gene therapy should be delivered to two regions of the brain when scanning MRI in real time.

The treatment aims to correct the mutant hunting gene. Surgeons use a modified virus to provide the potential treatment of gene therapy, called AMT-1130.

The potential treatment aims to reduce mutant hunting protein levels that disrupt the normal function of neurons in the brain. Genetic therapies at one dose cost $ 2 million.

The company said that it had a meeting with the United States Food and Drug Administration later this year to share data with the Agency, before its submission planned in 2026 to the FDA for marketing approval.