Possible breakthrough: Huntington's disease drug successfully tested

So far incurable nerve disease: New drug for Huntington's chorea

It could be a possible breakthrough in the fight against previously incurable Huntington's disease: British researchers have developed a drug for the rare nerve disease that has proven to be promising in initial tests.

Rare brain disease

Huntington's disease is a very rare, inheritable disease of the brain. So far, the nerve disease, which is also called Huntington's disease or Huntington's disease and formerly known as "Veitstanz", has not been cured. However, researchers from the UK have now developed a drug that gives hope.

Up to 10,000 people in Germany are affected

According to experts, around 8,000 to 10,000 people in Germany suffer from Huntington's disease.

The course of the progressing disease, which usually breaks out between the ages of 35 and 45, is individual and varies from patient to patient.

“Most of the time, at the beginning of the disease, the focus is on progressive psychological abnormalities: the patients are depressed or increasingly irritable and aggressive or uninhibited; others notice a loss of mental abilities or increasing anxiety, ”writes Deutsche Huntington-Hilfe e.V. on its website.

Dementia often occurs in the end stage

Many patients suffer from neurological disorders, for example movement disorders or psychological changes such as behavior disorders.

In addition, memory impairments, mood changes, gait and speech disorders and swallowing disorders occur. The facial expressions of the patients are also often influenced.

In the final stages of the disease, brain function is usually so severely impaired that dementia develops.

Fight the cause of the disease

The cause of the rare disease is an altered gene (gene mutation).

"Although the cause of Huntington's disease has been known for over ten years, there is still no causal therapy to this day. Only individual symptoms can be treated, ”writes Deutsche Huntington-Hilfe.

But something could change soon. Because British researchers have succeeded in the first tests to fight the cause of the disease.

New experimental drug

The University of London (UCL) team of scientists, led by Professor Sarah Tabrizi, tested a new experimental drug for Huntington's disease.

"The first drug to target the cause of HD was safe and well tolerated," said a UCL statement.

According to the experts, it successfully lowered the level of the harmful huntingtin protein in the nervous system. Mutations in this protein are believed to be responsible for the development of Huntington's chorea.

According to the release, the study included 46 patients with early Huntington's disease in nine study centers in the UK, Germany and Canada.

The course of the disease could possibly be slowed down

"The results of this study are fundamental to HD patients and their families," said Professor Tabrizi.

"For the first time, a drug lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well tolerated," said the scientist.

"The key now is to quickly move on to a larger study to test if the drug is slowing the course of the disease." (Ad)

Author and source information

Video: Help For Those With Huntingtons Disease - Nebraska Medicine (October 2021).