It is the first time a drug trial has slowed the rare disorder’s progression in an ongoing, significant way, researchers said.
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Doctors say Huntington’s disease, a fatal hereditary disorder that typically strikes in middle age, has been successfully treated for the first time as part of a small trial.
Huntington’s causes neurons, or nerve cells in the brain, to break down and die, leading to physical and cognitive decline. The rare disorder is caused by a gene mutation that orders cells to make a toxic version of brain proteins.
The experimental treatment aims to reduce levels of these toxic proteins using a single dose of a powerful gene therapy injected straight into the brain.
In a study involving 29 people, a high dose of the drug slowed the progression of Huntington's disease by 75 per cent over three years, according to the company uniQure, which makes the treatment.
It is the first time a drug trial has slowed Hungtingon’s progression in an ongoing, significant way, the researchers said.
The company reported the results of the trial in a press release, but they have not yet been reviewed by experts or published in a medical journal.
Independent researchers were cautiously optimistic about the results, saying they represented a potential major breakthrough but that larger studies with more people will be needed to confirm that the drug works well.
“This is incredible news for everyone affected by Huntington’s, a cruel and devastating disease,” Siddharthan Chandran, Director of the UK Dementia Research Institute, said in a statement.
“We’ll need to wait for the full peer-reviewed results to come out, but this is promising and gives real hope to families dealing with this disease,” Chandran added.
Huntington’s symptoms, which typically appear in middle age, include behaviour changes, cognitive decline, and involuntary movements that can make it harder to walk. Some patients also develop a tremor, similar to Parkinson’s disease.
The disease is normally fatal within 20 years, but people tend to become disabled earlier on. That means earlier treatment could improve patients’ quality of life for many years.
About 75,000 people have Huntington’s disease in the European Union, the United Kingdom, and the United States, according to uniQure.
However, other experts cautioned that previous attempts to treat Huntington’s have also appeared promising – only to fail later on.
“The results are encouraging but it is early days and we have been here before with other similar therapies for Huntington’s disease,” said Roger Barker, a professor of clinical neuroscience at the University of Cambridge.
