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Breakthrough drug for Huntington’s may have implication for Alzheimer’s and other diseases

January 2018

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The development of a new drug that stops Huntington’s disease is now being talked about as one of the biggest medical breakthroughs in half a century.

This is not just for its amazing potential for sufferers of Huntington’s disease but because there is also potential for it to be developed further into personalised treatments for a range of patients with severe genetic diseases.

Huntington’s is an incurable degenerative disease caused by a defect in a single gene that is passed down through families. The first symptoms don’t usually appear until middle age but there is a gradual onset of uncontrolled jerky movements, dementia and ultimately paralysis. About 10,000 people in the UK have the condition and around 25,000 are at risk. Patients usually die within 20 years after the onset of symptoms.

The drug, called Ionis-HTTRx, was developed by the California biotech company Ionis Pharmaceuticals and is based on its ability to intercept and destroy a messenger molecule that spreads the instructions from the faulty Huntington gene. The drug intercepts this messenger molecule and destroys it before it can instruct a toxic protein to be made, effectively silencing the effects of the mutant gene.

Following development of the drug, it was then put into a limited trial co-ordinated by University College London’s Huntington’s Disease Centre. 46 men and women in UK, Germany and Canada were involved in the trials, all suffering from early stage Huntington’s disease. They were each given four spinal injections one month apart and the drug does was increased at each session. Around a quarter of the participants had a placebo instead.

After being given the drug, the concentration of the harmful protein in the spinal cord fluid had dropped significantly, and in precise proportion to the amount of the drug given. This was so closely matched that the researchers were confident the drug was having a powerful effect. There appears to be no major side effects from the drug.

One of the lead researchers in the trials was Professor Sarah Tabrizi, head of University College London’s Huntington’s Disease Centre. She said the results of the drug were probably the most significant moment in the history of Huntington’s since the faulty gene was isolated.

The Swiss pharmaceutical giant Roche has paid a $45m licence fee to take the drug forward now to clinical use and there is now excitement in other areas of the scientific world looking at the potential to adapt the drug to target other incurable brain disorders such as Alzheimer’s and Parkinson’s.



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