A gene therapy developed by uniQure has continued to slow the progression of Huntington’s disease four years into treatment, though the scale of the benefit appears smaller than it did a year ago, according to new findings shared by medical news outlet STAT News.
Huntington’s disease is an inherited condition that damages nerve cells in the brain, gradually affecting movement, thinking, and behaviour. There is no cure. The uniQure therapy — a treatment that works by altering genetic instructions inside cells — has been under close watch as one of a small number of approaches targeting the disease at its biological root.
The four-year data point is a meaningful marker. Gene therapies are still relatively new, and long-term follow-up data of this kind helps researchers understand whether early gains hold, fade, or shift over time.
But the finding that the magnitude of benefit has waned since a similar analysis a year ago raises questions the research community will be watching closely. The four-year result — showing a 44% slowing of progression in high-dose patients compared with external controls — did not reach statistical significance on the composite disease-progression scale used as the primary measure, though a separate measure of functional capacity showed a statistically significant 61% slower decline. Whether that represents a natural plateau in effect, a gradual loss of therapeutic activity, or something else in the data is not yet clear from the information available.
STAT News, which covers medical research and the pharmaceutical industry, reported the findings without providing a full breakdown of the underlying figures in the post.
Huntington’s disease affects around 8,500 people in the UK, according to the NHS, with a further 25,000 at risk of inheriting the condition. It typically develops in mid-adult life and is caused by a faulty gene passed down through families.
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Anyone concerned about symptoms of Huntington’s disease, or with a family history of the condition, should speak to their GP in the first instance. The Huntington’s Disease Association helpline can be reached on 0151 331 5444. For general health concerns, NHS 111 is available by phone or online.
Source: @statnews