26
Oct 2021
Gene silencing medicine transforms debilitating pain
An innovative type of medicine, known as gene silencing, is due to be used through the NHS for people who live with crippling pain.
The drug treats acute intermittent porphyria, which runs in families and can leave people unable to work or lead a normal life.
Clinical trials have shown severe symptoms were reduced by 74 per cent with the drug.
Gene silencing gets to the root-cause of the disease rather than just managing the symptoms. Porphyria leads to a build-up of toxic proteins in the body, that cause the physical pain. The gene silencing “mutes” a set of genetic instructions to block that protein production.
The National Institute for Health and Clinical Excellence, which approves drugs for use in England, said the therapy was “value for money” and “would improve people’s quality of life”.
Professor David Rees, director of the King’s College Hospital National Acute Porphyria Service, said “To find a drug that really does transform people’s lives is extraordinary.”
However, the condition is rare with only around 17 people diagnosed in the UK each year.
Professor Rees went on to say “But if we can control genes and switch them on and off when we want to, then almost anything is possible in terms of treating diseases including Alzheimer’s and cancer and everything else.”
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Posted by Tony May, Partner/head of Clinical Negligence Department, Chadwick Lawrence LLP (tonymay@chadlaw.co.uk ), medical negligence lawyers and clinical negligence solicitors in Huddersfield, Leeds, Wakefield and Halifax, West Yorkshire.
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