An innovative type of medicine – called gene silencing – is set to be used on the NHS for people who live in crippling pain. The drug treats acute intermittent porphyria, which runs in families and can leave people unable to work or have a normal life. Clinical trials have shown severe symptoms were cut by 74% with the drug. While porphyria is rare, experts say the field of gene silencing has the potential to revolutionise medicine.
Prof David Rees, the director of the King’s College Hospital National Acute Porphyria Service, told the BBC: “To find a drug that really does transform people’s lives is extraordinary.” However, acute intermittent porphyria is rare. Only around 17 people are diagnosed in the UK each year. “[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,” Prof Rees said. Gene-silencing has already proven effective in other rare genetic diseases such as amyloidosis. Its ability to tweak how DNA works in the human body, without permanently altering it, has already seen it used as a twice-a-year cholesterol busting jab. Tara Moore, a professor of personalised medicine at the University of Ulster, said gene silencing had the potential to be as big as antibiotics. She told BBC Radio 4’s Inside Health: “It will be, it’s a very powerful tool, it is so specific, it’s really phenomenal. “There’s really nothing to stop us targeting so many different diseases from cancer to cardiovascular disease to cholesterol problems.”https://www.bbc.com/news/health-58988006