Gene silencing drug alters crippling pain
An innovative drug called Gene Silencing – will be used for people living in crippling pain on the NHS.
The drug treats acute intermittent porphyria, which runs in families and people may be unable to work or live a normal life.
Clinical trials have shown that severe symptoms are reduced by 74% with medication.
While porphyria is rare, experts say it has the potential to revolutionize medicine in the field of gene silencing.
Sisters Liz Gill and Sue Burrell have both changed their lives by gene silence.
Prior to treatment, Liz from County Durham remembered the trauma of living in “complete pain,” and worst of all, she spent two years paralyzed in the hospital. Younger sister Sue says she suddenly lost the night when she was suddenly in and out of the hospital, became redundant and she knew her partner would stay with her (he did).
“She was scary,” she tells me.
Both became accustomed to taking powerful opioid painkillers daily. But morphine also failed to prevent pain during a severe attack that required hospital treatment.
Gene silencing was more than simply managing the symptoms of the sisters rather than making them the root cause of the disease. Their porphyria cause the body to produce toxic proteins, which cause physical pain. Gene Silencing A set of “mutes” genetic instructions blocks that protein production.
Both were taking therapy as part of a clinical trial and are still receiving monthly injections.
“The difference is astronomical, we don’t have pain anymore,” Liz said.
“You don’t have to rely on opioid-based pain relief and this leads to things like being able to succeed at a job and buy your own home.”
Su, from Norfolk, said the therapy changed her life: “[You] are able to do things you couldn’t before, being able to be a mother, being able to be a better wife … just living life.”
Clinical trials showed a gene silencing therapy called genosyrenes, reducing the number of severe attacks by 74%.
The National Institute for Health and Clinical Excellence (NICE), which approves the use of drugs in England, said the therapy would “improve people’s lives” and “value for money”.
Professor David Reese, director of the National Acute Porphyria Service at King’s College Hospital, told the BBC: “It’s really fantastic to find drugs that can change people’s lives.”
However, intermittent porphyria is rare. Only 17 people are diagnosed each year in the UK.
“[But] if we can control the genes and turn them on and off when they want to, then almost anything is possible in terms of treating diseases, including Alzheimer’s and cancer, and everything else,” said Professor Reese.
Gene-silencing has already proven effective in other rare genetic diseases such as amyloidosis. Its ability to pinch how DNA works in the human body, without permanent change, has been used twice a year as a cholesterol-busting jab.
Tara Moore, a professor of personal medicine at the University of Ulster, said gene silencing has as much potential as antibiotics.
She told BBC Radio 4’s Inside Health: “It will be, it’s a very powerful tool, it’s so special, it’s really unprecedented.
“There’s really nothing to prevent us from targeting many different diseases, from cancer to cardiovascular disease to cholesterol problems.”