Gene silencing drug trialled at King’s transforms lives

Givosiran has transformed the lives of two King’s patients who were diagnosed with a rare genetic condition known as acute intermittent porphyria

David Rees

A gene silencing drug, named givosiran, has transformed the lives of two King’s patients who were diagnosed with a rare genetic condition known as acute intermittent porphyria.

Porphyria, which is inherited, leads to a build-up of toxic chemicals in the body that causes severe physical pain and can affect a person’s ability to lead a normal life.

Clinical trials, that took place at King’s College Hospital, showed that symptoms of severe pain, paralysis and anxiety were reduced by 74% after givosrian was administered. King’s College Hospital is the lead hospital for the national acute porphyria service in England, looking after more than 300 patients with the condition.

Sisters Liz Gill and Sue Burrell, who are both treated at King’s with givrosiran, explain that the drug has now transformed their lives. Liz says: "The difference is astronomical, we're not in pain anymore.

“You're not dependent on opiate-based pain relief and that leads to things like being able to succeed in a job and being able to buy your own home."

Before the trial, the sisters were relying on potent opioid painkillers and following severe “attacks,” would often require hospital treatment. Liz recalls the trauma of living in “total pain” and spending two years paralysed in hospital. Her younger sister Sue says the condition affected her work and personal life.

Professor of Haematology David Rees, who led the trial at King’s College Hospital’s national acute porphyria service, says: "To find a drug that really does transform people's lives is extraordinary.

“Gene silencing switches off the pathway which generates all the toxic chemicals and helps prevent the cause of the symptoms, rather than just using painkillers. Six patients took part in the trial at King’s College Hospital, and all of them benefitted very significantly from taking givosiran.”