Gene silencing, a novel type of treatment for acute intermittent porphyria, is going to be used on the NHS for those suffering from crippling pain.
This new drug can help treat acute intermittent porphyria, a disease that runs in families and can make people unable to work or have a normal life.
Clinical trials have shown that the medicine reduced severe symptoms by 74%.
While porphyria is uncommon, experts believe gene silencing has the ability to revolutionize medicine.
Gene silencing has changed the lives of sisters Liz Gill and Sue Burrell.
Liz, from County Durham, recalls the agony of living in “total pain” before therapy, and at her worst, she spent two years paralysed in hospital. Sue, Sue’s younger sister, claims she “lost it all overnight” when she was abruptly in and out of the hospital, thrown off, and unsure whether her spouse would stay with her (he did).
“It was scary,” she said.
Both became addicted to opioid painkillers on a daily basis. But even morphine could not stop the pain during a severe attack that required hospitalization.
Rather of only treating the sisters’ symptoms, gene silencing addresses the underlying cause of their sickness. Their porphyria causes a buildup of harmful proteins in the body, resulting in physical pain. Gene silencing “mutes” a set of genetic instructions to prevent the formation of that protein.
Both had been receiving the therapy as part of a research trial and continue to receive monthly injections.
“The difference is astronomical, we’re not in pain anymore,” Liz said.
“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.”
Sue, from Norfolk, said the therapy had transformed her life: “[You’re] able to do things that you couldn’t do before, being able to be a mother better, being able to be a wife better… to just live life.”
Clinical trials revealed that the gene-silencing drug givosiran reduced the number of severe attacks by 74%.
The UK’s National Institute for Health and Clinical Excellence (NICE), the drug approval agency, said the treatment “would improve people’s quality of life” and was “value for money”.
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