A MOTHER from Yate has called for the wider use of a ‘wonder drug’ for cystic fibrosis, in the hope that she might live to see her daughter grow up.

Following last week’s report in the Gazette on the Brennan family’s desire to see their two-year-old daughter Annabelle receive the revolutionary treatment ‘Orkambi’, fellow cystic fibrosis sufferer Helen Toghill has spoken out in support of the drug – despite it currently being ineffective to her.

The drug treats the underlying genetic mutation that causes the condition, unlike traditional medicines which treat only the symptoms – resulting in healthier lungs, fewer infections and less need for treatments.

But unfortunately the drug is not yet prescribed on the NHS, with the National Institute for Health and Care Excellence (NICE) saying there was too much uncertainty around Orkambi’s long-term impact and thus  deemed it not to be cost effective.

Ms Toghill, 41, told the Gazette that when she was born, doctors said that she would be unlikely to live past her teens, but is still fighting by taking “an eight-seater dining room table’s worth” of medication each week, taking around an hour out of her day.

Despite her genetic mutation of the condition meaning that Orkambi would have no effect on her, she said she hoped that a suitable variation might one day be produced.

As a result of her condition, Ms Toghill also suffers with rheumatoid arthritis and diabetes, and on particularly bad days requires the help her of nine-year-old daughter Amy.

“I feel extremely lucky that I have an amazingly supportive family and good friends that help me when I am really ill and need IV treatment.

“Amy does this without question and with great maturity. She is the reason I follow my daily regime, I can't imagine life without her.

“I would love to be able to repay them by winning my fight for even longer, and watch my daughter reach adulthood. Orkambi could do this for me!”

Orkambi is currently only being prescribed to people on compassionate grounds from just 25 cystic fibrosis centres, where the drug’s manufacturer, Vertex Pharmaceuticals, allows people who are seriously ill to take the drug free of charge.

“I have waited for some news like this and now I am denied the opportunity to take it,” said Ms Toghill. “It is sad and frustrating to say the least.

“I understand the constraints on the NHS, however, I would ask anyone to consider how they would feel if they had a family member or friend that was chronically ill and they were denied access to a drug that could potentially transform their health and life.

“All the CF community asks for is a chance to try. If this drug works it has proven to decrease hospital admissions and reduce the need for other medicines that we take.”