Hopes high of breakthrough for fibrosis sufferers

Dawn Braham
Dawn Braham

GROUND-BREAKING new treatment for cystic fibrosis sufferers is a step closer after an “agreement in principle” to fund a trial study.

As previously reported in the Mail, the lives of thousands of cystic fibrosis sufferers could be improved dramatically after specialists made huge progress with their Gene Therapy Trial.

The trial focuses on a healthy gene being inserted into the lungs of a Cystic Fibrosis patient to ensure the damage caused by the faulty gene is stopped in its tracks.

Due to the condition, which affects the internal organs by clogging them with thick sticky mucus, more than half of sufferers have a life expectancy of just 38 years.

But this new treatment is expected to extend lives by decades.

After the delight of discovering the potential new treatment, medics became unstuck. They needed to find £6m by the end of this month to fund the start of testing new products on patients.

Residents in Hartlepool joined people throughout the country in organising fundraisers with town events boosting the bid by thousands of pounds.

Despite the tremendous efforts of the community in raising £1.6m, the target was still way off.

But the trust received a timely festive boost just weeks short of their fundraising deadline when an agreement for the treatment to be funded by the Efficacy and Mechanism Evaluation (EME) programme was made “in principal.”

A final decision isn’t expected until the new year, but trust bosses and patients have spoke of their delight.

Dawn Braham, 41, a cystic fibrosis sufferer who lives in Greatham, described the announcement as “really good news.”

The part-time mobile beauty therapist who is mum to Daniel, 22, said: “Hopefully now the therapy will be successful.

“There is no absolute guarantee that it will be, but is definitely going to help a lot of people.

“It isn’t a cure as such but what it will do is stop the lungs of sufferers from getting any worse.”

Tamsyn Clark, director of marketing at the CF Trust, said: “The CF Trust has heard that the EME programme has made an “in principle” decision to fund part of the Gene Therapy Clinical study.

“The final decision will be made when the changes to the project are agreed with the UK CF Gene Therapy Consortium.

“This is very encouraging news, the CF Trust has been overwhelmed by the support of the community in helping to fundraise the money needed to help this trial go ahead.

“We hope that in the new year a final decision will be made.”