Drug brings new hope for cystic fibrosis victims

It is hoped the treatment can be provided quickly. Picture: Getty
It is hoped the treatment can be provided quickly. Picture: Getty
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SCOTTISH researchers believe a new drug could ease the symptoms of people with cystic fibrosis, one of the UK’s most common life-threatening inherited diseases.

Lynovex, produced by Aberdeen-based biotechnology company Novabiotics, controls the bacteria and fungi causing lung infections.

“We know it’s safe, we need to prove its effectiveness”

Dr Douglas Fraser-Pitt

Cystic fibrosis is caused by a single defective gene and affects more than 10,000 across the UK and one child in every 2,500 is born with the condition.

Sufferers’ internal organs, especially the lungs and digestive system, become clogged with thick sticky mucus, resulting in chronic infections and inflammation in the lungs and difficulty digesting food.

Those affected often die shortly after the age of 40 as mucus from the disease damages their lungs, risking infection.

Lynovex is one of a number of new drugs being developed to tackle the condition. It works by breaking down the sticky mucus in patients’ lungs.

The results of phase II clinical trials (to test effectiveness, as part of a 0 to III range) were presented yesterday in Brussels at the annual conference of the European Cystic Fibrosis Society.

The active component of the new medicine is Cysteamine, a drug used for 20 years to treat an unrelated rare condition called cystinosis, which leads to kidney, thyroid, eye and liver problems and impairs growth. The fact the drug is already approved for clinical use means it could be quickly approved for people with cystic fibrosis.

Novabiotics concentrates on developing drugs for rare or difficult-to-treat conditions.

Last month, it was announced that a new combination drug had been developed which stops the genetic disorder damaging the lungs. But it is not yet a 100 per cent cure and there remains a critical need for more effective, safe, long-term 

Dr Douglas Fraser-Pitt, principal scientist for Novabiotics, said: “We know it’s safe. Now we just need to prove its effectiveness in this group of patients in further trials. It has the potential to make a big difference to care.

“It is also a re-purposed drug so there is lots of potential for this drug to reach everyone who needs it quickly.”

Janet Allen, director of research at the Cystic Fibrosis Trust, said: “Co-funded by the trust, the promising results of this trial move us a step closer to the first drug specifically aimed at targeting the pulmonary infections associated with cystic fibrosis.

“Further studies are required to confirm these preliminary findings and if the results are positive, we hope that because the drug is already used to treat another rare genetic condition, cystinosis, Lynovex will be made available to people with cystic fibrosis more quickly compared to if this were a completely new drug.”