Patients in Scotland are set to be the first to test a new drug to treat the devastating effects of cystic fibrosis. Research company NovaBiotics, based in Aberdeen, has been developing a treatment known as Lynovex for the past 18 months to tackle the symptoms of the disease and also keep patients healthier for longer. They now hope to start testing the drug on patients this year, with those in Scotland set to benefit first.
The Cystic Fibrosis Trust said it looked forward to seeing the results of the trial. More than 9,000 people across the UK are affected by CF. The hereditary disorder affects the internal organs, especially the lungs and digestive system, by clogging them with thick sticky mucus, making it hard to breathe and digest food. Only half of those living with cystic fibrosis are likely to live past their late thirties.
The new treatment tackles the symptoms of CF, while also reducing the chances of more damage being caused to the lungs. This could offer patients needing a lung transplant valuable extra time in their wait for organs to become available.
Deborah O'Neil, founder of NovaBiotics, said during their research they realised that the treatment would be good at breaking down what are known as bacterial biofilms - the slimy infections which form in people with CF to try to protect themselves against the immune system and antibiotics. "It seemed to have this property. But we went further and realised that it didn't just break down biofilms but broke down mucus," she said.
"It also had this extra property that it was antibiotic in its own right as well, so not just killing bacteria because it breaks down these biofilm structures, but it actually killed the bacteria directly." The researchers also found that the drug worked with the antibiotics currently used for CF patients and discovered that in combination it made these more powerful.
Dr O'Neil said: "This could mean that the drugs would be used less, reducing the chances of them becoming resistant more quickly. The evidence suggested that with Lynovex you can reduce the amount of antibiotic and that it delivered this effect which means that dosing could go for much longer intervals and you would have to dose less frequently."
So far the drug - which would be taken from a nebuliser or inhaler - has only been tested in the laboratory, but that is set to change this year with the start of patient trials.
Getting the drug ready for use in patients could also be speeded up as the treatment has already been used in another condition - a rare metabolic disease known as cystinosis - meaning many safety tests have already been carried out.
Between 20 and 40 patients are set to take part in first stage of trial. Dr O'Neil said the drug was also expected to be very cheap - maybe only a few hundred pounds, or even less, per patient each year.
Matthew Reed, chief executive of the Cystic Fibrosis Trust, said: "Any new drug that has the potential to significantly improve the lives of people with CF, helping them to live longer and have a better quality of life, is to be welcomed.
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NovaBiotics Initiates Development of Orphan Drug in Cystic Fibrosis
17th December 2013
Pharmacells to expand following funding boost
24 September 2013
NovaBiotics Enters into Agreement with Taro
28 August 2012
Pharmacells in talks to extend reach overseas
24 January 2012