Scientists at Queen’s University Belfast have discovered a new molecule which has the potential to prolong the life of individuals with cystic fibrosis (CF).
The molecule represents a possible future treatment and works by altering cellular ion channels resulting in improved airway hydration and significantly increased mucus clearance.
For individuals with CF, normal airway clearance mechanisms which keep the lungs free of infection are defective. As a result, a build-up of mucus occurs and predisposes the individual to chronic bacterial infection. The ensuing cycles of chronic infection and airway inflammation cause progressive destruction of the airways, which is ultimately fatal.
The innovative approach could reduce the frequency of these infections through a novel protease inhibitor which prevents activation of the epithelial sodium channel; ENaC.
The research involved a team of scientists from Queen’s University alongside colleagues at the Royal College of Surgeons in Ireland and the University of North Carolina, and was funded by The Cystic Fibrosis Trust.
Speaking about the breakthrough, Dr Lorraine Martin from the School of Pharmacy at Queen’s University Belfast, said: “This is an important finding which could provide a novel therapeutic opportunity relevant to all individuals with CF, as the targeting of ENaC is independent of their underlying CF mutation. This strategy could prevent the significant lung damage that results from chronic cycles of infection and inflammation, with potential impact on quality of life as well as life expectancy. This is a further example of Queen’s University’s research advancing knowledge and changing lives.”
Ed Owen, Chief Executive at the Cystic Fibrosis Trust, said: “We are thrilled with these initial findings and are excited to see how the next stage of preclinical testing progresses. We are pleased to have been able to fund this world class project at its early stage and welcome the drug development programme planned over the coming years. Research is the biggest single area of investment for the Cystic Fibrosis Trust and it’s wonderful to see projects like this making such positive progress in our fight for a life unlimited.”
Currently available pharmacological alternatives are only suitable for a small subset of patients depending on the disease-causing genetic mutation.
Learn more: QUEEN’S SCIENTISTS DEVELOP NEW TREATMENT TO PROLONG LIFE OF THOSE WITH CYSTIC FIBROSIS
The Latest on: Cystic Fibrosis
[google_news title=”” keyword=”Cystic Fibrosis” num_posts=”10″ blurb_length=”0″ show_thumb=”left”]
via Google News
The Latest on: Cystic Fibrosis
- $100B Vertex Pharma Working To Recreate $10B Cystic Fibrosis Success - This Time Focused On Pain Management Drugson April 26, 2024 at 11:34 am
Vertex Pharmaceuticals Inc‘s (NASDAQ:VRTX) blockbuster drug Trikafta for cystic fibrosis (CF) has shown remarkable efficacy, potentially extending the lives of some patients by up to 45 years. Despite ...
- Vertex Announces European Commission Approval for KALYDECO® to Treat Infants With Cystic Fibrosis Ages 1 Month and Olderon April 26, 2024 at 8:43 am
Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Commission has granted approval for the label expansion of KALYDECO® (ivacaftor) for the treatment of infants down to 1 month of ...
- Its blockbuster cystic fibrosis treatment costs $300,000 a year. Now Vertex wants to solve the opioid crisison April 26, 2024 at 2:00 am
The number of people living with CF worldwide is now 105,000, up from 70,000 in 2012, according to estimates from US non-profit the Cystic Fibrosis Foundation — a rise that experts attribute both to ...
- Woman with cystic fibrosis can’t believe she managed to run 26.2 mileson April 23, 2024 at 11:05 pm
A WOMAN with cystic fibrosis ran the London Marathon thanks to “miracle” drugs. Nell Summerley, 21, from Britwell Salome, took part in Sunday’s race to raise money for the Cystic Fibrosis Trust. She ...
- Vertex has top orphan asset in the pipeline, as pharma eyes shiny obesity drugs: reporton April 23, 2024 at 9:00 pm
Vertex’s cystic fibrosis triple combo is poised to become the top selling orphan drug in terms of net present value, amid shifting sentiments in the pharma industry for rare disease drugs, according ...
- Personal trainer born with cystic fibrosis on mission to run 36 marathons in 36 dayson April 23, 2024 at 7:01 am
Sophie Holmes, from Southend, was told she would not live to be 16 years old after the life-changing diagnosis, but has repeatedly beaten the odds ...
- Arcturus Therapeutics' ARCT-032: Might Be The Next Breakthrough In Cystic Fibrosison April 23, 2024 at 3:01 am
Arcturus Therapeutics is on the verge of obtaining regulatory approvals for two gene therapies targeting rare diseases. See why ARCT stock is a Buy.
- Southend woman with cystic fibrosis to make world record marathon attempton April 20, 2024 at 10:03 pm
A woman with cystic fibrosis is is lacing up to set a new world record by running 36 marathons in 36 days. Sophie Holmes, from Southend-on-Sea, was told her cystic fibrosis (CF) meant she would not ...
- Study supports use of cystic fibrosis drug in infants from four weeks of ageon April 11, 2024 at 8:32 am
A cystic fibrosis drug targeting the basic defect that causes the condition has been shown to be safe and effective in newborns aged four weeks and above, new research involving RCSI University of ...
- Wexford mother shines a light on the incredible work of Cystic Fibrosis Irelandon April 11, 2024 at 4:59 am
Wexford people are being urged to support Cystic Fibrosis Ireland to help it meet its fundraising target of €300,000 ahead of 65 Roses Day on Friday, April 12.
via Bing News