Beating the cystic fibrosis barrier

Cystic fibrosis is a hereditary disease that kills thousands each year because it is difficult to treat, but scientist in America have synthesised nanosized particles able to deliver medication across the mucus barrier characteristic of the disease.

Cystic fibrosis transmembrane conductance regulator (CFTR) – disruption in CFTR causes cystic fibrosisBiodegradable nanoparticles capable of penetrating the mucus barrier in the lungs and gut of cystic fibrosis (CF) sufferers have been developed by researchers at John Hopkins University, led by Justin Hanes, professor of chemical and biomolecular engineering.

“Cystic fibrosis mucus is notoriously thick and sticky, and represents a huge barrier to aerosolised drug delivery,” said Pamela Zeitlin, CF expert and professor of paediatrics, who collaborated on the study.  “Nanoparticles were engineered to travel through cystic fibrosis mucus at a much greater velocity than ever before, thereby improving drug delivery,” she said.

The nanoparticles comprise of two parts made of molecules regularly used in existing medications.  An inner core of polysebacic acid (PSA) traps therapeutic agents inside while a dense outer coating of polyethylene glycol (PEG) molecules allows the particle to move through the mucus by preventing it from reacting with proteins.

Hanes previously demonstrated that latex coated with PEG could slip past mucus coatings, but because the body could not break it down into harmless components it was impractical. “The major advance here is that we were able to make biodegradable nanoparticles that can rapidly penetrate thick and sticky mucus secretions and that these particles can transport a wide range of therapeutic molecules,” said Hanes.

The nanoparticles have potential applications treating lung and cervical cancers, and inflammation of the sinuses, eyes, lungs and gastrointestinal tract where mucus is produced to protect sensitive areas, said Benjamin C Tang, lead author and postdoctoral fellow in the department of chemical and biomolecular engineering.  Zeitlin concludes: “This work is critically important to moving forward with the next generation of small molecule and gene-based therapies.”

3 Responses to Beating the cystic fibrosis barrier

  1. Lovelyn Allen says:

    I have two grandchildren with cf 6yrs and 2yrs.the oldest a grandson has just had a bout of bowel obstruction. he did not need surgery and he is also celiac.he takes for his bowel a power called PEG(polyethylene glycol).They showed it to me on a visit this past Wednesday.I myself at 65 was diagnosed with cf.All my life i’ve had problems with chronic constipation-bowel obstructions-chronic pancreasitis and lung infections at least now i understand why.this is very interesting.thanks for the info.i will tell my doctor about the website

  2. Audrey Gothard says:

    This is great news. We have a granddaughter in her 20′s with cystic Fibrose and pray this will mean a longer healthy life mfor her..Thank you.

  3. Sara Hummel says:

    I am the sister of the Granddaughter that Audrey mentioned, and all I can say is that I am elated by this news. I am always on the lookout for postings regarding new treatments and/or medications, and this appears to be the most promising that I’ve read about. I want to thank all the dedicated people who spend their lives researching this disease, and who work on developing new treatments. You provide hope to friends, families, and patients with CF.

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