Gene Therapy for Cystic Fibrosis: New Hope

By Shlomo Maital


  CF Gene Therapy…new hope!  


 Cystic fibrosis is an awful disease, inherited genetically. It is caused by a single faulty gene. Some 90,000 people suffer from it worldwide.  Their prognosis is bleak.  Average age at death for a CF sufferer is 41.  The disease causes a painful buildup of mucous in the lungs. 

    With all we know about genes and gene splicing, why not just replace the faulty gene with a healthy one?  It turns out to be a massively difficult task. The body itself is designed to keep out foreign stuff, including new and healthy genes.   Now, a team led by Prof. Eric  Alton at London’s Imperial College is about to try.  The BBC reports that a clinical study has begun, with 60 CF sufferers being given gene therapy monthly, while 60 other CF sufferers will be given a placebo, every day for a year. In two years the results will be known – it works, or it fails.   

  How does it work? Cells are taken from the patient’s cheek.  The offending gene is cut out in the laboratory. Bacteria are introduced into the cell, that manufacture the healthy gene, lots of them.  Then the cells are breathed into the lungs with a ‘nebulizer’.  Evolution, and the lungs themselves, are the problem. Human lungs have evolved to keep out bacteria.  So the researchers have had to employ every trick in the book to keep the body’s immune system from rejecting the healthy genes.  The healthy genes have to be introduced into the body every month, because they need to keep displacing the old, sick gene that remains in the lungs.  

   The British team spent a decade working on the problem. They did it using highly cooperative science – convening all the scientists in Britain, all the doctors in Britain, and brainstorming for different ideas, tricks and approaches to get the job done.      

    “Think what the Wright Brothers did in 1903,”  Alton says.  “They flew 50 yards. We’re trying to prove the concept first time,  doing gene therapy for the first time in the world,   and   we have another product coming through… this is really difficult science… “