New Hope for Cystic Fibrosis Patients

Author:  Shrestha Suvash
Date:  September 2008

Great relief is what Cystic Fibrosis (CF) patients must have felt when; Dr David Sheppard from the University of Bristol, UK presented his works on CF treatment at the BA festival of science (an annual international conference organized by the British Association for the advancement of science) in Liverpool on 9th of September 2008. The doctors seem to have moved a step closer to the effective treatment.

Today, there are around 70,000 persons with CF in the world. It is a single gene disorder that destroys the structure of the protein called CFTR. Thus all the organs involving this protein - lungs, liver, pancreas, intestines and reproductive organs - do not function properly. The ducts in these organs are blocked with thick mucus, as they cannot simply brush out their secretions. A very common symptom is salty sweat as the ducts fail to reabsorb salt.

The sad part of the story is that CF has no treatment at all. Whatever treatment options we have are all just symptomatic e.g. physiotherapy to clear airways, antibiotics for lung infections, enzymes to digest food. However, Dr David Sheppard's research group, supported by Cystic Fibrosis Trust, has been studying how new drugs can restore the function of defective CFTR.

"The early results with VX-770 (the drug produced by Vertex Pharmaceuticals) suggest that drug therapies which target defects at the root of the disease have the potential to improve greatly the quality of life of CF patients," said Sheppard. VX-770 has already been tested in CF patients in the US. The early results are very encouraging. Using 150 mg of VX-770 twice a day reduced the amount of salt in sweat by half and improved the lung capacity by 10%.

This discovery is significant. CF is one of the most common life shortening diseases with a current life expectancy of only 30 – 40 years even with the best available treatment facilities. 1 in 25 people in the UK is a carrier of the defective CF gene, which means that there is a huge chance for babies to be born with this disorder if they inherit a copy of the defective CF gene from each parent. This makes it all the more important to get an effective treatment for CF.

Written by: Suvash Shrestha

Edited by: Nadia Ramlagan

Published by: Hoi See Tsao