Spring 2002 ~ Newsletter

Hope: While Having Cystic Fibrosis

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Where Is Hope When Having CF?

by Raymond Lyrene, MD

Hope is the expectation that events will turn out for the best.  We all need hope to help through life’s difficult and discouraging times.  Hope is especially important in a chronic progressive disease such as Cystic Fibrosis were discouragement and defeat seem all too common.  On the other hand, false hope cane be destructive and create distrust since it paints an unrealistic picture of the future. 

Is there reason for hope for those with Cystic Fibrosis?  The answer is an emphatic yes!  The journey toward better treatment and event a cure is a long one, but we can definitely see progress.  Median survival of individuals with Cystic Fibrosis has increased 10 years in the past 30 years and is now over 32 years.  What has made the difference?  The answer is probably improvements in the many types of therapies and not just a single advance.  Some of the changes that come to mind are better nutrition and airway clearance and more effective use of antibiotics including aerosolized antibiotics.  The use of anti-inflammatory medications such as high dose ibuprofen may also play a role in slowing the progression of lung injury.  Some states have begun neonatal screening for Cystic Fibrosis.  Early diagnosis of CF also helps to improve nutrional status that may ultimately lead to a longer life.

Furthermore, there are thousands of research scientists around the world who are studying Cystic Fibrosis.  These scientists are helping use to understand more about Cystic Fibrosis.  The greater our understanding the more likely it will be for us to develop effective treatments.  We Alabamians are fortunate to have one of the best Cystic Fibrosis research centers right here in Birmingham.

Dr. David Bedwell (Department of Microbiology) has devised an intervention for patients with a rare form of Cystic Fibrosis (i.e., CFTR with an “X” mutation such as G542X, W1282X, or R552X).  Approximately 5 – 10 % of patients with Cystic Fibrosis have this form of the disease, which causes a shortened version of the CFTR protein that does not function properly.  Working together with Dr. J.P. Clancy (Department of Pediatrics), investigators at UAB have show than aminoglycoside antibiotics partially correct these forms of CF. The same conclusion was reached by investigators in Israel (where CFTR “X” mutations occur in nearly 90% of the Cystic Fibrosis patients) and in a mouse model of cystic fibrosis developed at UAB.  Investigators at the university of Pennsylvania showed that the same strategy could be used to treat of Duschenne’s Muscular Dystrophy in a mouse model.  Dr. Bedwell helped arrange a screen of nearly 800,000 compounds that identified several new drugs with promise in correcting this type of CF defect.  A multi-center trial is planned for the coming you with UAB as a lead site.  Dr. Bedwell’s approach has recently been reviewed in editorials in the Journal of the American Medical Association, The Lancet, and The Scientist.

The UAB CF Research program also has been at the foremost of gene therapy approaches for Cystic Fibrosis.  UAB recently published the first trial in the United States to investigate liposomes for correcting CF defects in the lower airways of CF patients.  We have also been selected as a site for a CFTR gene transfer trial using an adeno-associated virus to carry genetic corrections in to the lungs of CF patients.

These sorts of clinical results, together with research efforts at UAB and throughout the nation, will help us better understand the reasons patients with cystic fibrosis develop lung disease, and the ways these problems can be overcome.  The progress provides a reason for hope in the coming year.  With continued hard work and dedication, scientists and clinicians are determined to improve the health and well being of patients with CF.

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