Spring 2002 ~ Newsletter
Hope: While Having Cystic Fibrosis
Newsletter Navigation:
Quick Navigation:
Is There Any Real Hope in CF Research Today?
by Eric Sorscher, MD
Research in Cystic Fibrosis is bringing new hope to families with the disease. If you have been diagnosed with CF in the past year, the information provided here may help explain some of the recent progresses in Cystic Fibrosis and how this will continue in 2002.
Twenty years ago, very little was understood about why patients with CF became ill, or how we might be able to overcome CF lung disease. The average life expectancy was about 20 years. Since that time, cystic fibrosis scientists have learned that the basic problem in the disease involves a chloride channel protein that is not working properly in the lungs of patients with cystic fibrosis. New scientific discoveries such as decoding the genetic information of pseudomonas (an important bacteria that infects the lungs of patients with cystic fibrosis), the identification of the genes that lessen the severity of cystic fibrosis, and the establishment of new therapies that improve the health and well being of CF patients (inhaled Tobramycin and Pulmozyme, for example), together with improvements in other CF therapies have led to better and longer lives for people with cystic fibrosis. At the national level, hundreds of thousands of compounds are being screened to discover new medicines that correct the most common mutation associated with CF (ΔF508 CFTR). Approaches to decrease inflammation in the lungs of patients with CF are being tested at centers across the country. The Cystic Fibrosis Foundation has mobilized a network of care centers, including UAB, to move discoveries as quickly as possible into the clinical testing phase. The results of some of these clinical trials will be available in the coming year.
As part of this newsletter, you may be aware of the ways UAB has contributed to CF research. The University of Alabama at Birmingham is home to the first center designated for research in cystic fibrosis in the United States. Many important findings concerning the disease were made in Birmingham. At UAB, our research emphasizes the development of new cystic fibrosis treatments. In the past year, we have published the results of three clinical trials regarding experimental CF therapies, with a fourth trial nearing completion. Dr. Chris Makris (Department of Pediatrics) and Valerie Eubanks M.S., R.D. (CF Research Program Coordinator) will publish a paper in the Journal of Pediatrics in the next few weeks describing ways in which megestrol acetate can improve weight gain in CF and also stabilize lung function. It is too early to be certain where this drug will be useful as a long-term treatment. However, the results are encouraging and suggest the need for a national trial to order to specify both the safety and the effectiveness of this drug therapy.