Fall 2002 ~ Newsletter
Hope: A National Perspective
Newsletter Navigation:
Quick Navigation:
Cystic Fibrosis Research at the Children's Hospital of Alabama and UAB
Children's Hospital and UAB are both considered to be international leaders in the study of Cystic Fibrosis, due in large part to the Gregory Fleming James Cystic Fibrosis Research Center (CFRC). This research center was established in 1981, and brings approximately 50 researchers on campus together to study CF. The main goals of this center are to better understand the underlying causes of CF, and to test new therapies to treat the disease. Clinical research in CF at Children's Hospital and UAB has engendered some exciting recent results, which are highlighted below:
- In 1993, the UAB CFRC became the 1st site in the country to receive approval to evaluate lipid-based gene therapy in CF patients. The 1st CF patient receiving a working CF gene by this method was in 1994. Since that time, UAB and Children's Hospital were the only centers in the United States to test this method of gene therapy in the lungs of CF patients (where most CF symptoms occur). The results of this study were published in a scientific journal in 2001, and demonstrated that a working CF gene could persist in the lungs of a CF patient for up to 7 days. An inflammatory (flu-like) illness including muscle and joint aches, and short-term fever was noted in some study subjects, and will need to be overcome to advance this new technology. The results of this trial have important implications for many avenues of gene therapy, and are an important step in moving gene therapy from the bench to the bedside.
- In 2001, the UAB CFRC and Children's Hospital published an existing study testing a medication to help certain defective CF genes in CF patients. This medication, Gentamicin, is a commonly used antibiotic that has the additional ability to help certain types of mutant CF genes (called premature stop codon mutations) work better. The results sparked quite a bit of interest in the CF community, and have led to the development of a similar trial at multiple CF centers across the country. This study is headed by Dr. J.P. Clancy and Dr. David Bedwell at UAB. Recent scientific editorials in prominent medical journals including The Journal of the American Medical Association, The Lancet, and The Scientist have highlighted this emerging CF therapeutic strategy.
- In 2002, UAB and Children's Hospital published their results from a trial testing the drug megestrol acetate, commonly referred to as Megace ® in malnourished CF patients. Malnutrition is a very important problem facing CF patients, and predicts a shorter lifespan in those patients that are affected. Megestrol acetate is a steroid type of drug that has many effects, including stimulation of appetite and reduction in inflammation. The study tested the effects of megestrol acetate on weight, growth, and lung function in malnourished CF patients. Patients treated with Megace ® normalized their weight within 3 months, and had improvements in lung function over the entire 6 month treatment period compared wit the placebo-treated controls. The results were published in the Journal of Pediatrics in April 2002, with an accompanying editorial.
These studies describe exciting CF research recently completed or in progress through the UAB CFRC and at Children's Hospital. Several other research projects are in progress at Children's Hospital and UAB, and we are thankful to the many volunteers who have participated in these trials. We are also grateful to the CF Foundation and NIH for providing the funding for these studies. We hope that these and other therapies under investigation here at UAB may one day improve the lives of CF patients, both in the state of Alabama and throughout the world.