A new drug may become a treatment for cystic fibrosis. If proven to be effective, CPX, exclusively licensed to SciClone Pharmaceuticals, Inc., of San Mateo, Ca., could "correct" the basic molecular defect that causes CF. The Food and Drug Administration (FDA) recently gave permission to the company to conduct Phase I and Phase II clinical trials directly with cystic fibrosis patients. This allows SciClone to bypass the usual process of conducting trials on healthy volunteers, saving about a year's worth of development time. SciClone has already begun animal toxicity studies of CPX.
Cystic fibrosis is caused by mutations in the CFTR gene, preventing the transport of chloride and sodium across epithelial membranes in cells. The promise of CPX is that it may correct the root cause of CF-not the symptoms-by allowing the CFTR to perform properly its chloride secretion function. CPX was discovered by Harvey Pollard, M.D., Ph.D., and Kenneth Jacobson, Ph.D., of the National Institute of Diabetes and Digestive and Kidney Disorders of the NIH. Pollard and Jacobson discovered in an in vitro study that CPX was able to stimulate chloride to move out of the defective CF cells. Based on these results, the doctors are very hopeful that CPX may stimulate the CFTR to do its job properly in those CF patients with the Delta-F508 mutant CFTR as long as CPX is taken.
The aims of the Phase I/II trials are to determine safety parameters and efficacy of CPX. The studies will begin soon at approximately five CF centers throughout the country.
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