Prather Pediatric and Allergy Center - Ask Doctor Brent

Printer Friendly Version

Title: Exciting News for Cystis Fibrosis

Category: Child Care


A new drug may be approved by the FDA. It is called Dornase Alfa (DNaseI). It is a naturally occurring enzyme which helps to chop up DNA found in the thick, sticky mucous of cystic fibrosis patients. By reducing the thickness of this mucous the patients are less prone to recurrent pneumonia. The drug is manufactured by Genentech Inc. and is given to patients as an aerosol. It has been studied in over 900 patients over the past six months and the company is applying for a license to market it under the brand name, Pulmozyme. The US Food and Drug Administration committee is studying it and the FDA hopefully will find it safe and make it available to all patients who desire to take it within the next year or two. The study, so far showed that it gives significant short term benefit. The major concern is that it may be dangerous used over a long period of time. Since it is so new, we don't know what it will do over several years of use. Side effects include hoarseness, which seems to be transient, and a few patients have developed a rash and some swelling of the face.

Another very, very exciting breakthrough is the instilling of a gene directly into the lung of a 32 year old woman with cystic fibrosis. This was done at the University of Pennsylvania and is the first of several patients being treated in a multicentered clinical trial. The future patients will be given even higher doses of the genes. The gene is introduced into the lung by inserting the normal gene which does not have the cystic fibrosis defect into a common cold virus. If the insertion of this gene into the lungs works, then the hope is that the abnormal mucous accumulation characteristic of CF patients would stop. The optimistic hope for the long term is that this will one day become an effective and curative treatment for all CF patients. I am confident that we will see this within my lifetime and possibly within the next decade.

This is only one of many very exciting genetic cures which we are seeing so frequently today and which gives hope to anyone suffering with any chronic disease which may have been incurable in the past including cancer.