About 30,000 Americans suffer from cystic fibrosis, the nation's most common fatal inherited disease. Approximately 12 million Americans - about one in 20 - are carriers of the gene that causes cystic fibrosis, meaning that while they don't have the disease, their children could.
Yesterday's gene therapy treatment occurred about 1:30 p.m. at the Hospital of the University of Pennsylvania. A team of Penn physicians and scientists, headed by Dr. James M. Wilson, dripped about two teaspoons of fluid containing a genetically altered cold virus into the woman's left lung.
In the coming months, 19 other patients are expected to undergo similar treatments. Ten groups, containing two people each, will receive slightly elevated doses of the genetic material as the protocol proceeds.
"The goal of this study is to evaluate the feasibility and safety of this important technology," said Wilson, director of Penn's Institute for Human Gene Therapy. "Patients participating in this protocol will not directly benefit from the treatment because the corrective genes are exposed to a small portion of the lung. It is hoped the information gained from this protocol will be useful in designing a second-phase protocol which is therapeutic."
Over the next year, Wilson and his colleagues will check to see if the gene was actually delivered to the cells of the cystic fibrosis patients, if the gene switched on once it entered the cells and if the genes begin to reverse the buildup of mucus that characterizes cystic fibrosis. They will also closely monitor the health of the patients.
The experimental procedure is not risk free. Some scientists fear that attempts to fix genetic defects could trigger other serious medical problems - cancer, for instance.
People with cystic fibrosis lack a gene that controls the proper flow of
