Ground-breaking gene therapy experiment beginning at HUP

Researchers at the Hospital of the University of Pennsylvania began cutting-edge gene therapy treatment on a 32-year old woman with cystic fibrosis last Friday. This is the first use of human gene therapy in the Philadelphia area. The woman, who is from New Jersey, is only the fourth person with cystic fibrosis to undergo gene therapy in the United States. "My patient and the ones who follow are remarkable pioneers who have committed themselves wholeheartedly to helping us find a cure for this deadly disease so that others may have normal lifespans," James Wilson, leader of the team of University physicians and scientists who conducted the treatment, said in a statement. In the first stage of this cystic fibrosis gene therapy, scientists alter a common cold virus so that it cannot reproduce. A healthy copy of the cystic fibrosis gene is then inserted into the virus. Scientists then deliver the virus to the lungs by way of a tube that extends into the patient's airways. The virus, carrying the healthy cystic fibrosis gene, invades cells lining the airways of the lungs. Once in place, the gene directs the cell to function normally. During the next year, Wilson, director of Penn's Institute for Human Gene Therapy, and his team of scientists will check to see if the cells reproduce the new gene in the woman. Wilson hopes to find a cure for deadly hereditary diseases such as cystic fibrosis by correcting genetic defects in the body's cells. But, he cautions, the New Jersey woman's cystic fibrosis will not be cured by the therapy because only a small portion of the lungs are exposed to the corrective genes. Instead, the researchers hope to find a cure by studying the way her body reacts to the gene. "The goal of this study is to evaluate the feasibility and safety of this important technology," Wilson said. The therapy trial will include more than just the first patient; ultimately, there will be a total of 20 patients who are to be segmented into 10 two-person groups. Each group will receive a slightly increased dose of therapeutic genetic material than the preceding group. Wilson hopes his next step will be to apply the information gained during the trials to directly benefit the patients. "It's one thing to design an unproven medical strategy; it's quite another to allow that strategy to be played out within your body," Wilson said. The University trial marked the second time that gene therapy has been used on humans in an attempt to find a cure for cystic fibrosis. On April 17, scientists at the National Heart, Lung and Blood Institute in Bethesda, Md., used gene therapy treatment on a 23-year-old man with cystic fibrosis. Three other gene therapy trials involving cystic fibrosis are awaiting approval from the federal Food and Drug Administration.

Please note All comments are eligible for publication in The Daily Pennsylvanian.