December 23, 1997
SickKids scientists overcome gene therapy hurdle
TORONTO - A research advance by scientists at The Hospital for Sick Children (SickKids) has the potential to overcome two key obstacles preventing successful and safe gene therapy in patients with cystic fibrosis and lung cancer. The advance enables the therapeutic gene to work only in the desired location in the lung and uses only human genetic material to make the gene work.
Research carried out by Dr. Yu-Hua Chow in the laboratory of Dr. Jim Hu of the Hospital's Lung Gene Therapy Program, has led to the development of a unique delivery package which makes a normal gene function in the cells lining the airways of the lung. The research is reported in the December 23 issue of the Proceedings of the National Academy of Sciences USA.
One type of gene therapy is to place a normal gene into the part of the body where the native gene is defective; this represents a new approach to treating inherited diseases. In gene therapy for cystic fibrosis, a lethal inherited lung disease, the goal is to deliver a functioning gene called the cystic fibrosis transmembrane conductance regulator (CFTR) to the surface cells of the lung. It is only these cells which are affected in the lungs of cystic fibrosis patients.
"Our work represents a new direction in gene therapy research," explains Dr. Hu, who is also an assistant professor of Paediatrics at the University of Toronto. "Until now, much of the research and early clinical trials in this field has focused on using genetic material from viruses to make the genes work. However, this method has been largely unsuccessful to date, in part, to the fact that it doesn't target the specific lung cells. There have also been concerns about the safety of using viruses. We have overcome these problems by using normal human genetic material that specifically targets the lung surface cells to make the transferred gene function. By chance, we also discovered that our newly developed delivery package may also be very useful in the gene therapy of lung cancer since many lung cancers arise from the cells lining the lungs' airways."
SickKids Lung Gene Therapy Program was established in 1995 under the direction of Dr. Hugh O'Brodovich, then head of Respiratory Research and now Paediatrician-in-Chief at SickKids and chairman of Paediatrics at the University of Toronto. The program's initial focus has been to identify and overcome the biological and technical barriers to successful lung gene delivery and develop effective agents for the safe and normal functioning of transferred genes. This research supports this program's strategy.
Funding support for this research was provided the Canadian Cystic Fibrosis Foundation (SPARX II), the Medical Research Council of Canada, and The Hospital for Sick Children Foundation.
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