Genzyme to pursue therapy, conduct clinical trials

EDITOR'S NOTE: A media briefing will be held at 11 am PDT (2 pm EDT) on Oct. 7. Reporters may join the briefing by conference call (see details at end of release). This is a joint release of Beth Israel Deaconess, Genzyme and the Cystic Fibrosis Foundation.

SEATTLE--Researchers say they have discovered a reversible lipid imbalance that may be responsible for the common symptoms of cystic fibrosis. The discovery, in mice carrying the genetic defect that causes cystic fibrosis, could lead to a treatment for a disease that affects an estimated 30,000 people in this country, according to Juan Alvarez MD PhD and Steven Freedman MD PhD of Beth Israel Deaconess Medical Center in Boston.

Alvarez and Freedman reported their findings today at the Cystic Fibrosis Foundation's annual scientific conference in Seattle. The researchers are working with the foundation and the biotechnology company Genzyme General, of Cambridge, Mass., to develop a treatment for cystic fibrosis.

To date, research on treatments for cystic fibrosis has focused on gene and protein therapies and antibiotic drugs. The discovery reported by Alvarez and Freedman points toward a new approach to treating the disease--one that relies on correcting a fatty-acid imbalance in the membranes of cells affected by cystic fibrosis. The researchers propose that the imbalance is the source of the chronic lung inflammation, excess mucus, and other symptoms of the disease. "They've made some tantalizing observations in an animal model," said Peter Durie MD, director of cystic fibrosis research at The Hospital for Sick Children in Toronto, where the cystic fibrosis gene was discovered 10 years ago. "No current therapy for cystic fibrosis deals with the underlying problem. Potentially, this is something closer to the fu
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Contact: Carol Cruzan Morton
cmorton@caregroup.harvard.edu
617-667-4431
Beth Israel Deaconess Medical Center
9-Oct-1999