Looks like a resurrection is upon us!!
Genzyme to Pursue New Approach to Treating Cystic Fibrosis
CAMBRIDGE, Mass., Oct. 9 /PRNewswire/ -- Genzyme General (Nasdaq: GENZ - news) announced today that it is collaborating with researchers at Beth Israel Deaconess Medical Center in Boston to develop an entirely new type of treatment for cystic fibrosis. The potential treatment is based on a discovery the researchers reported today at the Cystic Fibrosis Foundation's annual scientific conference in Seattle.
According to Juan Alvarez, M.D., Ph.D., and Steven Freedman, M.D., Ph.D., of Beth Israel Deaconess, a reversible imbalance between two fatty acids found in cell membranes may be responsible for the common symptoms of cystic fibrosis. Alvarez and Freedman reported they were able to reverse signs of the disease by correcting the imbalance in mice carrying the genetic defect that causes cystic fibrosis. The researchers corrected the imbalance by feeding the mice one of the fatty acids known as DHA. They also said that preliminary evidence shows a similar lipid imbalance exists in people with cystic fibrosis.
Genzyme is working to develop a therapeutically effective formulation for a DHA-based product designed to correct the lipid imbalance in cystic fibrosis patients and alleviate the chronic lung inflammation, excess mucus, and other symptoms of the disease. Genzyme plans to initiate clinical trials of a DHA- based product within a year and expects to conduct its trials in collaboration with the Cystic Fibrosis Foundation, which has a specialized network of medical research centers to expedite the early phases of clinical trials.
``We are moving very rapidly to develop a therapy for cystic fibrosis based on this extraordinary new approach to treating the disease,' said Henri A. Termeer, chairman and chief executive officer of Genzyme Corp. ``The research and pre-clinical work that have been completed will allow us to evaluate a DHA-based product in clinical trials very soon. Genzyme has a deep and long- standing commitment to patients with cystic fibrosis. This is truly an exciting moment for us.'
Genzyme has been at the forefront of cystic fibrosis research for nearly a decade. It has conducted eight gene therapy clinical trials in patients with cystic fibrosis and has also begun research to identify potential small- molecule drugs to treat the disease under a grant from the Cystic Fibrosis Foundation.
Genzyme intends to request fast-track designation from the FDA for its DHA development program. Genzyme also expects to apply for orphan drug designation for a DHA-based product. Under an agreement with Beth Israel Deaconess Medical Center, Genzyme has been granted an exclusive license to intellectual property related to DHA therapy. Patent applications have been filed on the use of DHA in the treatment of cystic fibrosis, and additional patent filings are expected on various formulations being developed by Genzyme in collaboration with Beth Israel Deaconess.
Research Findings
In the study reported today, Alvarez and Freedman examined differences in the relationship between levels of two fatty acids in normal mice and mice that had the genetic defect responsible for cystic fibrosis. They found that the cystic fibrosis mice had abnormally high levels of arachidonic acid (AA) and abnormally low levels of docosahexaenoic acid (DHA) compared to normal mice. The lipid imbalance was confined to organs most affected by cystic fibrosis, including the lungs, pancreas, and intestine.
To test whether the imbalance between AA and DHA was responsible for the expression of the disease, doses of DHA were fed to the cystic fibrosis mice for one week. The researchers found that administration of DHA not only corrected the lipid imbalance in affected organs but also reversed the signs of cystic fibrosis in the pancreas, intestine, and lungs of the mice.
DHA is a fatty acid found in sources such as fish oils and breast milk and is available in over-the-counter nutritional supplements. The researchers caution that current formulations of these supplements will not be effective at the recommended doses or well tolerated at the doses that may be required to achieve clinical effect. In previous studies of the use of fish oil supplements to control lung inflammation, people with cystic fibrosis have shown no statistically significant clinical improvements compared to control subjects. Also, the main component of fish oil supplements is another fatty acid that may block the DHA action.
``Although a number of steps remain before the benefit of DHA to patients is known, this exciting research represents an entirely new strategy to correct and possibly even prevent some of the ravages of this disease,' said Robert J. Beall, Ph.D., president and chief executive officer of the Cystic Fibrosis Foundation. ``The discovery also illustrates a true partnership in action-as industry, academia and a private non-profit health organization come together to translate science in the laboratory into innovative new treatments for our patients.'
Cystic fibrosis is the most common fatal hereditary disease among Caucasians in the United States, affecting an estimated 30,000 people. The median life expectancy of patients with cystic fibrosis is 32 years. Cystic fibrosis is caused by a defective gene that affects multiple aspects of cellular function. The most debilitating consequence of the disease occurs in the lungs of cystic fibrosis patients, where abnormally thick, sticky mucus clogs the airways, leading to fatal lung infections.
Beth Israel Deaconess is a major teaching hospital for Harvard Medical School and a founding member of CareGroup.
Genzyme General develops and markets therapeutic products and diagnostic products and services. Genzyme General has three therapeutic products on the market and a strong pipeline of therapeutic products in development focused on the treatment of rare genetic diseases. A division of the biotechnology company Genzyme Corporation, Genzyme General has its own common stock intended to reflect its value and track its economic performance.
This press release contains forward-looking statements, including statements concerning the estimated U.S. cystic fibrosis patient population, the planned initiation of clinical trials and related collaborative efforts, a request for fast-track designation, and an application for orphan drug status and additional patent application filings. Actual results may differ materially due to numerous factors, including, among others, the accuracy of the parties' information about the incidence of cystic fibrosis in the United States, the results of a study concerning the relationship of AA and DHA in people with cystic fibrosis, the results of efforts to develop a therapeutically effective formulation for a DHA-based product, the timing and content of submissions to and decisions by regulatory authorities, the enrollment rate for-and results of-clinical trials, the ability to satisfy the criteria for fast-track designation and orphan-drug designation, and the competitive environment for DHA-based products.
Genzyme's releases are available on the World Wide Web at genzyme.com. They are also available from Genzyme's fax-on-demand service at 1-800-436-1443 within the United States or 1-201-521-1080 outside the United States.
SOURCE: Genzyme General |
