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Cystic fibrosis gene therapy trial results encouraging

Case Western Reserve University : 06 February, 2007  (Technical Article)
Scientists and physicians in Cleveland have announced encouraging results from the first-of-its-kind gene therapy trial involving cystic fibrosis patients and a new compacted DNA technology.
The Phase I trial involving 12 patients was launched one year ago by University Hospitals of Cleveland, Case Western Reserve University School of Medicine, Children's Hospital of Denver and Cystic Fibrosis Foundation Therapeutics Inc, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation.

Cleveland-based biotechnology firm Copernicus Therapeutics Inc. produced the non-viral gene transfer system used in the clinical trial. Working together with UHC and CWRU researchers, Copernicus formulated a way to 'compact' or tightly bind strands of DNA so that it is tiny enough to pass through a cell membrane and into the nucleus. The ultimate goal is for the DNA to produce a protein needed by people with CF to correct the basic defect in CF cells.

'The primary goal of this Phase I study was to determine if this gene therapy method is safe and tolerable as administered in this trial. All participants in this study completed the trial without significant side effects and the treatment itself was well-tolerated,' said Michael W. Konstan, M.D., associate professor of pediatrics at CWRU and director of the LeRoy Matthews Cystic Fibrosis Center, Rainbow Babies & Children's Hospital of UHC. 'The secondary goals of the trial were to evaluate if the CF gene was successfully transferred to airway cells and if it functioned normally, results which would suggest that this therapy may be of benefit to people with CF. Our data were very encouraging with indications that this gene transfer may have occurred.'

'This gene therapy research has exciting potential as a new approach to addressing the genetic root cause of CF,' said Robert J. Beall, Ph.D., president and chief executive officer of the Cystic Fibrosis Foundation. 'We are pleased that this novel method of gene delivery has cleared the first hurdles of clinical research by proving both safety and tolerability. We eagerly anticipate results of future clinical studies utilizing this novel approach.'

CFFT helped fund this initial trial, along with Copernicus and the National Institutes of Health through a Core Center Grant to the CF Center at UHC/CWRU and through the General Clinical Research Center.

Copernicus Therapeutics recently received more than one million dollars in state funding through the Technology Action Fund of the Ohio Department of Development to continue development of compacted DNA. The company is currently working on an aerosol version that would enable people with CF to have the healthy gene delivered directly into their lungs.

During the recently completed study, patients received the compacted DNA in a saline solution dripped into the nasal passages. Future clinical trials will study the safety and efficacy of the aerosol approach.

'Gene transfer technology is expected to revolutionize treatment of genetic disease by using DNA as a novel therapeutic,' said Jeff Wagener, M.D., professor of pediatrics at the Children's Hospital of Denver, which enrolled three patients in the study. 'Striking at the root cause of CF should ultimately provide an even more effective treatment for CF than those available today, which are aimed at managing the side effects of the disease.'

The underlying cause of CF, which affects approximately 30,000 Americans, is a defective gene that upsets a delicate salt/water balance in the lungs. At the crux of the process is a protein, produced by the CF gene, which controls the flow of salt and water in and out of cells. In CF patients, this protein does not operate normally in the cells that line the airways. In turn, the airways accumulate thick and sticky mucus. Bacteria proliferate in the mucus and cause chronic infections that permanently damage lungs.

Under the direction of Konstan, the research teams for this recently completed Phase I study delivered the healthy gene into twelve adult CF patients in a saline solution dripped slowly into their nasal passages. Investigators monitored salt transport in the nose, called the 'nasal potential difference,' as a barometer of the procedure's success.

'CF patients have a markedly abnormal nasal potential difference,' Konstan said.

Through biopsies of nasal tissue, researchers determined whether the healthy gene was 'taken up' by the cells and produced enough protein to affect the transport of salt and water in and out of the cells. They found that two-thirds of patients treated had a meaningful increase in the transport of chloride ion in the nose.

The researchers and physicians involved in this study will present their findings at the American Society of Gene Therapy meeting in Washington, D.C., in early June and at the Cystic Fibrosis Foundation Williamsburg meeting at the end of May.
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