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New gene therapy treatment for cystic fibrosis?

9 June 2005
By BioNews
Appeared in BioNews 312

The inherited lung disorder cystic fibrosis (CF) could be treated using parts of two viruses to deliver a therapeutic gene, US researchers say. A team based at the University of Pennsylvania says that it has successfully used a 'hybrid' of the viruses that cause HIV (human immunodeficiency virus) and Ebola to deliver test genes to the lungs of monkeys. Now, according to a report in New Scientist magazine, the scientists are planning a more extensive monkey trial to convince the Food and Drug Administration (FDA) that the technique would be safe to use in people.

CF is a progressive, life-threatening condition that mainly affects the lungs and digestive system. It is caused by a mutation in the cystic fibrosis transmembrane conductor (CFTR) gene. Researchers have been trying to develop gene therapy treatments for CF for several years, but their efforts have been plagued by difficulties in delivering enough copies of the working CFTR gene to the affected tissues. The Pennsylvania team have tried to overcome this problem by combining elements of two different viruses to create a more efficient 'gene delivery system', or vector. Since the Ebola virus is very effective at targeting lung surface cells, the researchers took a surface protein from Ebola and added it to HIV - one of several viruses that reproduces by copying its own genetic information into that of the host cell. They then used the new hybrid virus to deliver a test gene to the lungs of monkeys, and found that after two months the gene was active in around 20 per cent of the lung cells.

The results, which are currently being prepared for publication, suggest that the hybrid virus is more efficient at delivering the CFTR gene than vectors used previously. Although it uses parts of two potentially deadly viruses, the researchers are confident the hybrid virus would be safe to use in humans.

However, US gene therapy researcher Ray Pickles, who was not involved in the study, told New Scientist that he has abandoned similar research because he thinks that opposition from a fearful public will prevent the method being used. In addition, because the approach targets lung surface cells, which die off after a few months, patients would require repeated treatments. UK expert Steve Hyde, of Oxford University, also expressed reservations about using the virus. 'The body generates antibodies to it', he told BBC News Online, adding 'the therapy works the first time but it's not guaranteed to work the second time, or subsequent times'.

HIV and Ebola lung disease 'cure'
BBC News Online |  9 June 2005
Should deadly viruses be used to treat cystic fibrosis?
New Scientist |  9 June 2005
31 May 2011 - by Dr Kimberley Bryon-Dodd 
Two genetic regions that might explain some differences in disease severity in cystic fibrosis (CF) patients have been identified by US and Canadian researchers. The two regions contain genes that can modify lung function...
26 July 2009 - by Dr Rebecca Robey 
Scientists have used two new techniques to fix defects in lung cells from people with cystic fibrosis, raising hope for new treatments for the disease in the future. The first study, published in the journal Plos Biology, used a gene therapy technique to treat the cells, whilst the second study, published in the American Journal of Respiratory Cell and Molecular Biology, used a drug called miglustat....
2 June 2006 - by Heidi Nicholl 
A paper has been published in the Journal of Medical Ethics that investigates the possibility of trialling gene therapy for cystic fibrosis (CF) in children with the condition. Conventional wisdom has generally suggested that it would be unethical to use untested new treatments, such as gene therapy...
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