Where Research is at for CF Cure

Last week Dr. Thompson told us about some of the research that is happening in order to get closer to a cure for CF. This week, it happened to hit the news. We are constantly praying for a cure, but I'm also praying for these researchers. A miracle is out there, it's just a matter of time.

So, Jeb, if you happen to be reading this years from now wondering what research was doing to help you. Here is an article on what is happening in 2009. I hope that you are reading this in the future and thinking, yep, that worked...


CYSTIC FIBROSIS GENE CLOSER (BBC, 17 Feb, 2009)
UK researchers plan to begin the first large scale trial of gene therapy in CF patients this year.
Proof of concept

In the future it is hoped that gene therapy will provide a cure by replacing the defective CF gene which disables or destroys a protein known as CFTR. In order to get the new, healthy gene into the cells, scientists use a harmless virus.

The University of California team, working with experts from the University of Iowa, found a way to make this virus more effective at entering lung cells. They mutated the adeno-associated virus to develop two major possessions - the ability to bind to different receptors or bind to a more plentiful receptor on the surface of lung cells and to make it past the cell surface membrane and into the cell.

Lead researcher Professor David Schaffer said: "In human lung tissue, it completely rescued the chloride ion transport properties of the cells after delivering the correct copy of the CFTR gene to replace the mutated copy of the gene that is present in CF patients."

A spokeswoman from the UK Cystic Fibrosis Gene Therapy Consortium, which is trialling gene therapy in patients, said: "The techniques used in this research are innovative, but more work is needed before this could be translated into clinical benefit for people with CF. "Although the results are positive in human lung tissue in a laboratory, it remains to be seen if it would work in the patient."

The UK CF Gene Therapy Consortium, funded by the Cystic Fibrosis Trust, are using a non-viral method to get the gene therapy product into the body and anticipate going to a major multi-dose clinical trial in 2010.

CF TRUST RESPONSE:
"The techniques used in this research are innovative, but more work is needed before this could be translated into clinical benefit for people with CF. The virus only carries a shortened version of the CF gene and it remains to be seen if it has the same function as the whole gene; the long-term treatment of a CF patient would require repeat administration of the virus which is usually not feasible as the body develops immune responses against the virus; and although the results are positive in human lung tissue in a laboratory, it remains to be seen if it would work in the patient.

The UK CF Gene Therapy Consortium funded by the Cystic Fibrosis Trust is currently trialling gene therapy in patients for clinical benefit. Our Wave 1 gene therapy research is using a liposome to get the gene therapy product into the body. Following this year's pilot study in humans, we anticipate going to a major multi-dose clinical trial in 2010."