Promise, and caution, as gene therapy shows potential for cystic fibrosis
- In patients with cystic fibrosis (CF), the effects of treatment with gene therapy from Imperial Innovations were significant, but came with modest and variable improvements in lung function.
- The techique replaces the faulty gene behind CF and relies on using inhaled DNA molecules to replace a faulty gene.
- Results of the study were published in The Lancet Respiratory Medicine journal.
Overall, CF is the most common genetically inherited, deadly disease—one that affects more than 90,000 people worldwide. The results of the 12-month, placebo-controlled study, which included 136 CF patients aged 12 and older, are still considered preliminary. But they still provide a great deal of information about what works.
One thing that seemed effective was the delivery mechanism that the research team used. The inhaled molecules of DNA were wrapped in liposomes in the first study, but now researchers are considering using a more potent gene transfer system, including a hybrid virus vector.
Gene therapy is still highly experimental and many development efforts have been riddled by failure, as well as an unwillingness to go too far when there is not a clear signal of potential success. In addiition, there is the cost factor. Gene therapy is expected to cost up to $1 million for a treatment. However, companies continue to develop gene therapies, hoping for the ultimate upside: A treatment that is curative, once and done.