Imperial Innovations is negotiating with pharma companies over its gene therapy that has shown the potential to treat cystic fibrosis. A trial of the gene therapy posted patchy but promising data this week, putting the tech-transfer business in a position to strike a deal for further development.Study leader Eric Alton
The clinical trial gave monthly doses of either the gene therapy or a placebo to 136 people aged 12 years and older with cystic fibrosis. When assessing both trial arms after one year of treatment, researchers found no differences in treatment-attributable adverse events and a significant, but modest, improvement in lung function among patients who received the gene therapy. The trial consequently hit its primary endpoint but the data were far from a slam dunk, notably because some patients responded much better to the treatment than others.
"The effect was inconsistent," Eric Alton, a professor at Imperial College London and leader of the study, said in a statement. However, when viewed in the context of the previously fruitless 25-year struggle to turn knowledge of the role of CFTR in cystic fibrosis into an effective gene therapy, even inconsistent data are cause for encouragement. The focus now shifts to finding ways to improve the efficacy of the treatment and breadth of patients it benefits. Increasing the dose or combining the gene therapy with drugs to boost the activity of CFTR are two options being considered.
Imperial Innovations is in talks with pharma companies about financing a larger trial of this tweaked formulation. The underlying idea behind the revised formulation will be the same as the one struck upon by the UK Cystic Fibrosis Gene Therapy Consortium (CFGTC), a group of universities that came together in 2001 to work on the problem. The asset CFGTC finally advanced into the clinic is plasmid DNA encoding for CFTR that is encased in a liposome and delivered via a nebulizer. CFGTC struck upon the approach in an attempt to evade the defenses of lung cells.
While Imperial Innovations is shopping this approach to pharma companies, the consortium is looking at another way of delivering CFTR to the lungs. This alternative method would replace the liposome with a viral carrier designed to efficiently infect lung cells. If CFGTC can raise the cash, it wants to test the idea that the lentivirus carrier will trigger a stronger, more sustained response in a clinical trial by 2017.