Back from Boston & Bethesda

CEO Ed Owen and Director of Research Dr Janet Allen spent the first half of this week in the US, meeting with colleagues from across the American cystic fibrosis community. Ed fill us in on some of the highlights from meeting with our US counterparts.
Weeks of sub-zero temperatures and Arctic-like weather have left much of the North Eastern coastline of the US encased in ice and snow. Yet our visit this week to see Vertex and Novartis in Boston, and the Cystic Fibrosis Foundation (CFF) in Bethesda, demonstrated that the work to beat cystic fibrosis continues to generate a great deal of heat and light.

What we heard from all those we saw gives me renewed confidence about the future prospects of research. But, while competition in the pharmaceutical industry is an important driver in pushing the development of new drugs, this needs to be matched by new forms of international collaboration and partnership that pools effort and resource in the best interests of people with cystic fibrosis.

Our strong relationship with the CFF is crucial here. We have shared goals, represent communities of people who are themselves increasingly interconnected, and both have access to key clinical, academic and business expertise. We can bring this together on tackling the big issues facing people with cystic fibrosis so the potential for greater and quicker advances becomes a reality.

The Foundation’s success in helping to develop and promote new therapies in cystic fibrosis has been extraordinary.  We in the UK owe a great debt to them – and their $3.3bn windfall late last year resulting from the selling of their Kalydeco royalty rights gives them significant new resource to invest in new research to bring hope to everyone with cystic fibrosis wherever they live.

But, as the Foundation will readily admit, they do not have a monopoly on wisdom or expertise – and are keen, too, to explore areas of collaboration with the Cystic Fibrosis Trust in particular areas that can bring impact.

At our meeting, we therefore discussed a range of areas of potential joint research activity. One was tackling the challenges posed by what are called ‘nonsense mutations’ of cystic fibrosis, and developing novel treatments that would benefit around 1,000 people here in the UK alone. The existing pipeline of small molecule therapies being developed by Vertex and other pharmaceutical companies would not affect this group so it is an important area for ourselves and the Foundation.

A further area we explored was transformational work in gene therapy and stem cell research. The UK has particular expertise here, not least that generated by the Gene Therapy Consortium, supported by the Trust over many years and soon to publish the results of its Phase 2b study. In stem cells too, the UK has a strong scientific base and we are keen to see more of this focused on cystic fibrosis over the coming years.

We also discussed joint approaches to tackling emerging infection threats to people with cystic fibrosis such as NTM and aspergillus. The CFF is supportive of the Strategic Research Centre on NTM being funded by the Trust, and keen to follow its progress.

Finally, we talked about our SmartCareCF programme and working together to harness new technology to give people with cystic fibrosis the tools to take greater control of their lives, as well as increasing access to clinical trials here in the UK.

I believe that in these and other areas we can demonstrate real collaboration over 2015 to bring together the greatest possible expertise and resource in our collective fight to beat cystic fibrosis for good