We want to build on previous achievements, provide leadership to harness these changes and ensure our research investments benefit everyone with cystic fibrosis.
Research – some of it supported by the Trust – and improvements in clinical care have already had a significant impact on the quality and length of life of patients with the condition. What was once considered a childhood genetic disease is now seen as one that extends well into adulthood and we are working towards everyone with CF having a life unlimited by their condition.
chance in 1964 that a child with cystic fibrosis would reach school age.
of people with cystic fibrosis in the UK CF Registry in 2011 were over 16.
was the median predicted life expectancy of a person with cystic fibrosis in 2011.
Dealing with a condition that has seen life expectancy increase substantially brings new challenges. New drugs and therapies can add to an already onerous treatment burden for people with cystic fibrosis. The NHS has to provide ongoing care for an increasing number of adults with cystic fibrosis, and the wider research community needs to confront the new clinical challenges that emerge.
However, the ultimate goal for research in cystic fibrosis is to understand the basic fundamental defect of CFTR. Investments in this transformational research are long term and high risk: results do not appear overnight.
For instance, we have invested for over ten years in the Gene Therapy Consortium and the wave 1 product is now in Phase 2b clinical trials. We have also supported fundamental research that provides a better molecular understanding of the basic defect and how mutations of the gene affect the functioning of the protein, CFTR. This work is underpinning drug discovery programmes currently funded by other agencies and the biopharmaceutical industries.
Changes in the wider research environment
Over the last year, there have also been some significant changes in the wider medical, policy and science environments.
- The biopharmaceutical industry now places more emphasis on working with external partners, rather than relying on its own in-house discovery programmes.
- For the first time, a drug – ivacaftor (Kalydeco) – that tackles the root cause of the disease has been licensed for use in the UK.
- The cystic fibrosis community may be on the cusp of a significant change towards personalised medicines to treat the condition.
- The government has placed a priority on health research to benefit patient welfare and care.