Thursday 26 March 2015

Things I learned on a summer studentship

Kate Ryan, an undergraduate medical student at Keele University, took part in the Cystic Fibrosis Trust’s Summer Studentships programme in 2014, where she worked on a PhD project on cystic fibrosis-related diabetes. Following her placement in the academic holidays, Kate won first prize for her poster presentation at the UK CF Conference.

Initially I wanted to complete a studentship over the summer as I have been interested in research ever since my first year of medical school. I gained a lot from participating in a summer project funded by the Cystic Fibrosis Trust with regards to lab skills, working as part of a team and becoming more familiar with the process of research; from planning, right through to results and presentation.

This particular project allowed me to present at my first conference which was a great experience for someone considering a career in research and helped me recognise areas I did well in as well as those I could have improved on. I also got the opportunity to meet some of the top names in cystic fibrosis research and to attend workshops about exciting new treatment options. 

Although I have had some experience in medical school, working in a lab was a relatively new experience for me. I was taught about general lab safety as well as the relevant techniques needed for my project. This hands-on approach to research was the part I found most enjoyable.

This research project has led me to consider intercalating next year and possibly considering further research in my career. It has helped me realise the importance of research and how enjoyable it can be.

I would highly recommend this to any medical student, whether you are interested in a career in research or not.

Thanks to the Cystic Fibrosis Trust for such a great opportunity last summer!

Find out more about the studentships programme at

Monday 23 March 2015

Understanding the VX-661 Phase 2b Results

Pharmaceutical company Vertex, today released the results of a recent Phase 2b trial, which looked at safety of a drug known as VX-661 in combination with ivacaftor (Kalydeco). There has been a lot of anticipation about these results because it is only the second time that the company has issued trial data. Below, Dr Anoushka de Almeida-Carragher, Senior Research Manager looks a what they tell us.

This study involved 39 people with cystic fibrosis who have two copies of the F508del mutation, and confirmed that the treatment is safe and well tolerated.  The stated improvement in lung function, from baseline, within four weeks of treatment is also encouraging. It should be noted that the number of people who took part in the study is extremely small. However the results provide solid support for the next stage of trials – Phase 3 – which Vertex commenced in the early part of this year.

This program of four Phase 3 clinical trials will look at lung function after the administration of the VX-661/ivacaftor combination, but this time involves not only people with two copies of the F508del mutation – who were studied in the phase 2b trial, and make up roughly 51% of the CF population – but also includes people who have one copy of the F508del mutation and a second mutation that is either a gating mutation, residual function mutation or a mutation that results in minimal CFTR function.

This will the first time that the efficacy of this drug regime is being tested in a large population of people with cystic fibrosis (approximately 1150, aged 12 and older). Furthermore, the fact that 4 different ‘mutation combinations’ are being investigated, thus impacting on a larger number of CF patients, means this next-stage trial is vital in enhancing our knowledge-base in striving to reach our goal of beating CF for good.

The Trust will be watching the Phase 3 trials with interest and ensure that our CF community remain updated on the progress in transformational treatments.

Friday 6 March 2015

Working with Pharma. Ed Owen's US blog part 2

As well as meeting the Cystic Fibrosis Foundation in the US last week, the Trust’s Chief Executive, Ed Owen, and Director of Research & Care, Dr Janet Allen, also dropped in on two pharmaceutical companies of importance to cystic fibrosis, Novartis and Vertex. Here is Ed’s second blog explaining what they find out:

Much is said about the pharmaceutical industry. Some of it is fair, some less so. But the fact remains that only pharma companies have the size, expertise and capability to develop and produce the drugs that can make a difference to people with cystic fibrosis. So it is vital that we have strong relationships with key companies, like Vertex and Novartis, and work with them for the benefit of our community. 

The Vertex story is an extraordinary one, and one in which our sister charity, the Cystic Fibrosis Foundation (CFF), has played a central role. It also tells us much about the industry, its weaknesses and strengths, limitations and potential.

Long frustrated by the lack of industry interest in cystic fibrosis in the late 1990s and early 2000s, the CFF began funding a number of commercial programmes, including one led by a biotech company called Aurora Sciences, later bought by Vertex, to find compounds to develop drugs to tackle the genetic causes of cystic fibrosis.

A decade and a half on, and Vertex is a pharmaceutical trailblazer. Ivacaftor (Kalydeco), is transforming the lives of those with the G551D mutation and a combination therapy of ivacaftor and lumacaftor has been submitted for approval for use in people with two copies of the F508del mutation.

Being a trailblazer however comes at a price, and the high price of ivacaftor has been controversial – with many questions about the likely price of the combination therapy if it is granted approval from the regulators.

With a cash-strapped NHS, and a system of drug appraisal that in England, at least, is ill-suited to assess particular issues relevant to conditions like cystic fibrosis, there is a real risk that this combination therapy is given regulatory approval in Europe later this year for use in four out of 10 people with cystic fibrosis but will not reach those that need it here in the UK for a considerable amount of time, if at all.

Our focus must be to ensure that approved therapies get to those who need them as quickly as possible - and we are discussing these issues now with all key players.

But, when we met them last week, Vertex continued to be tight-lipped about future pricing in advance of regulatory approval. Along with other pharmaceutical companies, the company argues that the price of drugs reflects both the high cost of investment that has been made over many years to develop and trial therapies (estimates vary, but the average cost of producing a new drug is usually well above £1bn) and the relatively small numbers of patients with ‘rare diseases’ like cystic fibrosis.

Like every other pharmaceutical company, Vertex has shareholders who demand an appropriate financial return on their investment. The long-term prospects of the cystic fibrosis ‘market’ means that Vertex is continuing to invest in both their immediate pipeline of potential therapies and in longer-term research to develop what they describe as ‘second generation’ drugs that they believe could be even more effective in future years.

We are delighted they are doing so, and are keen to see other drugs companies invest in cystic fibrosis research too. One such company is Novartis. It already produces cystic fibrosis drugs, most commonly Tobramycin. But we have had concerns about its future commitment following its decision to move its wider respiratory research base from Sussex in the UK to Boston in the US in late 2013.

We were therefore pleased to meet the Novartis team in Boston last week and to receive their reassurance that their cystic fibrosis-related drug programmes were alive and well.

For the first time in decades, the pharmaceutical industry is investing heavily in cystic fibrosis. As President Obama said in January, the condition is at the cutting edge of new advances in ‘precision medicine’ with new therapies being developed focused on defined patient groups with particular genetic mutations.

That investment will only continue for as long as companies believe that health systems will pay for the drugs that follow. Therein lies the challenge for the NHS, ourselves and all those wishing to see innovative treatments that transform the lives of people with cystic fibrosis accelerated as quickly as possible.

High-cost drugs are the future. How they are paid for is the key question that we all need to address.