Cystic fibrosis

Four challenges in the next five years: Elborn


In the closing plenary to wrap up this year’s conference outgoing ECFS President Stuart Elborn gave an insightful talk outlining what he thinks are the biggest challenges in treating and managing CF over the next five years.

Challenges according to Elborn:

1. Treat early and prevent disease

No P. aeruginosa chronic infection in the first two decades.

Speaking as an adult CF physician Elborn challenged paediatric centres to have no P. aeruginosa coming into adult care. “Send us patients with FEV1 greater than 90% predicted… I believe that will make a further significant impact on the outcomes for our patients,” he told delgates.

2. Restore CFTR function in all patients

with mutation specific or mutation agnostic precision therapies

There have been fantastic advances in the understanding of CFTR and the functional consequences of CFTR mutations that occur in CF patients, Elborn told delegates.

“We are now living in an era where we have therapies that treat basic defects and we shouldn’t take this for granted,” he said.

Recounting what he says was one of the most exciting days of his life, Elborn tells the audience about a dark February evening when he had a teleconference with colleague Bonnie Ramsey and the team from Vertex.

“For the first time we saw the effect ivacaftor had on Fev1 and exacerbations [phase III results]…It was way beyond my expectations and it was a seminal moment for us as a community,” he told delegates.

It also had a wider impact in medicine by showing that if you have focused targets you can make a difference, he said.

“We are really on the road here but there’s much still to do… for the next five years we need to really commit ourselves as CFTR scientists for better and even more effective precision targeted treatments for the basic defect in all of our patients,” he said.

3. Precision medicine for lung disease

Biomarkers to personalise treatment

Lung disease in CF is a toxic and difficult environment that we need to understand better in order to deliver targeted therapies that stop the processes driven by bacteria, inflammation and injury, said Elborn.

“We have an opportunity with molecular testing and diagnostics in microbiology to make a real shift forward in understanding this complex microenvironment,” he said.

This will help us to figure out how to use antibiotics, anti-inflammatories and mucolytic drugs in a much more effective way, he said.

“There’s much much more to do to get the precision we need to use the new drugs in a much more targeted way that’s personalised to individual patients.”

4. Optimal and affordable CF care worldwide

where all patients have access to CF team care and transformative therapies.

By 2025 there’s going to be a 75% increase in the number of adults with CF and the number of CF centres and resources will need to double, Elborn told the audience.

Coupled with an era of austerity in Europe, where there isn’t going to be a lot of money for health spend, we need to figure out how we’re going to provide optimal CF care, he said.

“As a CF community this is something we need to be thinking and talking about together,” he said.

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