Young Investigators give research a breath of fresh air!
The European Young Investigators Meeting of cystic fibrosis (CF) researchers was held in Paris at the end of February. It was hosted by the French CF Association ‘Vaincre la Mucoviscidose’ and co-funded by a number of CF patient organisations under the umbrella of CF Europe. Research on every aspect of cystic fibrosis was presented including understanding inflammation and infections, exploring new knowledge for future treatments, and looking at better ways to support people with their symptoms.
The aim of the conference was to strengthen the knowledge and skills of the 35 young investigators attending, and to allow them to build and develop relationships that will last throughout their careers. We asked Dr Belinda Cupid from the Cystic Fibrosis Trust in the UK to share some of her highlights from the meeting.
Learning about a cellular ‘pac-man’
Inflammation is one of the body’s natural defence mechanisms, but it goes into overdrive in CF which leads to lung damage. Although we know that there is excess inflammation in CF, it is not clear what causes it. Macrophages are cells in our body that are involved in inflammation. They act like the computer game ‘pac-man’, engulfing bacteria or dead cells and destroying them. One cause of the excess inflammation in CF could be that the macrophages are going wrong.
As macrophages are involved in fighting off lung infections, it can be hard to tell whether the excess inflammation is linked to the lung infections people with CF develop, or if the inflammation was there first. This is what Florian Jaudas from Germany is investigating in his research. As he explained at the conference, it is like trying to work out which came first – the chicken or the egg!
In the same morning we heard from Francesco Luly from Italy, who is looking at an earlier step in the process: what switches the macrophage activity on, what switches it off again, and are these triggers different in people with cystic fibrosis? The work that Florian and Francesco and other researcher’s are doing, could give us a better understanding of inflammation, which could lead to the development of treatments to reduce it.
Better treatments for CF
Research into new and better ways to treat the underlying cause of CF was presented at the conference. As part of this, ‘organoids’ were definitely a hot topic of conversation!
‘Organoids’ are 3D balls of cells that act like mini-organs and can be created in several ways, including scraping cells from the inside of the nose or taking a biopsy sample from the rectum.
“The awesome thing is that organoids carry the same mutation as their CF-er”, explained Eva Furstova from the Czech Republic, who is using rectal-derived organoids to look at individual responses to Symkevi and Orkambi. “By assessing treatment responses in the lab on organoids, we want to bring the perfect medication to individual patient regardless of the mutation”.
As well as testing the effectiveness of current CFTR modulators on the market, organoids are also a good way of looking for new drugs for treating cystic fibrosis. One approach is to develop drugs that don’t work on the CF protein, but work on proteins that do a similar job in the cell. These proteins are known as ‘alternative chloride channels’. If they prove to be effective, these drugs could work for all people with CF irrespective of their genotype.
Improving how CF is managed
While most of the conference was about lab-based research, it was reassuring to discuss the improvements to the day-to-day treatment burden and mental health challenges for people with CF and their families too.
Presenters from the Netherlands and Turkey told us about what they were doing to support people with CF and their families with anxiety and depression. As well as talking to people about how they are feeling, CF teams now have questionnaires that help them assess anxiety and depression.
If you’ve come across these questionnaires, Dr Marieke Verkleij from Amsterdam would like to know your thoughts about them. You can tell her and her team by completing this online questionnaire. The survey is anonymous, and it will take between 5 and 10 minutes to complete.
‘Top Gun’ researchers of clinical care
Dr Hettie Janssens, a paediatrician from Erasmus University in Rotterdam in the Netherlands, set the scene of current clinical management in her talk. She highlighted topics requiring further research and finished by describing the CF clinical team as ‘pilots with a cockpit of measurements to manoeuvre the best path of clinical management of cystic fibrosis’. Her lovely explanation made me compare the other young investigators who presented in the same session to confident new pilots, similar to those in the ‘Top Gun’ film!
One young ‘pilot’ investigator who presented their research was Martinus Oppelaar. Martinus’ research explores better ways of measuring lung function in children under the age of five, investigating particularly if a shortened version of the lung clearance index (LCI) test is as accurate as the current, longer version.
Renate Kos’ topic also related to young children with CF and the urgent need to find better ways of detecting infections. As she explained in her competition-winning tweet during the science communication workshop, she is exploring whether “using something as simple as a breathalyser for detection of harmful bugs in the lungs might be possible in the future?”. She found that “CF children with a lung infection exhale tiny compounds that can be used to identify bacteria present in the lungs. #CF #Breathe”.
I’ve only included a few of my highlights, as there was so much impressive science presented! With all of the changes in CF care and treatment happening or on the horizon, the enthusiasm of these young doctors and scientists made me optimistic for the future, knowing that everyone with CF and their families will benefit from the knowledge they uncover, irrespective of the CF mutation they have and where in Europe they live.