Spectrum Summer 2019

SPECTRUM Summer / 2019

Let me tell you a story... The launch of "Our Baby has Sixty-Five Roses"

www.cfireland.ie

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CONTENTS Latest News | 1 - 3

* Hospital Updates * Travelling with CFTR Medicines * Community Events * The Hyde Family * The Irish Donor Network * Working with other Patient Groups * Thank You Erin

Spotlight | 4 - 6

Gastrointestinal Issues & CF * A Parent's Perspective * Personal Experience

Hospital Hub | 7 Member Services| 8 Community Events| 9 - 14 * Seven Summer Stars * Our Baby has Sixty-Five Roses

Kids Corner | 15 Youth Reach | 16 Conference Reports| 17- 23

* CFI Annual Conference * ECFS

Research | 24 - 26

* Phages as a therapy against infection * PERT Pilot Study * Study update on treatment of lung infections * Superbugs Webinar

CEO’s Message Welcome to the summer issue of Spectrum. We have a bumper issue that includes a report from the European CF Conference in Liverpool; our work with other patient groups to improve services; our regular hospital update and much more.

Philip Watt Chief Executive

It has been a busy few months for the CF Community beginning in March with the Annual Conference, we then had 65 Roses Day in April. The Malin 2 Mizen Cycle, the 2nd annual CF Art Exhibition in Limerick and the launch of the Helium Arts Project in Waterford all took place in May and so far in June we have had the One in 1,000 campaign and launch of "Our Baby has Sixty-Five Roses." The big news from this year’s European conference was the very promising forthcoming Vertex triple combination drug therapy. CFI understands that this drug therapy is being fast-tracked, but it may still take up to 18 months before it is approved by the European Medicines Agency (EMA). We also include a report on our own conference held in Carlow in March – many of the podcasts are now available on our website. There have been a number of positive developments with the hospitals and we continue to advocate for our members both here and abroad on improving CF services and care. Finally, we would like to say farewell and many thanks to our Regional Officer, Erin Sugrue, who has contributed much over the past 5 years to improving CF services in Limerick Hospital in partnership with TLC4CF.

Philip Watt, CEO, CFI Samantha Byrne & Nuala McAuley, Editors

Fundraising |27 - 37 * Challenges and Events * Thank You

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DISCLAIMER: The views of contributors, when expressed in this publication, do not necessarily reflect the position or policy of Cystic Fibrosis Ireland.

Spectrum / Summer 2019

LATEST NEWS: Beaumont Hospital Update The proposed new 20-room in-patient unit in Beaumont Hospital has received planning permission and the hospital is currently in the process of inviting tenders for the building of the unit. This means that contractors will be invited to submit bids to undertake this major building project. We have been told this process will be completed by July / August 2019 and a decision to appoint a preferred contractor will require a further number of weeks – so it is likely a decision will be made in early Autumn. The final decision on when this project receives funding is still to be taken by Government, but we will keep up the pressure. CFI undertakes this work in partnership with local charity CF Hopesource. We have pledged to fundraise ₏1m towards the cost of this project. In short, there is good continuing progress which follows on the decision by Dublin City Council to approve planning permission in January 2019. Senior Registrar in Beaumont CFI and CF Hopesource have funded a CF Registrar (senior doctor) specialist post in Beaumont Hospital for approximately two years at a cost of ₏100,000 per annum. We were recently told that this post will now be funded by the HSE / Beaumont Hospital, which is welcome news. Mater Hospital Following representation from CFI over many months, the Mater Hospital have indicated their intent in converting 7 additional pre and post-operative rooms in MMUH. Some of these rooms are presently used for out-patient clinics and admin duties so this process is likely to be incremental over the next few months. We are delighted to confirm that the post created and funded by CFI, Post-operative Clinical Nurse Specialist (CNS) has been made permanent and from now on the post will be funded by the Mater Hospital. In partnership with our branches in Galway and Sligo, we also continue to fund posts in their local hospitals. Clinical Genetic Services CFI along with other patient groups recently met with senior staff in the HSE about resourcing and other concerns in relation to Clinical Genetic Services (based in Crumlin). This service plays a vital role in diagnosing, advising treatment and providing genetic counselling, including to CF patients. Following lobbying over many months, 2 key staff are being appointed including a new Professor of Genetic Services. The appointments will be made circa August 2019. Travelling with CFTR Medicines A recent issue that has arisen is the challenges for PWCF travelling abroad for work or extended tourism if they are on a CFTR Drug (Kalydeco, Orkambi, Smykevi). We understand clinicians/hospitals are reluctant to give more than a 3-month supply because of the very high cost of the drugs and fears that the drugs will deteriorate or may be lost. Cystic Fibrosis Ireland will prioritise this issue over the coming months. www.cfireland.ie

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Community Events "Our Baby has Sixty-Five Roses" is the title of a new book just launched by Kildare mums Eilís Moroney and Ruth Cahill to help explain cystic fibrosis (CF) to other children in an ageappropriate way. The book is about helping siblings and relatives better support the person with CF in their lives, while also giving comfort to parents at a very difficult and challenging time. We were very happy to host the launch of the book in Rathmines on June 19th. On May 29th, CFI were delighted to attend the launch of "Seven Summer Stars", an exhibition of art work from a group of young teenagers with CF who took part in a pilot programme led by Helium Arts, in the Waterford Healing Arts Trust centre. The project took place in spring 2019 and saw seven young people with CF aged 12-16 using technology for mediated meet-ups. Over the six online sessions the teenagers took part in a group art journaling experience and had access to professional artist mentors. Separately, the second annual art exhibition for people with CF took place in the CB1 Gallery in Limerick in June. Over fifteen pieces by eight artists were on display. A huge congratulations to all those who took part in the exhibition and to Sara Cross, curator for organising the exhibition once again. It is great to see the CF community using their creativity to help raise awareness and understanding of cystic fibrosis. Hyde family in Australia An Irish family in Australia who emigrated in 2009 are now being threatened with deportation because their son, Darragh (born 3 years ago in Australia) was diagnosed with CF and is considered a ‘treatment burden’. The family are well integrated into Australia – the mother is a Vice Principal and the father is a part-time bus driver and carer. Their appeal failed on 7th May but CFI understands that a final appeal will be considered by the Australian Minister for Home Affairs. A letter written by CFI to support the Hyde family received much media profile in Australia. Transplants CFI is an active member of the Irish Donor Network (IDN) which includes 7 other patient groups including: The Alpha One Foundation, COPD Ireland, Cystinosis Ireland, The Irish Lung Fibrosis Association, Irish Heart and Lung Transplant Association and the Pulmonary Hypertension Unit. The IDN brings together like minded patient groups to campaign for soft opt out organ donation and to provide support in our major transplant centres. We will feature our work with MRCG, IPPOSI, Lung Health Alliance in future editions of Spectrum.

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Spectrum / Summer 2019

Why CFI Works Closely with Other Patient Groups CFI plays an active role in working with other patient groups to address some of the issues that impact on all of us, for example in relation to accessing new and innovative drug therapies. International Research published in April 2019 shows that Ireland performs poorly in respect of: • Speed of Access to new medicines Ireland is 20th out of 27 European countries • The wait time for Irish patients for new medicines Patients in Ireland wait on average 486 days for some medicines. The average wait time across 27 countries was 413 days and Denmark, which has similar population to Ireland, has a wait time of only 146 days • The wait times for new drugs in Ireland are getting longer The average wait time in 2017 in Ireland was 408 days, now its 486 days • Availability of new medicines Ireland ranks 16th out of 27 countries. Just 42% of EMA licensed medicines in 2018 were available in Ireland • Accessing drugs for rare (orphan) diseases Just 29% of new rare disease medicines licensed in 2015-17 are available in Ireland by the end of 2018 Some of the drug therapies that have featured in recent media reports involving significant patient advocacy include, for example: Orkambi (Cystic Fibrosis); Respreeza (Alpha One) Translarna (Duchenne Muscular Dystrophy) Spinraza (Spinal Muscular Atrophy/SMA). Part of the problem for delays or lack of availability of new drugs in Ireland may be related to the way drugs are currently assessed and the resources for assessment. A further issue is the need for more patient and clinical involvement in assessing drugs in Ireland. This involvement is much better in Scotland where we might look to ideas for improving the system. This means that access to and delays with new drugs is a problem for many patient groups and it makes sense that we collaborate through bodies such as the Rare Disease Task Force; Medical Research Charities Group (MRCG) and the Irish Platform for Patient Organisations, Science & Industry (IPPOSI) to address these problems jointly. Thank You Erin Many thanks to Erin Sugrue who is leaving CFI after 5 years as our fantastic regional officer in Limerick working in partnership with the 3 local branches – Tipperary, Limerick and Clare - TLC4CF. We were very lucky to recruit Erin who came with great experience from Pobal, an agency which works on behalf of Government to support communities and local agencies toward achieving social inclusion and development. As part of the work of CFI / TLC4CF in the Mid-West region, Erin contributed to: •

Preparing the liaison meetings between UHL and CFI/TLC4CF

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Helping to coordinate fundraising in the region

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Supporting many of the meetings that led to the Leben building (adult CF unit) in University Hospital Limerick

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Liaising with TLC4CF and CFI

Erin coordinated the CFI / TLC4CF office in Limerick and is a fantastic organiser and coordinator and she is a pleasure to work with. With many interests outside CFI including in the cultural and media sphere, it is in this arena that Erin is seeking to get further involved in the near future. Thanks again Erin, we will miss you. You made a real difference in your work over 5 challenging years to people with CF and their families in the Mid-West region. www.cfireland.ie

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SPOTLIGHT: Gastronintestinal Issues and CF

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ystic fibrosis is widely known as a respiratory condition, however it is actually a multi-organ disease which affects the lungs, pancreas, digestive tract, gastrointestinal system and the reproductive system.

According to the 2018 CFRI Annual Report, over 90% of people with cystic fibrosis in Ireland experience gastrointestinal issues such as gallbladder disease, distal intestinal obstruction syndrome, gastro-esophageal reflux disease, constipation, GI bleeds, peptic ulcers and pancreatitis.

"The first step in solving a problem, is recognising that one does exist." As an organisation, CF Ireland are committed to increasing awareness of all aspects of cystic fibrosis. We will continue to advocate for better understanding of the impact cystic fibrosis has on the gastrointestinal system and improved integration of gastroenterology in CF care. We receive a lot of feedback from members requesting more information about the gastrointestinal issues associated with CF. We are grateful that two members of the CF Community have chosen to share their own experiences with us. As this is a sensitive and personal subject, their experiences are being published anonymously. We hope that these personal stories will help individuals in the CF Community recognise that they are not alone, but also help start the process of viewing cystic fibrosis as the multi-organ condition it is.

A Parent's Perspective Tuesday 23rd April 2013 we sat in Crumlin hospital waiting to see the Gastroenterology Consultant. We had been referred by a locum in our GP surgery after a long period of my son suffering ongoing abnormal bowel movements and tummy pain. We were told it was toddler diarrhoea and that he would grow out of it, but as he got older he would say his tummy was sore most days and I knew something wasn't right. I booked another appointment with his GP and there was a locum Doctor covering that day. She took a good look over him and I told her I felt there was something wrong. She had a friend who worked in the gastro department in Crumlin and made a phone call. We were referred for an appointment and had to drop a sample down to the hospital that day. I was relieved it was getting looked into so we arranged the sample straight away. A week later as I was driving home from work, I pulled over to take a call from the GP surgery. The Doctor explained the sample was very abnormal and they wanted to see us in Crumlin the following week. I was so upset as she could not tell me anything more. I was just lucky that we saw that Doctor on that day and am forever grateful to her.

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Spectrum / Summer 2019

We went to the appointment and the nurse took some information about my son's diet, explaining the pains and bowel movements. He had a great diet and ate most foods, but he could have up to 10 bowel movements in one day. He had chronic tummy pain every single day. He was very tired and pale and never slept very well. The consultant came in and immediately asked if anyone in the family had cystic fibrosis, we had just lost my husband's friend to CF the year before so we were really shocked and not expecting to hear those words as I always thought CF was lung disease. He arranged a sweat test for the following day and those 24 hours were an agonising wait. 24th April we arrived back in to Crumlin hospital for the sweat test, not knowing what to expect. The test was really easy and not distressing for my son who was only three at the time. One of the nurses told me she would phone us later that day with the results. It was an awful wait but we got the phone call that evening confirming a diagnosis of cystic fibrosis. We were heartbroken. I will never forget that day. Four days later we met the whole team and the wonderful Consultant, Dr Cox. They answered all our questions, but most of it was a blur to be honest we were still in shock. We had to change up our routine and teach him how to take all these new medications and make sure they were given correctly. He was a dream, he took to everything so well and was swallowing capsules within a couple of weeks! My son was in playschool at the time he was diagnosed and was settled there and really enjoyed it. He went back to school a few weeks after being diagnosed and when I had a chat with the manager, I found out that another member of staff had CF. I remember being told in Crumlin hospital that he was not allowed mix with anyone else with Cystic Fibrosis and I panicked. His teacher who had it was absolutely amazing, swapping his days so that my son could still attend a couple of days a week and gave me some information packs from CF Ireland. He was so nice about it and we were so thankful to him that we did not have to change playschools as my son had settled and made lots of friends. However, he got on so well with the teacher and really missed having him around. It was really sad but we had to stick by the rules to keep everyone safe. They were happy to give him his Creon tablets at lunchtime and encourage hand hygiene. It was a long road getting the dosage right and his bowel movements settled, but the constant stomach pain went on for many more years and unfortunately is ongoing. He has had endoscopes, colonoscopy, bronchoscopy, coeliac testing, all of which came back fine. He has learned how to cope with the pain and is a very happy young boy. He never complains and takes all the advice he gets from his team. He has seen the psychologist on the CF team for some coping strategies. I find it hard to get answers and nobody is sure why exactly he is suffering with his stomach so much. His lung function is great and he has been well in relation to his lungs. People always associate CF with the lungs, but it is so much more. I never found much CF gastro related information when researching on google etc. about stomach pain, it mentions frequent bulky stools, but not to the extent we had experienced. He has a fantastic teacher who understands his needs. He takes his medication during school and is allowed toilet breaks without asking. His friends in school always look out for him and will sit with him in yard if he is having a bad day with his pains. It never affects his confidence. He deals with it all really well. He is such a happy, polite, outgoing young boy and makes the most of every single day. He makes us so proud and we are so grateful to his team in Crumlin who look after him so well.

www.cfireland.ie

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A Personal Experience Back in 2014 I spent my summer off college in Chicago, it was so fun. I was waitressing part-time, going to the beach and exploring the city! I was also training for the New York City marathon. My health was good, my CF was behaving itself. But on my last week there I became very unwell – terrible headaches, dizziness and nausea. I wasn’t sure what the problem was, I had to spend the time in bed. I also didn’t like the thought of the flight home when being so unwell. Once back in Ireland I rested up for a while and felt OK again. I took part in the marathon that November and was delighted with the achievement. But I kept getting the dizziness and nausea. I was so sick that Christmas, having to spend most of the time in bed. Finally the doctor sent me for an x-ray of my abdomen and it showed I was all blocked up – this was strange as I had been having bowel movements all along. I had to go home and take a combination of laxatives to try sort it out. It was a very slow process, the doctors told me to have patience that it would take time for the medication to work. I have always had bowel problems due to my CF, but this was different, it was a lot worse. I was sent for numerous tests, scopes and of course tried out different diets. The special diets didn’t make any difference and the tests didn’t show up anything unusual. As well as the dizziness and nausea, I was now having problems with a distended stomach and terrible pains. I was referred to the gastro surgical team in Beaumont, they were great, so nice and understanding to how difficult it all was. I felt the CF team never had the interest or understanding with my gastro problems – their answer to it was always that they only deal with respiratory issues. At this stage I was very unwell, I had to do my college work from home. I ate only plain food, I had lost a lot of weight and my brain function wasn’t great. I spent most of my time at home drinking Movicol! My routine was 12 Movicol throughout the day and 2 dulcolax tablets at night – I tried Lactulose as well but it kept fermenting in my stomach causing it to be extra distended and painful. The surgical team say my gastro issues are to do with producing too much mucus, causing mucus plugs, which are stopping things being able to work. After 2 years of trying out numerous combinations of laxatives and medications for my bowel nothing was working, so the surgical team said it was time for me to get a stoma (an ileostomy). I knew this was something I wanted to put off for as long as possible, I asked them could I wait until it was completely necessary – my thoughts were that I’d prefer to feel sick than to have to deal with that just yet! So I was referred to the constipation nurse – her label actually read ‘constipation nurse’ which I found so funny! The constipation nurse was a great help, she discussed alternative treatments – she organised for a nurse to come to my house to show me how to irrigate my bowel (not exactly something I’m dying to talk about but that’s CF life). Nowadays my daily routine is to irritate my bowel every morning using Qufora IrriSedo Cone system, drink 6 Movicol sachets throughout the day and take 2 dulcolax tablets every night. This works for me some days and then sometimes it doesn’t, but the difference is that I can cope with it. I no longer panic when I don’t have a bowel movement for a few weeks, I know if I continue with my meds it will be OK eventually. I also drink a minimum of 3 litres of water a day, walk most days and eat a healthy balanced diet. I hope that in the near future gastro will become more involved in the treatment of CF. Whether that’s better communication between the CF team and gastro surgical team, or ideally each CF Team should include a gastro doctor.

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Spectrum / Summer 2019

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Hospital Hub Noticeboard he summer months are upon us, and even the most pessimistic of us will surely still be holding out a little hope for a return of last year’s glorious sunny summer days.

Whether the summer sun does arrive in Ireland this year, or you’ve hedged your bets and booked a sun holiday, maintaining hydration in hot weather is essential. In CF, high salt losses from sweat can lead to salt levels in the blood becoming too low. This can cause a dulling of the sensation of feeling thirsty and can quickly lead to dehydration if not addressed. To maintain hydration safely in hot weather you therefore need to drink plenty of fluids without waiting to feel thirsty, and replace the salt you lose in sweat by eating salty snacks (like pretzels, salted nuts or popcorn) and by adding a pinch of salt to food and drinks. If you are heading somewhere very hot, exercising in hot conditions, or if you perspire a lot normally speak to your CF team about getting some salt supplements on prescription to take away with you. Roisin Fitzpatrick, Senior Dietician, St. Vincent's University Hospital

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LC4CF and the CF community of the mid-west region were delighted to present a ‘special contribution to CF’ award to Peig Harnett, to mark her retirement from nursing. Peig has been caring for people with CF for over 30 years and we have been honoured to have her as the Clinical Nurse Specialist in Limerick. Over the past three decades she has been trusted and loved by her patients; relied upon by their parents; esteemed by her colleagues and feared by hospital management! She worked incredibly hard to get the new CF adult unit in Limerick running properly and she has always undertaken her role with dignity, professionalism, efficiency and most importantly compassion.

According to her patients "she cared enough to sit on the edge of our bed to talk fashion, relationships or give us comfort in our darkest hours". Peig was always contactable by mobile and took calls at all hours of the day and night, regardless of whether it was the weekend or she was on holidays. A gesture that is rare and valued by all for whom she was their first, last and only resort. We congratulate Peig on her award and her well deserved retirement.

www.cfireland.ie

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Member Services

Exercise Grant Round 2 opens Monday 9th September Application form available on CFI website from mid August Round will close when funds are exhausted Apply early to avoid disappointment

Did you know? If you are post-transplant PWCF you may be entitled to re-imbursement of the cost of 50+ SPF Sunscreen. Talk to your post-transplant nurse, or call us here at CFI for more information!

Heading away in 2019? Got Questions?

Travel Insurance

Taking medications abroad

Traveling with Oxygen

CF Clinics abroad

Talking to your CF team

Call Rory Tallon on 087 932 3930 or Caroline Heffernan on 087 932 3933 Enjoy your holiday!

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Spectrum / Summer 2019

Seven Summer Stars

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even Summer Stars are a group of young teenagers with cystic fibrosis who have been making art and connecting creatively as part of a pilot online art programme led by Helium Arts, the national children’s arts and health organisation, in partnership with Cystic Fibrosis Ireland, the Irish Museum of Modern Art and Waterford Healing Arts Trust. The first phase of the project took place in spring 2019 with seven young people aged 12-16. Using digital tools and online mediated meet-ups along with access to professional artist mentors, the project supported a group art journaling experience over six online sessions. Artist Rachel Tynan, who has been collaborating with young people on Helium’s creative projects since 2012, is the lead artist. An Cosán Virtual Community College provided the online technology and moderated the online sessions. So how did it all work? The young people connected via a virtual classroom. A multiple-screen format meant everyone could see each other during group discussions and the screen could also be reconfigured to just show one person; for example, when Rachel was sharing PDFs of artwork and journaling processes. Participants typed ideas and suggestions into a live messaging box and breakout sessions enabled smaller group conversations which were a great opportunity for participants to get to know each other better.

Artist Rachel Tynan sharing examples of her artwork using the virtual classroom.

A very cool Ladibug document camera allowed for high quality art demonstrations. Before each workshop, the young people could take part in an online artist ‘drop-in’ session with Rachel to talk about what they were working on and how they might progress their ideas.

“I thought it was really cool to meet other people with CF and have that connection. And I like how we’re sort of all in the same room at the same time and that’s comforting.” Each workshop was recorded for anyone who couldn’t make a session. The recordings were stored on Google Classroom, which also became a space to connect between workshops. Participants uploaded artwork and images from their project journals here, shared photos that ‘tell a story’ as part of a project led by guest filmmaker Linda Curtin, and watched art tutorial videos created by Rachel. One of the videos demonstrated how to create the fairy light roses seen in the image above.

“I think it’s really fun and I like how we can all find different ways of being creative” www.cfireland.ie

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To add another dimension of excitement between sessions, Rachel posted the young people art packages. Before the project started, each participant received an art pack in the post and it was great to see everyone using the materials in their journaling work. The young people also received surprise packages (‘Do not open …yet!’), postcards of artwork from the National Gallery which tied into themes they were exploring, and mugs that they could decorate. Fostering connections between young people with CF in an online creative environment was one of the main aims of the project. Participants explored connections they shared on a visual map, from owning a dog (nearly everybody) to loving chocolate (everybody) to playing the guitar.

Surprise packages posted to participants.

“At the beginning we did that drawing where we connected what we had in common and I think that really just started to connect us. And then as we moved on we kind of became good friends and we haven’t even met each other in real life.” Through a word association game and a digital poll, the young people decided to call themselves Seven Summer Stars and designed their own group logos. Rachel combined their design suggestions into one logo and everyone received their own Seven Summer Stars t-shirt in the post. The project also sought to increase access to the arts for young people with CF. Participants explored artworks from IMMA’s Freud Project ‘Gaze’ exhibition via a pre-recorded gallery experience, led by curator Mark Maguire from IMMA’s Engagement and Learning Department with artist Rachel Tynan. Artists with CF who use their life experiences as inspiration for their work was another area of exploration and Mayo-based artist Katie Moore proved to be a big hit!

“I actually have realized I really like drawing things that relate to my CF , that’s because of you guys!” The group were excited by the idea of sharing their artwork at the end of the project and a public showcase took place at Waterford Healing Arts Trust on May 29th. Participants were brought on a virtual tour and took part in a virtual ‘meet the artist’ session with project partners and showcase attendees where they discussed the inspiration behind their work.

The public showcase at Waterford Healing Arts Trust on May 29th.

“I’m really excited to have a proper showcase and have my art in it. It’s not something you would do every day or something that most people would get [to do].” 10

Spectrum / Summer 2019

“Thank you for such inspiring, insightful and revealing artworks! Love them!” – showcase visitor For a lot of the young artists, their CF experience was a key theme, with purple roses and hues populating their pictures, thorns protruding from a woman’s body, and roots mimicking the double helix. The artwork was positive (“My illness doesn’t define me”); poignant (“Cystic Fibrosis grew roses in my lungs. Yes, they’re beautiful but, I can’t breathe”); and optimistic, with a purple butterfly signalling change and hope.

The artwork created by the Seven Summer Stars

The showcase also included a project film created by Linda Curtin with animator Dave Lawless, featuring some of the young people’s project work and audio reflections, and a response artwork by Rachel inspired by the theme of connections and featuring the young people’s hands waving to each other during the online sessions.

Since the showcase, Seven Summer Stars have had their artworks exhibited at the CFI celebration for the VHI Women's Mini Marathon and the book launch for Our Baby has Sixty-Five Roses. Many thanks to the seven talented young artists for your creativity, imagination and dedication, and showing us that even if we can’t always connect in person we can create amazing connections in other ways.

Left: Guests at the launch of "Our Baby has Sixty-Five Roses" admiring the art work of the Seven Summer Stars. Right: Participants in the One in 1,000 Campaign showcasing the work of the Seven Summer Stars

Join us this Autumn! The next phase of the online art programme will take place this Autumn and is open to young people with cystic fibrosis aged 12-16. The programme is free for participants and no art experience is necessary. If you would like to get involved, register your interest with Samantha Byrne at CFI: sbyrne@cfireland.ie or 01 4962433 The Summer Stars online art programme is supported by The Arts Council, The Ireland Funds, Dublin City Council and The Health Services Credit Union. Helium Arts is an award-winning charity supporting the social and emotional needs of children facing long-term medical conditions through arts-based creative projects in hospital, community and public settings. www.helium.ie www.cfireland.ie

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Book Launch

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’ve always wanted to write a book. As the saying goes, ‘There’s a book in us all.’ I’ve toyed with short stories before. I’m a primary school teacher, and I’ve often used unassuming classes as guinea pigs as I’ve read aloud extracts from different stories that I’ve dabbled with. Children are the harshest of critics so I could never ask for better feedback! My beautiful daughter Aibhín was born last October. My husband and I were so excited to meet her, and thankfully she only kept us waiting for one week extra after her due date! Our family and friends celebrated Aibhín’s arrival with visits, dinners, presents and lots of new baby snuggles. Aibhín is a very calm and happy little lady. She never complained throughout her first three weeks. She drank her milk, produced dirty nappies and slept, just like any other baby. Except for one big difference to her peers –she drank a lot of milk and produced a lot of dirty nappies too. Being my first child, I didn’t think that her feeding and toilet habits were anything out of the ordinary. But all that changed when I received a phone call from Tallaght Hospital and the dutiful nurse kindly and sensitively asked me to bring my daughter in to meet the doctors the following morning, amid their concerns for Aibhín and the possibility that she might have CF. And she did. Anyone who has been on that journey will know how painfully long those 24 hours are between getting that fateful phone call and receiving your child’s diagnosis. All I wanted was to go to the hospital there and then and find out one way or another. The wait was excruciating. My husband and I feel so privileged that Aibhín is under the care of the unbelievably dedicated CF staff of Tallaght Hospital. It was strange to think that this team of people had such important knowledge about our daughter, that we as her parents were completely unaware of it, and that they were waiting to take us under their wing, and mind us and guide us. And that’s just what they did and continue to do. When I first met Ruth, I didn’t know that she was a storybook illustrator. Fate definitely played a part in our friendship and business endeavours, and our book was born! I wrote this storybook, as I found myself overwhelmed with the words ‘Cystic Fibrosis’ in the initial weeks and months that followed Aibhín’s diagnosis. It crept up everywhere that I looked, in the drains of the sink in the bathroom and kitchen, in the exercise ball that I thought I’d be rid of after my pregnancy, in the baby weaning spoons that appeared in our home all too soon, in the multivit that destroyed all of my beautiful bibs for Aibhín! This story popped into my head one day when I found myself thinking about what Aibhín would say if she had a voice. She would probably have told us all to stop being upset, that she was and is doing mighty, and that she has so much enthusiasm and excitement for life and she wants to share it with everyone that she meets! I wrote this story to remind myself to see the bigger picture - which is that anyone, anyone at all with a medical condition, or any type of condition, is not and should not be defined by it. We all have something in our lives, nobody is perfect. I wrote this book to really encourage families, friends and society to detach a diagnosis from an individual and to recognise that everyone on this planet has so much to offer, achieve, celebrate, share, see, smell, experience, learn…the list is endless. Thanks to the inclusion of CF testing in the heel prick test over the past seven years, I also believe that this book will be of benefit to families who have other young children in the home, be they siblings, cousins or neighbours.

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The words and pictures gently explain a day in the life of a baby with CF, and subtly refer to ways in which the entire family can get on board with physiotherapy and essential attention to germ control through hand washing etc.

"Our Baby has Sixty-Five Roses" is available to buy on www.cfireland.ie

Ruth’s drawings are sensitive, humorous and heartwarming. They perfectly compliment the words of the story. Though these words are the story of my family, I firmly believe that my words are the story of many others too. And I hope that our book, ‘Our Baby has Sixty-Five Roses’ will help others to take the paralyzing fear out of a CF diagnosis for a newborn baby. Love them, and enjoy them and go easy on yourself too.

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Eilís Moroney

ince I was a child I loved to draw and paint, endlessly doodling in my homework journal instead of studying. My passion for art brought me to study Graphic Design in the Dublin Institute of Technology followed by a Postgraduate Diploma in Advertising. In 2009, following the birth of my first child Harry, and his diagnosis of CF, I established Duck Blue, an illustration and design studio based in Maynooth, Co. Kildare. I work at my studio part time when my children are at school for a few hours a day. I really enjoy creating bespoke cards and illustrations for all occasions from birth announcements to keepsake wedding cards. Every Christmas I produce a range of Christmas Cards that I sell online and at markets. I give 10% of these Christmas proceeds to CF Ireland. Often I get repeat customers at these markets. Last December one such customer came to buy some cards and explained to me how her cousin’s little baby had just been born and diagnosed with CF. Having been in this position 10 years previous I gave this girl my card and said that when her cousin was ready she could call me and we could have a chat if she wanted, especially because we only lived a few miles from each other. And so began a wonderful friendship… On first meeting Eilís in the beautiful Castletown House in Celbridge we chatted about her precious newborn baby, how scary and challenging the CF diagnosis is, the heel prick test, hospitals, family and friendship. We hit it off from the get go. During this meeting Eilís told me that she had written a short story about a newborn baby being diagnosed with CF and asked if I would like to read it and perhaps illustrate the story. I knew instantly that I wanted to be a part of this. The simplicity but honesty of her writing really moved me. The detail of what she wanted portrayed in each drawing was just what I felt was needed and over a 6 week period we worked tirelessly on creating the book that we proudly have today. We really feel that our book, ‘Our Baby has Sixty-Five Roses’ will become an invaluable resource not just for new parents and families, but for nieces and nephews, pre-schools and primary schools, and of course hospitals too. I know from my own personal experience, that for the first few years after my son’s CF diagnosis, I felt very lost and I yearned for the life that I dreamed my child would have, and of course the life that we as a family would have. Both Eilís and myself have experienced our firstborn child being given a CF diagnosis. We both agree that being a parent for the first time is hard enough without a diagnosis like CF being included. We really hope that families that face the diagnosis going forward can see our book and realise that they are not alone, that their baby does have CF but that they are also their own individuals with so much more to offer, share and celebrate. The future is looking bright… and together we will fight for a better future for our children and all PWCF.

Ruth Cahill www.cfireland.ie

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The launch of "Our Baby has Sixty-Five Roses" took place in the Pot Bellied Pig, Rathmines on June 19th

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KIDS CORNER E

xercise is important for everyone, but especially for people with cystic fibrosis as it helps to clear the

mucus from the lungs. It is recommended that everyone should do at least 30 minutes of exercise every day. For people with CF exercise is part of their everyday routine. When we do something all the time it can sometimes become boring, but exercise doesn't have to be boring! Whether you are alone or with a group of your friends there are plenty of activities that you can do to ensure you are exercising and clearing mucus off your lungs, but that are fun such as playing with your dog, learning to play a wind instrument, flying a kite or a water balloon fight! Since school is finished for the

CFI would like to congratulate PWCF Evan Nealon, aged 9 and his brothers Dylan (12) and Alex (9) who were recognised for their outstanding efforts with the Overall Super Junior Award at the UNESCO Young Environmentalist Awards. Since they started "Evan, Alex and Dylan’s Tralee Canal Clean Up" over 500 bags of rubbish have been removed from the Tralee Canal and surrounding areas.

summer there is plenty of time to try out new ways to exercise!

Well done boys!

Have fun!

Can you find all the summer sports in the wordsearch below? MAZE PUZZLE SOLUTION Spring 2019

Badminton Rollerskating Surfing

www.cfireland.ie

Cricket Skateboarding Swimming

Cycling Skipping Tennis

Golf Snorkeling Volleyball

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YOUTH REACH C

ystic Fibrosis is widely known as a respiratory condition, but in fact it affects several organs in the body from the lungs to the digestive system, pancreas to the reproduction system. While the impact of CF can vary from person to person, with so many areas of the body impacted by CF it is no wonder that a lot of people with CF struggle with their self-esteem and body image.

My stomac h is swollen ! e ls e e on ry ve e ! ' sho rte r than Im I don't have any muscle, even though I exercise regularly!

However, you are not alone. Body confidence is something lots of people struggle with regardless if they have a medical condition or not. Almost everyone has a part of their body they are unhappy with. However, we should try and focus on the positives rather than the negatives! Sometimes we can't see the beauty that is right in front of us! What do you like most about your body? List out what you think are your three best features: 1) 2) 3) Then ask your parents, your best friend, your siblings etc. While we listen to the negative thoughts about our own bodies, we very rarely take on board the positives. Hearing someone else tell you what they love about you or writing down your own positive comments to read back can help when we are struggling with our confidence. This is something you can do for others too! Focus on what your body can DO! There is much more to our bodies than our looks. With CF impacting various organs it is easy to focus on what our body can't do in comparison to others, but if we try to focus on the positives and not the negatives we will see there is a lot our body can do that we don't acknowledge. What is your biggest accomplishment to date? Standing in front of a class to deliver a presentation? Your personal best in a park run? An A on an exam? Think about what you achieved, how you felt, both physically and emotionally. You achieved that. You, not CF. CF is a part of you, it does not define you, so it shouldn't define how you view yourself.

Remember you don't need a perfect body to have body confidence and a positive body image.

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Congratulations to Benat Broderick, PWCF & CF Ambassador, aged 15, who was recently awarded the Pramerica Spirit of Community Award in the Junior Section. Benat was honoured for his volunteer work with Cystic Fibrosis Ireland and in recognition of his efforts to help raise awareness of CF.

Spectrum / Summer 2019

Annual Conference The 2019 CFI Annual Conference and Annual General Meeting took place in the Mount Wolseley Hotel in Tullow, Co. Carlow (29-31 March). CFI CEO, Philip Watt, opened the three-day event by drawing conference delegates’ attention to the incredible evolution in CF care which has occurred since he became CEO in 2009. This has included the development of a network of specialised CF hospital centres, improved transplant rates, the 2011 introduction of newborn screening for CF and the approval of a range of innovative and groundbreaking drug therapies which address the root cause of CF including Kalydeco® (2013), Orkambi® (2017) and Symkevi® (2019). Dr Laura Kirwan of the CF patient registry outlined the dramatic impact these improvements have had on the quality of life for people living with CF in Ireland: Mean lung function, BMI and median age of survival have all seen marked improvement over the last ten years. Dr Michelle Murray, Consultant Transplant Respiratory Physician at the Mater Misercordiae University Hospital, highlighted that, as a result of these improvements, many patients are now not requiring transplant until well into adulthood. She went on to say that transplant is not for everybody, transplant patients are a very unique group of CF patients and they face their own particular challenges and have their own specialised needs. Ireland carried out its first single lung transplant in 2005, its first double lung transplant in 2006 and opened its first Adult Lung Transplant facility in the Mater Hospital in 2011. PhD researcher, Michael Twomey is a 47 year old person with CF who is developing a patient management system that enables people with CF to capture their medical or health history accurately between appointments and record key information communicated by clinicians. He published a Medical Appointment Checklist Booklet in January 2019 which has subsequently been distributed to every CF patient and carer in Ireland and won a Gartner award for innovation. Professor Barry Plant, Director of the Adult CF Centre at Cork University Hospital, highlighted the importance of long term, real world data on Kalydeco® and Orkambi®, in addition to the short-term benefits measured in clinical trials. These drugs have significant “slow-burn” benefits which have the potential to dramatically alter the course of this condition. But then patients with CF (like Bevin Murphy, Jillian McNulty and Caroline Heffernan) and parents of children with CF (like Bernie Martin, mother of Eva Rose Martin) are well used to taking the long view. Their presentations, covering the past 10 years, included stories of worsening health as well as the health advances, goals and achievements that kept them going. Their presentations also included ideas and strategies on how to cope with the added responsibilities of either having CF or caring for a child with CF which include developing a routine and prioritising, enlisting the help of others, talking with other individuals and families affected by CF and consulting with the CF care team. Nicola Hurley, PhD researcher at Dublin City University (DCU), returned to this theme in her presentation. Evidence suggests that regular exercise training improves cardiovascular fitness (strength of your heart and lungs), which is directly associated with an increased lifespan for people with CF. Bernie Martin, summed up the conference well, in her presentation, when she said that, notwithstanding the great medical advances, the lives of families affected by CF remains one of ups and downs with many conflicting emotions and slow incremental progress.

Daniel Costigan, CFI

www.cfireland.ie

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CFI Conference

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2019 Gallery

www.cfireland.ie

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ECFS Conference CFI would like to thank the following for this report and their participation in European CF Conference in Liverpool (2019): Philip Watt; Daniel Costigan; Carolyn Cavey; Caroline Heffernan and Nuala McAuley. Please note that this report is based on our understanding of the research sessions attended, but are not of course a substitute for reading the original research. With significant improvements in cystic fibrosis (CF) care and services over the past decade, CF remains one of medicine’s positive news stories, but there are many outstanding knowledge gaps and unmet clinical needs where research plays a key role. That was the message of the 42nd European Cystic Fibrosis Society (ECFS) Conference which took place from June 5-8, 2019 at the Arena and Convention Centre in Liverpool, England. Over 2,500 doctors, biomedical scientists, healthcare professionals and patient groups from 58 countries were in attendance. There was a strong focus on the real lived experiences of patients, which was evident from the opening ceremony that saw a performance from the talented dancer, Thomas Oakley (who also has CF) and a ‘virtual CF choir’ which included both a Beatles song and ‘Take me to Church’ by Hozier. (Since 3 of the Beatles have direct Irish connections and Hozier is from Dun Laoghaire, there was a definite Irish flavour to the opening ceremony!) This was also the occasion to honour Professor Stuart Elborn from Belfast for his lifelong contribution to CF care and research.

CFTR Modulator Treatments There was of course also a great deal of discussion at the conference about cystic fibrosis transmembrane conductance regulator (CFTR) modulators. This spanned from new information on the positive effects of ivacaftor on GI complications to updates on the new Vertex triple combination drugs. Professor Barry Plant contended that CFTR drugs have clinical and quality of life benefits beyond lung function. Lung function is an important (but not the only or best) sign that a CFTR modulator is having a disease modifying effect. Focusing solely on lung function undervalues and under-predicts the long-term systemic benefits that patients with CF derive from CFTR drugs.

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Vertex presented on its VX-445 (elexacaftor) triple regime therapy which it is going to apply for regulatory approval of in the US and Europe over the third and fourth quarters of 2019. This triple combination is made up of VX-445, a CFTR corrector, added to tezacaftor (VX-661) and Kalydeco (ivacaftor), and it is intended for CF patients with two copies of the F508del mutation or one copy of that mutation and another minimal function mutation. Vertex has been looking at the potential of VX-445 and VX-659 triple combinations and has now decided to advance the VX-445 combination regimen for global approvals, based on final positive data from two Phase III trials - A 24 week study and a 4 week study. In the trial of the F508del / minimal function mutation population there were 403 patients (12 and over) who received either the VX-445 triple combination or a placebo during a period of up to 24 weeks. Final trial data showed that patients receiving the combination therapy had a mean improvement in lung function of 14.3 percentage points, from the study start through week 24 and a 63% reduction in the annual rate of pulmonary exacerbations when compared to placebo. Stop Mutations Eloxx Pharmaceuticals presented on novel ways to increase CFTR, not addressed by currently licensed drugs. They are developing drugs to improve ‘read through’ for nonsense or ‘stop mutations’. In these mutations, only part of the CFTR protein is made, as there is a ‘stop reading’ signal in the wrong place on the gene. Enterprise Therapeutics also discussed their programme of drug development which targets alternative proteins in the lungs. The hope would be that their new treatments could work with anyone with CF, irrespective of their genotype. 30th anniversary of CFTR Gene Discovery The CFTR gene was first discovered in 1989, this is the 30 year anniversary and so much has been discovered since. We are now in the era of precision medicine, which was a key focus of a satellite seminar organised by Vertex during the ECFS conference. There was great excitement around at this seminar about the Vertex triple combination drug, as explained by Dr Fred Van Goor of San Diego, USA, which he termed as ‘the next generation CFTR corrector’ and which involves three distinct and complementary mechanisms of action. These are: The Amplifier working at CFTR at ribosomal level, the Corrector working on CFTR mid-cell and the Potentiator improving CFTR channel on the cell membrane. This new triple combination drug therapy combines Tezafactor, Ivafactor and the new molecule VX-445. It appears in trials that PWCF homozygous for F508 del had a decrease of 45.1 mol/L of salt in sweat and an increase of 10% FEV. This drug is designed to assist not only those who are homozygous F508 del but also the heterozygous which have only one copy of F508 del and another gene with a minimum function. Indeed that is good news as still a significant group have not been eligible for modulators. Gene Editing The relatively new concept of gene editing was discussed by Patrick Harrison of University College Cork. He explained simply that all mutations are just 'spelling mistakes'. They require to be cut out of DNA (using CRISPR technique), copy donor sequence, paste and restore function. The method can be used for somatic cells (body), but more effective if in germ cells. There is interest in gene editing for Leukaemia, Huntington's disease and Muscular Dystrophy. The vision by these Scientists is that all three methods - Modulators, Editing and Gene therapy will work synergistically in long term fixing CF - watch this space for future discussion. www.cfireland.ie

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Gene Therapy Meanwhile new developments in gene therapy have emerged as presented by Eric Alton, London, UK. We all thought use of phage (type of virus) as a delivery system to our lungs with CFTR genes was not going to happen, as many efforts had no success due to the continuous need to replace the phage as expelled by the lungs. However scientists have merged two types of phage, the Sendai Virus capsule which can attach to respiratory epithelial cell receptors and the Lentivirus DNA which is lysogenic (will combine with our cell DNA) . This means the gene for CFTR enters the lung epithelial cells and has a long duration of expression for up to 18 months and longer if it enters the basal cells. There is a massive interest in this technique of using the qualities of two viruses. The idea is being explored for delivering correction genes for other genetic diseases too. Standard “Symptomatic” Treatments While there was understandably a great deal of excitement around the development of more new groundbreaking medications which address the underlying causes of CF, there were also many presentations at the conference on the need for more effective medical therapies to further prevent disease progression. There were over 700 presentations at the conference. Some of these were intended to increase the depth of knowledge in areas of CF research while others examined topics where researchers and clinicians know that more information is needed. These include better treatments to deal with infection, inflammation, irreversible lung disease and the extra-pulmonary complications of CF. More work will be required to address a host of extrapulmonary complications including sinus disease, CF-related diabetes (CFRD), osteoporosis, anxiety and depression all of which are becoming more and more common and as people with CF live longer and more fulfilled lives. It is still not clear, for example, what causes people with CF to develop CFRD. At the conference there were updates from researchers working in this area. A better understanding could lead to better management and even preventing CFRD from developing at all, if treated early enough. Additionally, while we know that the lungs of CF patients are affected by thick sticky mucus, chronic infection and inflammation, we are not sure how all these elements are linked together. During the conference there were a series of talks looking at mucus, how it is made, how its production is affected by CF and the effects it had on inflammation. A more detailed understanding of how the formation of mucus goes wrong in the lungs of people with CF will help researchers to improve lung clearance in the future. Professor Winstanley of Liverpool University also gave an update on the genetic differences and the evolution of different strains of Pseudomonas aeruginosa. Understanding how Pseudomonas develops could give doctors the opportunity to treat the bug earlier, when it is more responsive to antibiotics. Knowing which strain or ‘family’ of Pseudomonas different people with CF have can also give important information on effectively managing crossinfection in the clinic. Poverty, Social Exclusion and CF David Taylor Robinson of Liverpool University presented on how the socioeconomic status of patients can have a profound impact on their health status. His research showed that children with CF from the most disadvantaged areas in the UK have lower weight, height and body mass index (BMI) in the first years of life after diagnosis and are more likely to have chronic Pseudomonas aeruginosa infection and lower lung function than children with CF from less disadvantaged areas. In addition to social determinants of health status, Dr Abi Jackson of the CF Registry of Ireland presented on the relationship between better health outcomes and health literacy. Health literacy is defined as an individual’s ability to access, understand and evaluate information about their own health and act on their understanding of health. Nutrition/Anti-Oxidants Eat lots of Strawberries! This was the message from Oana Ciofu from the University of Copenhagen. Behind this

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message is the fact that they are full of Ascorbic Acid a part of the Vitamin C molecule. This wonderful water soluble vitamin is an Anti-Oxidant and so vital for reducing oxidative stress which causes chronic Inflammation and damage to tissues. Anti-Oxidant deficiency is a problem for PWCF especially due to malabsorption of fat soluble vitamins A and E, and Interestingly a low amount of Glutathione. This molecule is so important that it reduces intracellular oxidative stress, especially in the lungs and liver. This can be inhanced by taking N-acetyl-cysteine or NAC supplement which is an oral supplement that is a precursor of glutathione. This would of course need to be discussed with a dietician, but the more natural method is through diet, there is research into whey protein and apparently good old fashioned bone broth (or stock made from bones) is a wonderful source. A good reason to have Granny's Chicken Soup perhaps! Essential Fatty Acids: Brigitte Strandvik of the Karolinska Institut Stockholm, emphasised the need for Linoleic Acid an essential fatty acid in the diet. People without CF may require 1-2% but in PWCF it was 10-15% due to absorption issues. Linoleic Acid is a substrate for Arachidonic Acid and many lipid mediators, it is needed for synthesis of EPA and DHA, needed for metabolic and signalling processes. It is required for the pathways to produce the phospholipids in the cell and mitochondrial membranes which are 3D structures as seen in all Science and Biology books. A deficiency means that cell membrane structure is weakened which naturally affects the tissues in organs such as the Liver and Pancreas, more inflammation in the intestines and also a decrease in energy. In rats, Linoleic Acid has been seen to stimulate insulin and perhaps decrease steatosis in the Liver. So in a practical way, where do we find this Essential Fatty Acid? Rapeseed Oil and Evening primrose Oil for a start! Conclusion: Living with Uncertainty and Retaining Hope for the Future While unanswered questions may provoke curiosity for CF scientists and clinicians, families affected by CF must bear the pain and confusion of not knowing exactly what new treatments are coming down the line for loved ones. But, as we saw with the CF Community Afternoon (where eminent CF researchers gave a recap of the research highlights of the conference to the CF community), the CF community are resilient. They understand the lengthy and often frustrating process of clinical research and they still retain hope for the future of CF research and medicine. This will continue as long as the CF community are kept at the heart of CF research and the potential importance of the unanswered questions highlighted to them.

www.cfireland.ie

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RESEARCH

Phages that attack bacteria could be used as a therapy against infections in CF A recent case report of a patient with CF indicates that phages (viruses that naturally infect and kill bacteria) could be used as a potential therapy against infections which are caused by antibiotic-resistant bacteria. The study, “Engineered bacteriophages for treatment of a patient with a disseminated drug-resistant Mycobacterium abscessus,” was published in the Journal Nature Medicine. CF patients often have long-lasting bacterial infections in their airways and sometimes these infections are resistant to antibiotics which makes them difficult to manage. Antibiotic-resistant bacteria pose a particular risk to patients undergoing lung transplant, because they can flare up during the period of immunosuppression required as part of the transplant procedure. This case report relates to a 15 year old girl with CF who had a flare up caused by antibiotic-resistant bacteria after lung transplant and who was treated successfully with a new therapeutic approach based on phages. The patient had recurring infections of Mycobacterium bacteria for eight years and less than a third of normal lung function by the time she received a double lung transplant at Great Ormond Street Hospital in London. A number of weeks post-transplant, the doctors noted that the surgical wound was red. In addition, the patient demonstrated signs of infection and developed bacterial nodules in her arms and legs. The infection was unresponsive to traditional antibiotics. Phages were then considered as a potential treatment to resolve the infections. The doctors sent a sample of the bacteria infecting the girl to Graham Hatfull, PhD, a Howard Hughes Medical Institute professor at the University of Pittsburgh, who runs a programme called SEA-PHAGES, which is aimed at collecting, storing and studying different phages found in the environment. Hatfull’s team tested different phages from the collection to see if one, or some combination of them, could specifically target the bacteria infecting the patient. They discovered three phages that showed some specific antibacterial activity. They prepared a “phage cocktail” out of these three. They edited the genome of two of the phages to enhance their antibacterial properties. They also removed a gene which allows viruses to reproduce inside bacteria without causing any harm. Without this gene, phages are able to break the bacterial cells down and destroy them. Hatfull sent the “phage cocktail” to London, where it was administered intravenously to the patient twice daily for six months. After a month of the phage-based treatment, the surgical wound started to heal. After six weeks the patient’s liver and pulmonary function improved and after six months almost all of the skin nodules had disappeared. No harmful side effects or immune response against the treatment were observed. There were also no signs of bacteria resistant to the phage cocktail.

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New method to work out best PERT dose for young CF patients shows potential in pilot study A new method to work out the best dose of enzyme supplements for pancreatic enzyme replacement therapy (PERT) based on different meals is showing potential in young CF patients, according to a pilot study. The study “Clinical validation of an evidence-based method to adjust Pancreatic Enzyme Replacement Therapy through a prospective interventional study in paediatric patients with Cystic Fibrosis” was published in the journal PLOS ONE. The study indicates that doses do not need to be adjusted on the basis of individual patient differences such as age, gender or BMI. The most important factor in deciding the amount of enzymes to be taken relates to the type of food eaten. Up to 90 percent of patients with CF have to take enzymes before they eat. This is because a thick mucus in the pancreas blocks the discharge of enzymes needed for digesting carbohydrates, fats and proteins in food. To prevent malnutrition, CF patients with pancreatic insufficiency are treated with PERT, which generally consists of oral capsules containing pancreatic enzymes. Patients usually take PERT as a fixed dose per day or adjust it to the type of food they will eat, for instance, upping the dose prior to a fatty meal. This implies that patients “have to decide the dose of enzymatic supplement to take every single time they eat,” the researchers wrote. To address this problem, researchers from several European institutions have joined together to develop MyCyFAPP Project to establish an efficient method of helping CF patients and doctors identify the most appropriate dose of enzyme supplements. To this end, the team created a test to determine a theoretical optimal dose (TOD) of enzyme supplements per food product. Researchers evaluated the variability in fat absorption between CF patients after they had eaten a predefined diet and taken the optimal dose of enzymes determined by the new method. A total of 43 young patients, ages 2 to 18 years, referred to five European CF centres, were evaluated. They were asked to follow a fixed diet for 24 hours and to take the TOD of enzyme supplements for each meal. To evaluate the efficiency of nutrient digestion and absorption, the coefficient of fat absorption (CFA) was determined for each patient. CFA, which is the percentage of absorbed fat in the diet, is calculated by determining the amount of fat lost in the faeces, given the total amount of fat consumed. Results showed that taking the TOD of enzymes calculated by the new tool led to a median CFA of 90%, with little variability between patients. This value is slightly better than the one reported in previous studies and comparable with others without a predefined dose, according to the researchers. Data also confirmed that optimal doses of enzymes varied significantly due to not only the amount of fat, but also the food characteristics, such as the quantities of other nutrients and the type of food matrix. Moreover, the results indicate that the digestion of fat is more dependent on the type of food than on individual patient characteristics. No statistical associations were found between CFA and patients’ age, gender, type of CFTR mutations, or BMI. According to the team, the findings may offer a new perspective on the treatment of pancreatic insufficiency in CF. They also suggested that the newly developed method to adjust PERT “could be implemented in the clinical practice to support clinicians and patients’ decision on the dose of the enzymatic supplement.” To allow patients to self-manage PERT, the researchers have developed a mobile app that calculates the optimal dose of enzymatic supplements based on a database of TODs.

www.cfireland.ie

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Window for more effective treatment of CF lung infections is 2 to 3 years, study suggests Treatment of Pseudomonas aeruginosa lung infections in patients with CF could be more effective within a crucial two to three year timeframe when the bacteria are still susceptible to antibiotics, according to a newly published study. The study, “Evolutionary highways to persistent bacterial infection,” was published in the journal Nature Communications. Genetic changes that have accumulated over a long period of time have contributed to the increased ability of bacteria to adapt to new environments. There is a lot of diversity among bacteria in chronic CF infections, but researchers have previously agreed that increased antibiotic resistance or loss of virulence (the ability to cause disease) will ultimately occur regardless of the particular bacterial population. Although genetic adaptations associated with colonisation of a host have been identified in CF pathogens, few studies have looked at bacterial phenotypes, meaning the cumulative impact of mutations. There have also not been very many studies that have assessed the early stages of infection, when bacterial strains (often P. aeruginosa) become successful pathogens in the lungs of CF patients. To better understand pathogen adaptation to the host, researchers from Denmark used statistical methods that account for the body’s effects on bacterial lineages and assessed their adaptive paths. Volunteers needed A Health Research Board funded study called ‘Patients’ Preferences for Health’ are looking for volunteers. In general people have preferences over being in one state of health over another. This can often be a preference to have good health over bad, or to be in good health for so long and to be in less well health for a shorter period of time. By asking the public in Ireland to make these decisions or trade-offs we can say something about the general preferences of people in Ireland. Preferences gathered in this way can then be used to guide choices around decisions in relation to health. Using preferences from the general public is deemed to be a fair way of deciding on how to assign healthcare resources. However, it is likely that a person’s experience of being in a health state will alter their preferences compared against those who are only imagining being in that state. As such, this work will examine the differences between patients with CF and their valuations of health states, and those of the general population to see if this is actually the case. If you are interested in helping with this study, please contact Dr. Stephen Erskine at erskines@tcd.ie Superbugs Webinar Prof John Moore and Prof Cheri Millar in collaboration with CFI are hosting a series of 4 webinars (commencing in September) to answer questions from our community in relation to Superbugs. If you haven’t already sent in a question there is still time to forward questions to Caroline at cheffernan@cfireland.ie. Dates to be decided closer to the time. The webinars will be at 7.30pm on the given evening. Each webinar will be approximately 1 hour long with the opportunity to interact via online questions and polls. The first topic is on Bacteria, i.e. the different types of bacteria, where particular bacteria is found etc. The second topic is on Real Life Situations, i.e. children’s activities, gardens/play areas and travel etc. CFI will post on social media and send a group text when dates are fixed and give instructions on how to log into the webinar.

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FUNDRAISING: Challenges and Events For further details on any of our fundraising events, visit our website www.cfireland.ie, contact the CFI Fundraising Team on (01) 496 2433 or email fundraising@cfireland.ie Sept 15th: Head2Head Walk Our annual Head2Head Walk from Howth Head to Bray Head takes place on Sunday 15th September. Let’s make this the biggest year yet for this event which is organised brilliantly by the Eastern Branch. Over €70,000 was raised from last year’s event, our biggest year to date, with over 650 people walking on the day. The walk will begin at Howth Dart station at 9.00am and will finish in Bray. There will be lots of food and water along the route with organised food stops at the half way point at Sandymount Strand and at the finish in Bray. Participants have the option of completing the full walk from Howth Head to Bray Head or completing half the walk, either the first half from Howth Head to Sandymount Strand or Sandymount Strand to Bray Head. Registration is now open on www.cfireland.ie with the earlybird registration fee just €15 up to September 1st and €20 after that. Once you register, Cystic Fibrosis Ireland will send you your fundraising pack including your Head2Head T-Shirt. Medals are also provided to all participants on the day. We encourage everyone to play their part by taking a sponsorship card, setting up an Everyday Hero page or doing your own fundraiser and raising whatever you can to continue to help support people with Cystic Fibrosis. For further information please contact Christina on 01 496 2433 or email christina@cfireland.ie.

www.cfireland.ie

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Sept 14th to Sept 19th: Paris2Nice Cycle Our three cyclists Robin Cunningham, Enda Greehy and Owen Kirby will fly to Paris on Friday September 13th and begin cycling the following day, Saturday September 14th. The cycle will finish on the Promenade Des Anglais on Thursday 19th September where they will be greeted by the welcoming team followed by a celebration dinner that night. Sept 20th to Sept 27th: Paddy Kierans’ Memorial Walk in ‘Glorious Greece’ We still have a limited number of spaces left on the CFI International Walk, taking place from September 20th – 27th which will explore unique and beautiful Greece! We urge you to book as soon as possible to avoid disappointment! The Walk caters for various levels of fitness, so that each walker can take part at a pace they are comfortable with. The Walk, which starts in Glyfada and finishes in Athens, will see participants walking an average of 10km each day and taking in sights such as the green hills of Sounio National Park, the islands of Aegina, Poros, and Hydra, the National Park of Mt. Parnitha, the Ancient Footpath Into Delphi, the Temple of Poseidon, and the UNESCO World Heritage site of the Acropolis. The fundraising target for the Walk is €3,150 (per walker) and this will cover all costs including flights, accommodation and meals. For more information on the Cystic Fibrosis International Walk 2019, contact Rachel Byrne at rbyrne@cfireland.ie or call 01 496 2433. Alternatively, you can visit www.cfireland.ie and download the Walk application form. “I love going on the International Walk as I love to see different places, explore the world. I have also made brilliant friends on the walk, who I very much enjoy seeing every year. Of course I love to meet the new faces too! But most of all I enjoy helping to raise funds for Cystic Fibrosis Ireland, and knowing that my fundraising really makes a difference to people with CF in Ireland. I’m looking forward to Greece this year as it’s a beautiful place steeped in history. It’s somewhere different!” – Declan Flynn, lifelong Cystic Fibrosis Ireland International Walker

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Oct 27th: KBC Dublin Marathon Were you one of the lucky ones to get a coveted spot in the now SOLD OUT KBC Dublin Marathon? If not, there will be an extra 2,500 places on sale on Monday 1st July… go go go! For those of you who’ve managed to grab an entry… now that you’ve signed up and started your training, why not consider fundraising for a charity as part of your marathon challenge? We would be delighted if you could fundraise for Cystic Fibrosis Ireland! Contact Rachel at rbyrne@cfireland.ie / call 01 496 2433, and she will send you out your fundraising pack and let you know how to get started on your fundraising. Nov 3rd: TCS New York City Marathon There are still a limited number of guaranteed places available for the 2019 TCS New York City Marathon tour which takes place on Sunday 4th November. A booking deposit of €675 is required immediately in order to secure a place and final tour balances are due by Monday 9th July. The tour package includes: • Guaranteed Race Entry • Direct return flights from Dublin or Shannon to New York • 5 or 4 nights’ accommodation • Airport transfers in New York • Private Coach from the hotel to the race start on Staten Island • A celebration dinner with live music on Monday 5th November • Services of a Sportstravel guide If you are interested in taking part please get in touch with us as soon as possible on 01 496 2433 or email fundraising@cfireland.ie All Year: Kilimanjaro CFI facilitate treks to Kilimanjaro, so if you are thinking of taking part in a trek, we advise that you book your place now for 2019. Skydives A skydive has to be one of the most exciting things you will ever do in your life. Now is the chance to tick this off your bucket list, while raising much needed funds for CFI. Jump from 10,000 feet, free fall for 30 seconds before the parachute opens and take in the views like you’ve never seen them before.

www.cfireland.ie

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Dec 13th: Christmas Jumper Day 4CF On Friday 13th December, we are encouraging as many workplaces as possible nationwide to support Christmas Jumper Day 4CF. Interested in getting involved? All you need to do is… 1. Ask your staff or colleagues to support Christmas Jumper Day 4CF on Friday 13th December. Encourage everyone to wear their most seasonal-looking Christmas Jumper to work in return for a €5 donation to support people with Cystic Fibrosis in Ireland! 2. Bah Humbug donation! If there is a colleague who is not wearing a Christmas Jumper, they have to pay a Bah Humbug donation of €10! It’s all for a great cause. 3. Ask your company if they are willing to match fund the monies raised! 4. On the day, make sure to take lots of photos! Post them to our Facebook page (facebook.com/CysticFibrosisIreland), share them using the hashtag on Twitter or Instagram #CJD4CF and tag us (@cf_ireland), or simply email them to us at fundraising@cfireland.ie so that we can share your fun with everyone else. We can send you on some balloons, bah humbug badges, posters, sponsorship cards or collection buckets if needed – just let us know! 5. After the event you can · Lodge the monies raised: IBAN: IE59 AIBK 9310 7108 5785 93 BIC: AIBKIE2D · Post a cheque to Cystic Fibrosis Ireland, 24 Lower Rathmines Road, Rathmines, Dublin · Donate online at www.cfireland.ie (Please remember to include your company's name as a reference so we know who it’s from) To sign up, just email – fundraising@cfireland.ie or call the CFI Fundraising Team (01) 4962433 to register your workplace and we will help you organise everything for the day!

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Spectrum / Summer 2019

FUNDRAISING: Thank You It is not possible to list all of the Fundraising Events that have been organised since our last edition of Spectrum, but we would like to take this opportunity to thank everyone that has volunteered and raised money on behalf of people with Cystic Fibrosis. Every cent raised helps provide a better quality of life for people with Cystic Fibrosis and please keep up the great work. Here is a short summary of some of the remarkable efforts of our volunteers: 65 Roses Day Cystic Fibrosis Ireland would like to say a huge thank you to all our supporters who supported our national fundraising day - 65 Roses Day on Friday 12th April. So far, well over â‚Ź100,000 has been raised and we would encourage anyone who still has money to lodge to please lodge outstanding funds as soon as possible. We are really grateful to all our volunteers who sold roses across the country in shopping centres, schools and workplaces. Also, we would like to give a special mention to all our CF Branches around the country who get behind 65 Roses Day every year and provide much needed support. 65 Roses Day continues to grow each year and this is made possible with the help of all of our volunteers and branches. We look forward to making 65 Roses Day 2020 even bigger and better again. In addition to raising much needed funds, 65 Roses Day drives awareness of Cystic Fibrosis nationwide with lots of coverage this year across TV, Radio and in Newspapers. We would like to thank our ambassadors, Keelin Shanley, Rosanna Davison, Joe Brolly, Blathnaid Treacy and Dermot Bannon for lending their support to the campaign. We would also like to give a special mention to An Taoiseach, Leo Varadkar for also giving his time to promote 65 Roses Day. Joining the Ambassadors we also had a number of families with children with CF who took part in our photoshoots for which we are very grateful. We also ran a three week radio campaign again this year in the lead up to 65 Roses Day. We would like to thank Malone Group for sponsoring the radio advert and Keelin Shanley for doing the voiceover. A major new element to driving awareness this year was the support of our Corporate Partners with Roses on sale in Spar Stores and Smiths Pharmacy Chemists. This was outstanding support both in terms of raising funds but also raising awareness of 65 Roses Day and Cystic Fibrosis in every village and town across the country. Thank you to everyone involved for this amazing support! www.cfireland.ie

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65 Roses Challenges Well done and thank you to all the individuals, schools and workplaces who organised events as part of our annual 65 Roses Challenge. Due to the large number of events it would not be possible to list them all but they included gym challenges, coffee mornings, bake sales, 65 minute walks and even a 65km cycle to name just a few. Cystic Fibrosis Ireland would like to say a HUGE thank you and congratulations to PWCF Bevin Murphy who spent the whole month of April running / walking for 65 minutes a day in each county in Ireland! Bevin has raised over €6,000 and counting! If you still have funds to lodge from either 65 Roses Day or from a 65 Roses Challenge we would ask that you lodge the funds to the below fundraising account as soon as possible: Fundraising Account: IBAN IE59 AIBK 931071 08578593 BIC: AIBKIE2D

6.5k Remembrance Walks Cystic Fibrosis Ireland would like to say thank you to everyone who participated in the 6.5k Remembrance Walks. As this was our first year for the Remembrance Walks, they were a great success with 15 walks taking place across the country in memory of people lost to CF and plans are already in place for more walks next year. A huge special thank you to everyone who organised the walks in their areas and made it such a special day in memory of their CF Angels. The 6.5k walks have raised over €15,000 to date. Please get in touch with Brendán on brendan@cfireland.ie if you are interested in setting up a 6.5k Remembrance Walk in your area for 65 Roses Day next year.

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Spectrum / Summer 2019

Duleek 10k

Christmas Jumper Day 4 CF 2018

Cystic Fibrosis Ireland would like to say a huge well done to the participants of the Duleek 10k last Sunday, who have raised an incredible €15,000 so far and counting!

Cystic Fibrosis Ireland would like to say a huge thank you to Citibank who raised a fantastic €1,830 during their Christmas Jumper Day 4 CF last December!

Thanks to everyone who participated, with special thanks to Ann Noone, all her family and friends who got stuck in and organised yet again another fantastic race in Duleek!

Please talk to your employers and make sure they sign up for Christmas Jumper Day 4 CF 2019 on our website at www.cfireland.ie or contact the CFI Fundraising Team on (01) 496 2433 for more details

We are aleady looking forward to a great event again next year!

Pictured above, from L-R is Gwen O’Connor (Citibank), Brendán O’Regan (CFI), Jennifer Fisher (Citibank), and Rachel Byrne (CFI).

Scoil Bhríde Athgarvan

Top Oil

A massive thank you to Ms Cosgrove’s 6th Class at Scoil Bhríde Athgarvan for raising an incredible €2,320 for their one day sponsored silence! Rachel went down to Athgarvan to accept the cheque and thank the class. They were very excited as they had only a couple of days left of primary school!

Cystic Fibrosis Ireland would like to say a huge thank you to Top Oil - Fueling Ireland Mullingar for fundraising €1,600 for CFI! We are delighted to receive your support and a huge well done to all involved.

www.cfireland.ie

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Malin2Mizen Cycle4CF 2019 Thank you to all our amazing cyclists that completed our Malin2Mizen Cycle 4CF 2019, they are an extraordinary group of people. A big Thank You also to the brillant support team that supported the cycle over the 4 days, did a fantastic job and looked after all the needs of the cyclists throughout the cycle. Our cyclists were well fed and watered for the 4 days, thanks to our fantastic sponsors and to everyone else that supported the cycle– Lidl Cork, Spar, Cloverhill Prison, Donegal LSP, Maxol, CFI Southern Branch, Eurocycle Eurobaby, CF West, all our physios in Galway and Mallow, TLC Nursing Homes, CFI Galway Branch, Mobility Ireland, CFI Dublin West Branch, Dunnes Stores, CFI Meath Branch and Cycling Ireland

One in 1000 / Vhi Women’s Mini Marathon Cystic Fibrosis would like to say a HUGE thank you to each of the 540 ladies that took part in the Vhi Women’s Mini Marathon on Sunday June 3rd! What an incredible atmosphere we had on the day at Dtwo, with all of the ladies buzzing for the 10km with all their family and friends. We had a particularly fun time this year with a Latino warm up from Victor Poblano, getting all the girls ready for the route. Keep an eye on our Facebook page for updates on income, but I am very pleased to say that we are nearly at €25k already! An absolutely amazing feat and we couldn’t have done it without YOU! We’re looking forward to next year already.

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Spectrum / Summer 2019

Grafton Merchanting Thank you to Grafton Merchanting ROI, for choosing Cystic Fibrosis Ireland as one of their selected four charities to raise funds for as part of their Four Peaks Challenge with €17,114.03 raised to help people with Cystic Fibrosis The Four Peaks Challenge involved all 22 Grafton Merchanting ROI participants climbing the highest mountain in each of the four provinces over four days. The team ascended Mweelrea in Co. Mayo for CFI! Here they are pictured with Fundraising Manager of CFI, Fergal Smyth. Please join us in saying a huge thanks and well done to all involved! Conference Sponsors Thank you to our Conference Sponsors – Vertex Pharmaceuticals, PTI Proteosasis, Gilead and Chiesi without whose support our Annual Conference could not take place. Pictured left, attending our Annual Conference are Orla Geoghan, Aidan Grimley, Hannah Crookes and Jennifer Richards from Chiesi, with Philip Watt and Fergal Smyth from Cystic Fibrosis Ireland.

Malone Group Thank you to the Malone Group for once again sponsoring our Radio Advert for the 65 Roses Day Campaign and for organising their Annual 65k Cycle which was a great success with many of our Malin2Mizen Cycle 4CF participants using it as a training spin. The ongoing support from the Malone Group for Cystic Fibrosis Ireland as our Charity Partner is very much appreciated. Photo L-R – David Gannon, Managing Director, Malone Group Ireland; Philip Watt, CEO CFI; Fergal Smyth, Fundraising Manager, CFI. www.cfireland.ie

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Rotterdam Marathon

Virgin Media London Marathon CFI would like to say a big thank you and well done to everyone who took part in the 2019 London Marathon in support of Cystic Fibrosis Ireland. They were Olivia Cotter, Aisling O’Neill, Mary Gavin, Fergus Horan and David Crosby and his team. Everyone really enjoyed this world famous marathon and would highly recommend this event to anyone interested in taking part.

A huge thank you to Declan and Shane from Sligo who took part in the Rotterdam Marathon last month and raised a fantastic €2,937! Well done lads and a huge thank you to Mark and Sarah O’Keeffe who helped out with the fundraising! Great teamwork guys.

If you would like to participate in the 2020 London Marathon then please get in touch with our office on 01 496 2433 or email fundraising@cfireland.ie and remember, we can guarantee your race entry for this much sought after Marathon!

Valentines Ball Thank you to Jillian McNulty who raised €4,450 for CFI with her Valentine’s Ball and a further €1,968.23 from Church Gate Collections in Longford & Lanesborough. Thanks to everyone who got involved and supported Jillian with the Ball and Church Gate Collections. Easter Raffle Emma and Aisling O’Leary who organised an Easter fundraiser all by themselves and raised a fantastic €100! They even put their own money into the fundraiser to bring it up! Thank you girls!

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Spectrum / Summer 2019

Spar Congratulations to Aisling & Barry Jones, Gerard Farrelly and Helen Somerville who recently received the fundraising award for the most income raised by a Charity Partner in 2018 (SPAR Ireland) at our CFI Annual Conference in Carlow

Malin2Mizen Cycle4CF 2018 Congratulations to Rosie & SeĂĄn Dolan and members of the Malin2Mizen Cycle4CF Committee 2018 who received the fundraising award for the most income raised at a Community Event in 2018 (â‚Ź145,104) at our CFI Annual Conference in Carlow www.cfireland.ie

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Cystic Fibrosis Ireland Branch Network Carlow Leigh Bolger 087 684 3750 Clare Kieran McCarthy 086 324 4770

Cork (Southern) Aisling O’Neill / Cathy Carlton 085 863 8522 / 086 893 4335

Cavan Lorraine O'Neill 086 829 1561

Drogheda Cyril Gillen / Frances McDonagh 087 694 4922 / 086 075 4776

Dublin West Marie Duffy 086 237 1184

Dundalk Maire Gallagher 086 825 9656

Eastern Mary McCarroll / Christine Drummond 087 411 9812 / 086 874 0338

Galway Liz Gantly 086 173 0180

Kerry Aishling Spillane 083 450 1966

Limerick Padraig Gaffrey 087 288 7799

Mayo Billy O’Toole 086 059 6255

Meath Loretta Allen Byrne / Stephen Kennedy 087 205 8778 / 089 488 1371

Tipperary Mandy Quigley 087 612 0848

Wexford Claire Merrigan / Fiona Bodels 085 726 2206 / 087 167 9279

PWCF Group Antony Dempsey 086 823 0140

Sligo Iris Murphy 086 825 8525 Waterford Alan Barry 086 336 2229

Cystic Fibrosis Ireland 24 Lower Rathmines Road, Rathmines, Dublin 6, Ireland

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t: +353 1 496 2433

f: +353 1 496 2201

e: info@cfireland.ie

w: www.cfireland.ie

Company Reg: 449954

Charity: CHY6350

Spectrum / Summer 2019