Spectrum - Autumn 2024

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Autumn 2024

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CONTENTS Latest News | 3-4

*Beaumont Hospital Update *Kalydeco Update

Advocacy |5-8

*CFI Advocacy Campaigns *Finding hope for those with rare mutations *New Supports for parents *Creon shortage *Autumn Online Event Series

Brendan's Doodles | 6 Member Services | 9-11 *Digital Space from Anxiety *Exercise Grant 2025 Survey *My Medicines Global List *DARE Access to Third LEvel

Spotlight | 12-18

*A Letter to my younger self *Beyond Paris

*Ambassador Profile | 19-20 CFRI | 21-22

*Q&A *Irish National CF Conference 2025

Research Updates | 23-29 *Pathways Project *An update from Enhance *HIT CF

Legacy Month| 30-32 Fundraising| 33-39 * Challenges and Events * Thank You

Dear CF Community, We are delighted to bring you this Autumn edition of Spectrum. You can read about updates on our work addressing Creon shortages, the expansion of access to Kalydeco, progress on the new CF unit in Beaumont among many more.

We are continuing advocacy campaigns for access to modulators and clinical trials for those with rare mutations and our mortgages and insurance taskforce.

In spite of incredible progress in CF care, there remain significant challenges to living well with CF. As Dominic Murphy highlights, until there is a cure our work isn’t done. CFI continues to campaign and fight to ensure that everyone with CF will have the opportunity to live a long, fulfilling independent life.

It was wonderful to see the strength of the community in support of CF this summer. From the Head to Head walk, Malin 2 Mizen cycle, International Walk, our online chats and support groups, our community spirit is the secret weapon of CFI. Together we make a difference.

Our sincere thanks to all those who support us on this journey.

Sarah

FRONT COVER: CFRI and map of Ireland EDITOR: Nicola Delaney Foxe DISCLAIMER: The views of contributors when expressed in this publication, do not necessarily reflect the position or policy of Cystic Fibrosis Ireland

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Spectrum / Summer 2024

LATEST NEWS Official commencement of the new Cystic Fibrosis In-Patient Unit at Beaumont Hospital In a significant milestone for healthcare in Ireland, the official commencement of the new Cystic Fibrosis In-Patient Unit was celebrated onsite at Beaumont Hospital in August.

The unit, which will cost in the region of €20 million, has an anticipated opening date of October 2025. Former local charity, CF Hope Source began campaigning for the unit twenty years ago. In that time, they joined forces with Cystic Fibrosis Ireland and the CFI Eastern Branch to undertake major advocacy and fundraising drives, raising a combined contribution of almost €1 million for the unit.

The event was attended by representatives from Cystic Fibrosis Ireland, CF HopeSource, Beaumont Hospital Cystic Fibrosis specialist staff, including leading Cystic Fibrosis researcher Professor Ger McElvaney and Senior Respiratory Physician Professor Cedric Gunaratnam. Members of the construction and design team including Elliott Group and Scott Tallon Walker Architects were also present.

CF Unit site at Beaumont Hospital - August 2024

Sarah Tecklenborg, Interim CEO CFI commented “We are delighted to see the construction of the long awaited 20 room inpatient unit under way. A result of 20 years of collaborative effort and partnership between Cystic Fibrosis Ireland, CFI Eastern Branch, CF HopeSource, CF clinical teams, Beaumont Hospital and the HSE. The unit will provide the highest standards of care and facilities for people living with CF now and into the future. CFI and the CF community are so grateful to everyone involved in making this build happen.”

Beaumont Hospital is in the top three adult CF specialised units in the country, in terms of patient numbers and national importance to CF adult care. Until now, the hospital had a deficit in the number of specialist inpatient rooms available, compared with the total number of patients with CF attending. The new unit will have capacity to accommodate up to 160 people with CF and will allow for best practices in Cystic Fibrosis treatment.

CF Unit site at Beaumont Hospital - September 2024

www.cfireland.ie

Visiting the site, Maeve Mullin and Mary McLoughlin, CoFounders of Hope Source commented, "Thank you to everyone who has contributed funds over the years to CF Hopesource and CF Ireland. Thanks also to everyone who has lobbied for the unit. To Beaumont Hospital for all their work, and to the CF team who work tirelessly for the CF

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community, and of course to Elliott Group and everybody involved in the design and construction for bringing this project to life.

“The future is bright with this unit. It will keep people will CF well and allow them to take advantage of new medicines and improvements in CF care that are in the pipeline."

The unit was first proposed following consultations with the team at Beaumont Hospital, Cystic Fibrosis Ireland, HopeSource Co-Founders Maeve Mullin and Mary McLoughlin, and patient representatives. Following a commitment from the then Health Minister Simon Harris to the project at that time, planning permission was sought in 2018. However, the project was significantly delayed in recent years by the COVID-19 Pandemic which suspended major building projects on the Beaumont campus.

Commencement of the Beaumont project is the latest project dedicated to CF Care campaigned for by CFI. In October 2023, building began on a new out-patient department at Merlin Park Hospital, Galway providing single consultation rooms and multidisciplinary team areas for people with CF. Due to be completed later this year, Cystic Fibrosis Ireland contributed €700,000 of the €3.5m total cost for this unit.

Kalydeco reimbursed by HSE for babies from one month old with CF in Ireland In April this year, Vertex announced that the European Commission had granted approval for the label expansion of the CFTR modulator drug, Kalydeco, for the treatment of babies with CF from one month old.

This extension applies to those babies who have one of the following CF mutations: R117H, G551D, G1244E, G1349D, G178R, G221S, S1251n, S1255P, S549N and S549R. Cystic Fibrosis Ireland were delighted with this extension and the reassurance it could potentially bring to families around Ireland whose babies have been diagnosed with CF. In yet another positive development, CFI are delighted to share that the Health Service Executive (HSE) took the decision in early September 2024 to reimburse the treatment for babies with CF born in the Republic of Ireland from one month old.

Sarah Tecklenborg, Interim CEO CFI comments “this is a wonderful development for CF care in Ireland. Early intervention can slow the progression of this disease. This is an important step supported by the HSE towards people with CF living full, independent and healthier lives and we thank all those involved in making this happen.”

Full information on the extension can be found on Vertex’s website.

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Spectrum / Summer 2024

ADVOCACY Cystic Fibrosis Ireland Advocacy Campaigns A key purpose of Cystic Fibrosis Ireland is to advocate with people with Cystic Fibrosis in Ireland. Each year, and at times more frequently, we hear concerns from our members about how they or family members are enabled to live and work independently while managing the condition. In the past, CFI have fought successfully, arm in arm with the CF Community in Ireland for advancements such as the provision of Orkambi, the role out of Kaftrio, the inclusion of certain groups of our members in the Portfolio agreement, and have had key involvement in reports such as the Pollock Report, CF Model of Care, while producing our own Independing Living Report. Combined, these and other advancements have changed the landscape of Cystic Fibrosis and while many of our members are living healthier, different lives as a result of modulators, this has not diminished the difficulties of living with CF. It has simply changed the nature and the needs of many of our members.

To answer these needs, CFI have set up two working groups to address two very prominent issues currently causing concern to our members. By focusing on two distinct campaigns at a time, we believe we can ensure a more successful outcome.

Campaign for Access to Modulators for those with rare mutations A working group is being established to support the upcoming campaign for accesss to modulators for the 6.7% who do not have access due to rare mutations. An important meeting took place between the EMA and Vertex on the 5th September and CFI sent a submission to CHMP representatives in Ireland outlining the importance of access to trials of modulators for anyone who may stand to benefit. The submission included perspectives from parents of children without access to modulator therapies due to rare mutations.

Campaign for access to mortgages and mortgagae protection products CFI have established a working group to support our campaign for better access to mortgages for those with CF. Emma, Rory and Sarah recently met with representatives from the Housing Agency to outline the difficulties faced by people with CF in attaining home ownership and suitable, safe rental accommodation. We shared details of European policies which support people with long-term conditions such as the AERAS convention in France.

Please follow our communication channels for updates or to get involved, email Emma.Wallace@cfireland.ie.

www.cfireland.ie

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A Special Message from Two CF Mums – Finding Hope for Those with Rare Mutations As mums of two wonderful boys with cystic fibrosis, our hearts are full of love but also the weight of a journey that's harder than we could have imagined. Our sons both have rare CF mutations, which means they aren't eligible for the modulator treatments that have given so many families hope. Knowing these options aren't available to our children is incredibly tough.

We know we aren't alone in this. If your loved one is also living with a rare mutation, we understand how isolating it can feel, how many unanswered questions there are, and the quiet hope we all hold for a breakthrough.

That's why, with CFI’s help, we're starting a group specifically for PWCF and families like ours to offer support, share experiences, upcoming research and advocacy opportunities and all work together for a bright future.

We believe that together we can create a space where no one has to face this difficult journey alone.

If you'd like to join us, please contact memberservices@cfireland.ie. We're stronger together, and we'd love to have you with us.

With love and hope, Louise and Stephanie

Brendan's Doodles

by Brendan Lonergan @BOB_NARROW, CFI Member 6

Spectrum / Summer 2024

New Diagnosis?

New Supports for Parents of children with CF available A diagnosis of CF in a child can be overwhelming and emotionally challenging for parents.

Providing support, resources, and information to parents during this difficult time is crucial to help navigate the journey of caring for a child with CF. To address this need, CFI, in partnership with our parents working group, have developed a newborn care pack for distribution to new parents at the time of diagnosis by their hospital team. The objective of the care pack is to offer resources, information, and tools to help parents better understand and manage their child's Cystic Fibrosis.

Additionally, we have formed our Parent Connect Team which aims to create a sense of community and connection among parents facing similar challenges.

The Parent Connect Team will be an initial point of contact for new parents prior to registering with CFI. This is a peer a led service which aims to provide initial support and guidance to new parents. They can be contacted initially through the following email: parentconnect@cfireland.ie We are also thrilled to announce that our Parents Support Group for all parents of children with CF in their early years, will meet online monthly going forward with the next meeting taking place on 2 October at 8pm.

Registering for this event can be done through our website at www.cfireland.ie and is a great way to stay connected and share experiences with other parents. We hope to see you there!

www.cfireland.ie

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Update on CREON (PERT) Shortage We understand how frustrating and stressful it is that the shortages of CREON persist. Stock of Creon is regularly coming into Ireland albeit in reduced quantities. The Medicines Shortage list on the HPRA can contain updates on expected date of return during periods of shortages. https://www.hpra.ie/homepage/ medicines/medicines-information/medicines-shortages/shortages-list We understand the challenges this situation poses to members. Alternative enzyme products will be covered by your medical card/LTI scheme while Creon is out of stock. Please speak to your local pharmacist about alternative product and please contact your CF Dietitian for advice and prescription for alternative product if needed.

Our CFI Member services team have the most up to date information and may also know of available supplies. Please give them a call if you are in need.

We are in regular contact with Viatris, the HPRA, our colleagues in the UK and CF Europe and we continue to advocate on this issue. We will bring you updates as we receive them. Our team are involved in a CF Europe Drug Shortages taskforce who are also working to ensure a swift resolution to the shortages of Creon and other products.

Autumn Online Event Series Recording now available

Following the success of the Summer online event series, we will continue to host online coffee chats to enable the CF community to come together safely socially to share thoughts, experiences or just have a chat.

Our sincere thanks to everyone who has supported or hosted one of these events. You can find details of upcoming events on the Members Events page of our website.

Webinar series Following the nationwide Meet and Greet events, CFI began hosting a series of webinars, providing information on several topics of importance which were highlighted by our members. The first of these was a webinar entitled 'Mental Health and Modulators - The Paediatric Perspective' we were delighted to welcome Professor Paul McNally and Doctor Sarah Carroll to speak. A follow-up webinar on mental health and modulators from an adult perspective is being planned and details will be announced in coming weeks.

If you missed the first webinar, you can watch the recording on our website at www.cfireland.ie.

We would love to hear suggestions for other information evenings, supports and feedback from you, our members. Get in touch by emailing info@cfireland.ie or contacting our Senior Member Services and Advocacy Coordinator on emma.wallace@cfireland.ie.

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Spectrum / Summer 2024

SERVICES NotMEMBER Forgotten: The Last 10% Taking 'Space From Anxiety' to make space for positive mental health Maintaining positive Mental Health and wellbeing is vital to navigating the challenges that arise in our day to day lives. Our mental health affects how we think, feel and also how we act, impacting upon our coping mechanisms, relationships and decision-making abilities.

Mental health is vital through all the stages of our lives from early childhood through to adulthood.

Cystic Fibrosis brings with it an additional set of challenges, and so, it is important that we allow ourselves the time and space needed to support our mental health and wellbeing, building upon the resilience needed to work through these challenges. Depending on your life stage, support from a mental health professional can take the form of counselling, psychotherapy, art therapy or play therapy and can contribute significantly to building resilience, managing anxiety and supporting coping mechanisms.

CFI offers a counselling referral service to our members who wish to speak with a professional outside of the hospital environment in addition to a counselling grant which can also be used for such services including counselling, psychotherapy, art therapy or play therapy. Additionally, we can signpost into other agencies and organisations that have services that our members can avail of. One such organisation is the ISPCC who have three free programmes aimed at providing teens, parents/carer with skills and techniques they can employ in their daily lives that help the young person cope with anxiety: •

Space From Anxiety: aimed at 15–18-year-old young people who experience mild to moderate anxiety.

•

Supporting An Anxious Child: aimed at parents/carers of 5–11-year-old children who experience mild to moderate anxiety.

•

Supporting An Anxious Teen: aimed at parents/carers of 12–18-year-old young people who experience mild to moderate anxiety.

*In signposting to services offered by external organisations CFI does this in an information sharing capacity. All external organisations are governed by their own policies, procedures and compliance processes independent to CFI and are utilised at your own discretion.

www.cfireland.ie

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CFI Exercise Grant 2025 - Have your say The Exercise Grant 2024 has now drawn to a close and with that in mind we are looking towards 2025. To help us understand your thoughts and opinions around the exercise grant process we have designed a survey which you can access here.

This Survey will run over the month of October, and we would be grateful if you would take the time to complete this. The survey is anonymous and should take no more than 5 minutes to complete. Your feedback is really important to us to ensure your voice is part of how the exercise grant for 2025 is delivered.

Many thanks in advance, The Member Services Team

My Medicines List now available in 52 different languages The HSE has developed new resources to help promote the Know, Check, Ask Campaign and reduce medication-related harm. The HSE encourages everyone who takes medicines to keep an up-to-date list of their current medication by using a medicines list. The HSE’s ‘My Medicines List’ is a list of all the medicines and supplements a person takes. Keeping a medicines list can help individuals know and keep track of their medicines. It can also help when discussing medicines with a healthcare professional. These medicines lists are now available in 52 different languages and can be downloaded from www.hse.ie/safermeds. Hard copies of the ‘My Medicines List’ in English can be requested via www.healthpromotion.ie .

Comprehensive guidance on why and how to use a medicines list is also available through a new video, accessible on the Safermeds website or on https://www.youtube.com/watch?v=-92LfGenHb.

CFI MEMBER SERVICES TEAM

Contact us by telephone on 01 496 2433 or by email at memberservices@cfireland.ie

Emma Wallace

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Rory Tallon

Caroline Heffernan

Spectrum / Summer 2024

DARE - Access to Third Level Education What is DARE? DARE (Disability Access Route to Education) is a third level alternative admissions scheme for school-leavers whose disabilities have had a negative impact on their second level education.

DARE offers reduced points places to school leavers who, as a result of having a disability, have experienced additional educational challenges in second level education.

Who can apply to DARE? DARE is for school leavers under the age of 23 with a disability, who have been educationally impacted as a result of that disability.

Applicants to DARE can present with an Irish Leaving Certificate, A-Levels and/or other EU qualifications.

How do I apply to DARE? Applicants applying to DARE must: 1. Be under the age of 23 as of 1 January - double check this with DARE directly.

2. Apply to the CAO by 17:00 on 1 February- double check this with DARE and the CAO directly.

3. Disclose your disability in your CAO application and fully and correctly complete Section A of the Supplementary Information Form (SIF).

4. Download Section B Educational Impact Statement (EIS) and have your school complete the form.

5. Download Section C Evidence of Disability, and have it completed by the appropriate medical professional (only if you do not already have a report verifying your disability which meets the DARE criteria).

All relevant forms and further information can be found on the DARE website at https:// accesscollege.ie/dare/

www.cfireland.ie

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SPOTLIGHT A letter to my younger self...

What would you go back and tell yourself if you had the chance?

Grace Shesgreen looks back at starting college as a person with CF and shares what she knows now and what she wishes she knew then....

Heya, I’m Grace.

I’m 22 years old. I’m a PWCF and I just completed my four year BFA in Musical Theatre and Performing Arts from American College Dublin. I really enjoyed my college experience, although I still had those nerves of moving away and going somewhere new.

I started college in September of 2020, with my entire first year experience existing through a little computer screen.

At times, I felt this made my transition a little more difficult in terms of making new friends, getting settled in and gaining some independence by moving away from home, which I didn’t get to do until my second year.

However, we made it work. We were there for every class. All of us students had zoom meetings after class, had presentation nights, Netflix parties, and some of the best chats. We knew to get to know each other because we were aware that everyone was in the same boat. We were in a new place, didn’t know anyone and wanted to make the most of the college life in any way we could. And now, four years later, I’m here.

I thoroughly enjoyed the experience of being away from home, figuring things out and being responsible for myself and learning how to live in a city. And now that I’ve finished college, looking back, if I had to give myself any advice, I would want to tell myself the following tips;

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Spectrum / Summer 2024

Top Tips for Uni 1. If you have gotten into college through DARE, make an appointment to visit the DARE office within your college and discuss your needs. Unfortunately, my college didn’t operate through the DARE system, but thankfully, we were a small group of students and I had a good relationship with the course director. This allowed me to be able to speak to her whenever I had any issues. The disability access offices are a wonderful support system, but as a student, you need to engage with them in order to avail fully of all their supports. So don’t be shy, they want to help to make college experience easier for you!

2. Figure out your time table. From this, you could speak to your CF Centre with regards to arranging clinic dates that would suit you as much as possible. By doing this, you can avoid them clashing with exam season or around the times you know you may have multiple assignments due.

3. Be organised. Unfortunately we don’t have the luxury of being able to roll out of bed ten minutes before our 9am lecture. You want to be up early enough to ensure you get your treatments done, have a good breakfast and have a route to college already figured out in your head, which can often be weather dependent. Eg, you may love walking to college just like I did, however if it’s raining you may need to take a bus or some other form of public transport. (A good raincoat and/or a comfy warm coat is a must. Have that stylish one packed and ready to throw on) I usually packed my bag for the college day ahead of me, lunch prepped, or even prepared some overnight oats the previous night to give myself that extra few minutes of sleep.

4. Take care of yourself. In all aspects, physically and mentally. Always try to eat well. Go home from your night out or from your late night study session in the library to get your good nights sleep. Try not to allow yourself to stress too much with your workload. Maybe allow yourself to get ahead of the game. If you know you have an assignment due in a month, make a start on it. Take ten minutes out of the day and take away the potential stress you could have the day before it’s due. Don’t be afraid to ask for help from your fellow students or tutors!

5. If you are unwell and need to take a day off, don’t panic! You will catch up. That’s one or two days you’ve missed out of your entire four or more years at college. And if you are a DARE student, your college will possibly have the service where someone may come in and take notes for you, or you can receive assignment extensions.

Most importantly, enjoy your time in college. Whilst remembering to work hard to get to the end of your course and earn your degree, make sure to meet new people and make friends, have the nights out, join clubs and societies and make the most of this time in your life. It will fly by. It definitely has for me!

Wishing you the best of luck with this exiting time in your life!

Grace xx

www.cfireland.ie

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Beyond Paris: A Season of Post Transplant Sport This year, the world was treated to a global summer of sport. With the Olympics starting in July, then the Paralympics in August, we in Ireland, were treated to a performance from the most acomplished athletes - some bringing medals home, some breaking world records but all equal in passion and dedication to their sport.

But the games didn't stop in Paris. Members from our very own CF Community both people with CF and advocates for the CF Community, packed their bags and travelled, like our Olympians and Paralympians, to mainland Europe. This time to Portugal and Italy to the European Transplant Sports Championships 2024 and the first ever Transplant Football World Cup 2024.

Performing with an Olympic level of passion and dedication, our Transplant athletes have returned home - laden with pride, a sense of accomplishment and like the Olympics and Paralympics, more than a few medals. Here we talk to some of those athletes who made the journey, from Ireland to Europe, from transplant to Transplant Games to top off summer 2024 and what really was a season of sport.

The European Transplant Sports Championships 2024 (ETSC 2024) were held in Lisbon, Portugal from Sunday 21st July 2024 to Sunday 28th July 2024.

These joint games were organised by the two European Federations, the European Heart and Lung Transplant Federation (EHLTF) and the European Transplant and Dialysis Sports Federation (ETDSF).

ETSC 2024 promoted the importance of organ donation through sport and was a celebration of the Gift of Life. The Irish transplant team of 25 athletes led by Harry Ward travelling under the auspices of Transport Sport Ireland (TSI) returned home with a wonderful total of 65 medals, 31 gold, 22 silver and 12 bronze.

The TSI team included three athletes from the Irish Heart and Lung Transplant Association (IHLTA) heart transplants recipients Michael O’Shea and Robert Mc Cutcheon and CF double lung transplant recipient Victor Conroy.

The next European Transplant Sports Championships will take place during June 2026 in the city of Arnhem, The Netherlands. Our aspiration is to bring more lung and heart transplants to the Netherlands. If you would like more information, please do contact me, Robert McCutcheon, via https://ihlta.com/

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Spectrum / Summer 2024

My name is Victor Conroy and I am a person with CF and double lung transplant athlete. I went out to Lisbon with the Irish team of 25 athletes and was delighted to represent my country and honour my donor.

The main sports that I do every week are sea kayaking and white-water kayaking. I’m passionate about outdoor sports, Irelands coastline, rivers and wildlife. It’s a brilliant way to keep fit all year round in any weather conditions.

I took up the sport in 2018, a year after my double lung transplant in August 2017. I took a beginner’s course in Howth and never looked back. I’m now working towards my Canoe Ireland Level 4 Qualification and hope to be an instructor someday.

I went out to Lisbon with the Irish team of 25 athletes.

I was delighted to represent my country and honour my donor. I won gold in darts and shotputt, and silver in 50m freestyle swimming at the European Transplant Games.

The Transplant Games were one of the best experiences of my life. Seven years ago, I was on oxygen and struggling to walk to the hospital cafe for my scone and tea ritual in the morning. If you told me back then, in the future, I was going to swim a fifty metre pool, see dolphins off the coast of Lisbon, paddle to the Blasket Islands and around Mizen Head this summer, I’d say those dreams belong to someone else. The fact that they’re my reality now is something I’m still getting used to. I got to meet some of the most inspirational people and their families. The games represent an unrelenting spirit of never giving up and immense gratitude to the people who helped me get there.

I try to be consistent with fitting exercise in every day. It varies daily. If I’m not out paddling at sea or on the river, I’m in the gym. I’ll do at least 30 minutes cardio to raise the body temperature and stretch muscles on the bike, rowing machine or ski erg. Then it’s mainly kettlebell lifting, body weight exercises, push ups, pull ups, pulling and pushing a weighted sled.

I aim for overall fitness rather than being specific to one sport. I do a little bit of plyometrics to build core fitness and explosive energy and power. All the exercises I do are minimal equipment and can easily be done whether I’m in hospital or at home. I also have diabetes; this means really watching the sugar intake and weight gain. When I have it under control, energy levels are much better, feelings of fatigue are lower. The next goal for me is the World Transplant Games in Dresden, Germany in 2025.

I’ve been fortunate enough to get world class healthcare in every hospital I’ve attended. Before transplant and after transplant, the best advice I could give is the same. Turn up for your CF Team/Transplant Team, physiotherapy, exercise, medications, medical appointments. Listen to the advice, do your best to stay healthy and keep the head up. Talk and walk it out. Distract yourself on hard days, binge watch a box set, do a jigsaw puzzle, listen to music. No matter how hard it gets, believe that the future can and will get better.

www.cfireland.ie

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My name is Louis and I was one of a group of Irish players on the Irish Transplant Football Team who have all received the gift of life and a second chance. We recently represented our country in Italy at the first ever Transplant World Cup in September 2024.

The Ireland Transplant Football team were one of very few teams with Double Lung transplant recipients, with us having four out of the five Double Lung transplant players in the entire tournament. Jack Bentley, Lar Brennan, Keith Daly and I were honoured to represent Ireland and Cystic Fibrosis in the World Cup tournament.

This unique event marked a groundbreaking moment in both sports’ history and the global transplant community, as players who had undergone life-saving organ transplants competed on an international stage.

The event was aimed to raise awareness about the importance of organ donation and promote an active, healthy lifestyle for transplant recipients.

Pre-Match Our team landed in Italy on Friday 6th September, and got the bus to Cervia from Bologna. We arrived at the hotel and saw our new home for the next week, right beside the sea and near the centre of the small town. The next morning, we woke up for breakfast, cycled down to the beach and played some volleyball. It was a great team bonding exercise and got us a feeling of the heat we would be playing in. On Sunday morning, we got in our last training session before the tournament started. The opening ceremony was on Sunday evening where all the teams got to wave their flags and represent their country in the walkout. Speeches were made and we got some team photos.

Ire 3 - 0 Wales Monday was when the football started, with our first game against Wales at 9am Italian time. We had played Wales before, so we knew what to expect and had a game plan ready, which worked well as we beat them 3-0.

Ire 8 - 0 USA Our next match was against USA that same evening, a team we hadn’t played before, so we didn’t know what to expect. We played brilliant football and won the match 8-0, with Jack Bentley scoring the final goal of the match.

Ire 9 - 0 Romania The next morning, we had another early start, facing off against Romania at 11am Italian time. Another brilliant performance from the boys in green as we beat Romania 9-0. We were gaining confidence, and our football was looking good.

Ire 1 - 3 France Our next game was that same evening against a tough opponent, France. We stepped out onto the pitch again and shook hands with the French team, posing for more team photos. The French squad had less

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Spectrum / Summer 2024

players than our 16-man team in this seven a side tournament but looked like a team with real quality. We knew that if we won this match, it would mean qualification into the knockout stages. We went 1-0 up within the first 8 minutes and felt the confidence rise again. The Irish crowd began to chant Olé Olé Olé. Unfortunately, they equalised within a few minutes of us scoring and went 2-1 up just before the first half ended. We tried our best to get back into the match but conceded another goal to a late penalty, and it finished 3-1.

Ire 0 - 2 England

Jack Bentley, Louis Duffy Barrett, Keith Daly and Lar Brennan from the Irish Transplant Football Team

Our final group stage match was against England the next day and we knew we had to win to qualify, and of course, we wanted to beat our rivals. It was another early kickoff, and with the Italian heat belting down, both teams walked out, shook hands and posed for photos before the match began. We went behind within the first 3 minutes and conceded another just before the first half ended. We knew we had to have a good second half and tried to fire each other up at half time. We created some great chances and controlled the ball well in the second half but couldn’t get a goal, with the game finishing 2-0.

Ire 4 - 5 Italy With 3 wins and 2 losses we had another opportunity to compete and try get our finishing position in the tournament in the 5th/6th place playoff against the hosts, Italy. The match took place on the Thursday morning. We walked out in the green jersey for the last time in the tournament, hearing the supporters chant for Ireland and shook hands with the Italian team. We went 1-0 down in the first half but were playing good football and felt we could get back into the game. We had plenty of chances and were dominating possession as the Italians stepped back and tried to defend their lead. Time was running out as we tried time after time to get behind the Italian line, with balls into the box and shots from everyone in the team. Eventually, in the very last minute, we broke through as our centre back hit a long range shot into the bottom corner of the net. The match finished 1-1 which meant penalties were next. Jack Bentley stepped up to take the first penalty, burying it in the back of the next putting us 1-0 up. It went back and forth with both teams scoring 3 penalties each, until unfortunately one of our players missed. Italy scored their remaining penalties along with us, meaning it finished 5-4 with Italy winning on penalties. We finished 6th in the tournament.

Pride and post-Match Analysis While we were disappointed in some ways, we felt pride in representing our country and were excited to see what the future for the team was. Our performances gave us confidence and we felt we could beat these teams on a better day, and we had the quality to go even

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further. Jack Bentley played every match and made the Cystic Fibrosis, Double lung transplant community and donor family proud with his performances and commitment to the team. It showed what you can do post transplant and how much you can achieve with your new lungs and new lease of life.

More than just a sporting event - Team Connection There was a great team connection amongst the Irish squad, and we felt like we found another family. Aside from the football, we all got to know each other better and our backgrounds and stories of our lives up to this point. We enjoyed our journey throughout the trip to Italy. It was also amazing to meet new people who’ve shared similar experiences with transplant life, from Chile to Australia and from Spain to Wales. The first-ever Transplant World Cup was more than just a sporting event; it was a symbol of hope, resilience, and the global unity that organ transplantation can foster.

The Irish Transplant Football team is open to anyone who has received a transplant and wants to join a team, talk to people who have a familiar story, and who want to get healthier and fitter in their post transplant life. It has helped me gain the confidence and drive to improve my fitness and get back to playing football, something I loved before all the hospital visits and infections took it away from me. I have learned so much from the team outside of football too, and, after covid and the pandemic, I feel as if I’m finally getting back to normal.

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BEING AN AMBASSADOR "Until there is a cure, the work isn’t done.

The more Ambassadors, the more voices, the better."

Each year, CFI run campaigns and events to build awareness and educate people about the challenges of living with Cystic Fibrosis. To do this and to tell the story authentically, we engage with Ambassadors from the community who are comfortable to share their story in different settings. Whether it is in a media interview, or at a members webinar, it is these stories that tell the true story of CF. Around this time each year, we speak to one of these wonderful individuals who give their time and experience to CFI to help us reach our objectives. This year, we hear from Dominic Murphy, Tipperary, who is 40 and lives with CF. "Hi Dominic, thanks for giving your time to share your experience with us. Can we start by you telling us a little about yourself?

Hi, sure no problem. I'm Dom. I’m outgoing, positive, happy person and I studied art in college. I love to work when I feel good and have done a lot of heavy work in the past. I grew up with a good bunch of friends and am the youngest out of four. I like to help others wherever I can and if I have a goal, I will do my best to achieve it. I am interested in politics, am a coffee hopper and am very house proud. I am definitely not shy and love life.

Thanks Dom and when did you become a CF Ambassador?

I became an ambassador in summer 2023 – just before the programme began.

How did you become a CF Ambassador and I suppose more importantly, why?

I saw online that CFI were looking for people to tell their story about life with CF. I already knew some of the team at CFI, so I spoke to them to find out a bit more and when the programme started, I was all in. It means a lot to me. It is a very personal thing especially when you have CF yourself. And the whole idea for me is to spread awareness, be out there sharing my story, doing what I can to help and support everyone who is doing amazing things. CF is my personal friend – it taught me to survive so I want to share story to help others.

That's so important. So what type of tasks are asked of you?

I have been involved in brainstorming sessions to help the CFI team create campaigns and I give my experience of life with CF. I’ve been involved in 65 Roses day too and I’ve taken part in all the training provided for

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example the media training. It is really helpful to make sure we are all on the same page on campaigns.

So it can be a very varied role. Does it take much of your time?

Well, it takes only as much or as little as you can give, and I guess it depends on the project you are working on. If you are writing for Spectrum then there might be a bit of preparation and the time you spend writing, but if you are doing an interview, then it might only be a phone call or two to get the job done.

Do you get support from CFI or from the community?

Yes, I would say so. There is a schedule of events you can join which CFI put on to help prepare you for whichever area you are interested in taking part in. And you get to meet other people with CF virtually, which is cool too. But there is also never any pressure on you either. There have been times when I can join the trainings and times when I can’t. Life happens and the team know that.

So. do you think it is a rewarding experience?

Yes definitely. It is very rewarding to belong to a group and work on projects for a goal that is personal to me.

Would you encourage others to become CF Ambassadors?

Absolutely. We need to keep spreading awareness of CF. I know we have had so many positive developments in recent years but many still live with challenges and until there is a cure, the work isn’t done. The more ambassadors, the more voices, the better."

If you are interested in becoming an Ambassador for CFI, or would like to know more, you can find details on our website at https://www.cfireland.ie/ or email: ndelaneyfoxe@cfireland.ie

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The CFRI What do you want to know about the registry?

This is your chance to ask CFRI! CFRI Q&A

CFRI would love to know what you want to know about their work?

Do you want to know more about what registry data is used for?

Or how data is collected? Or maybe you want to understand the positive impacts the registry has had for CF in Ireland?

CFRI are here to answer any questions you have – you can submit questions anonymously via CFI on this link here. Alternatively, DM us at CF Ireland and we will anonymously pass on questions you have. CFRI will answer questions via future Spectrum contributions and via their social media channels.

Please note, CFRI cannot address questions relating to individual participation in the registry or an individual’s data. PwCF/families with questions regarding their data/participation in the registry can contact their clinical teams or info@cfri. ie.

What have CFRI been up to?

Summer time is usually a quieter time across the board, but rest-assured, the registry team have been busy working away on a number of things.

Annual report 2023 We are delighted to be in the final stages of pulling together the CFRI annual report for 2023 data. Our report will be published in the next few weeks alongside an infographic summarising some of the key data highlights. In the report, you’ll find data presented on our patient population, across several demographic, clinical outcomes and healthcare utilisation outcomes. In the meantime, you can browse our previous annual reports and infographics here: https://cfri.ie/annual-reports/

National Cystic Fibrosis Conference We are also very excited to be a part of the organising committee of the forthcoming National Cystic Fibrosis Conference in January 2025. This is a conference for clinicians and MDTs & provides an opportunity to learn about the latest in Cystic Fibrosis research as well as an opportunity to network and engage with colleagues from all over the country.

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Want to learn more about what CFRI do?

If you would have any questions or would like information about participating in the registry, please contact your CF Centre or the CF Registry at info@cfri.ie. Participation is entirely voluntary.

You are free to revoke your consent and withdraw from the registry at any time.

We would love if you could follow us on social media or via our website. We post updates on our work and on the exciting projects we are working on. Scan the QR code below to learn more & access the latest data & reports!

IRISH NATIONAL CYSTIC FIBROSIS CONFERENCE 2025 The CFRI and CFI are delighted to announce the save-the-date for the forthcoming Irish National Cystic Fibrosis Meeting for Clinical Directors, members of MDTs, Microbiology Department and other clinical staff who may be interested in attending.

Thursday 30th January 2025 starting @ 18:00 until Friday 31st January 2025 (all day) The Strand Hotel, Limerick Further conference information and registration link to follow.

On behalf of the Organising Committee incl. Prof Barry Linnane, Prof Barry Plant, and Prof Ed McKone.

*Please note this is not a Cystic Fibrosis Ireland Members conference. This event is for members of clinical teams only.

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RESEARCH

ENHANCE (Establishing Natural History in an Advanced New CF Care Era) is a five-year long CF study based in 13 different locations all over Ireland and the UK. Outcomes for children with cystic fibrosis (CF) have been getting steadily better over the years. However, a lot of what we understand about childhood CF comes from research that was completed before some of the more dramatic improvements of CF therapies.

In order to understand early CF in this new era of improving outcomes, a long-term study with a large number of CF participants is needed. This is why the ENHANCE study was established. ENHANCE will help us to understand much of what we currently do not know about how children live with CF through childhood.

The study began in Autumn 2023. At each paediatric CF Centre, a lead CF consultant coordinates the study locally. There are three groups in the study: children recruited from diagnosis at new-born screening, children who are already diagnosed with CF (attending one of the centres and are less than six years of age), and children who do not have cystic fibrosis (control group). We aim to recruit approximately 450 children with CF and 100 children without CF.

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Research studies can be time consuming for children and their parents. To help minimise the burden on participating families, we only collect data once a year around the time of the child’s annual assessment or CF clinic. ENHANCE focuses on various aspects of the lives and health of children with CF. Using the ENHANCE data, we aim to better understand: • • • • • • •

How often structural lung disease occurs, how common it is and any changes related to the new treatments.

The long-term natural history of lung function.

How often CF liver disease occurs, how common it is and any changes that are related to the new treatments.

How often exocrine pancreatic dysfunction occurs, how common it is and any changes related to the new treatment.

The natural history of gastrointestinal symptoms, inflammation and the gut microbiome compared to a healthy control population.

The natural history of annual sweat chloride levels in infants and children of different ages, the influence of different treatments on this and its association with other outcomes.

The natural history of mental health outcomes in children with CF compared to controls.

Children with CF can develop a number of medical problems as they grow, including abdominal disease, lung disease and mental health problems. Things have been improving for children with CF over the years, but it still appears that they do not have the same levels of health that children without CF have. If we want to improve outcomes for children with CF we must first understand clearly how the disease still affects them.

This is where research projects like ENHANCE come in – they allow us to paint a clear picture of the real-life problems that children with CF have and help us to figure out how we might help.

We will carry out a wide range of well-established tests, as well as some newer, more sensitive tests looking at how the health and quality of life in people with CF changes over time. Some more advanced tests like CT scans and multiple breath washout (MBW) are required as part of this study (age dependent assessments).

Although mental health is one of the top 3 research priorities of the CF community, major gaps remain regarding the study of mental health conditions in the CF population. ENHANCE will improve our knowledge within multiple mental health areas of focus. These outcomes will be measured annually using the following questionnaires: • • • •

The Paediatric Symptom Checklist (PSC): A brief and widely used parent/self-report (age dependant) questionnaire. The PSC measures cognitive, emotional and behavioural difficulties in children aged 0-17 years.

The PROMIS Paediatric Anxiety Scale and Depressed Mood Scale: Brief self-report questionnaires administered to children from the age of 8 years, providing discreet measures of depressed mood and anxiety for younger children.

The PROMIS Paediatric Cognitive Function Scale: This is a brief self-report questionnaire of children’s experiences of attention, memory and comprehension, which is validated for use from the age of 8 years.

Body image will be assessed using a three-item questionnaire designed for this study, completed by children from the age of 8 years.

The safety and well-being of ENHANCE participants is always our number one priority. In cases where concerns arise from completing the questionnaires, these concerns will be relayed to the child’s local PI, a CF nurse specialist and local CF psychologist. Further support and/or onward referral will be available to all

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participants if necessary.

As a small token of thanks to the children taking part, every child will receive an ENHANCE Teddy-bear buddy. A different animal Teddy will be given for each year.

We will soon run a ‘Name the Teddy’ competition to help us name our buddy!

BUNNY will take place over a two-year period, and is a sub-study to the ENHANCE study operated at the same sites. This study will focus on the gut health of children from birth with CF. As part of this study, the participating child will be asked to provide a stool sample and the parent will be asked to answer a short questionnaire monthly for the first 6 months, every second month until 12 months, and every three months for Year 2 of the study. We aim to recruit approximately 60 children with CF and 40 children without CF across all 13 sites.

ENHANCE and BUNNY are two important studies that are recruiting babies with CF from diagnosis. Collecting information from the time of diagnosis during long-term studies like these is vital as it allows us to understand the earliest origins of disease and work out how the disease itself and the treatments work.

We are currently recruiting newborn children (under 12 weeks old) without CF for ENHANCE and BUNNY. As this is the control group, the children must not have CF or have a sibling with CF. This control group are not required to attend the clinical site as all assessments will be completed remotely. Stool collection apparatus and questionnaires are posted to the control patient’s home and the study team will arrange for courier collection of samples and questionnaires.

If you or someone you know would like to support the CF community by taking part in these studies, or if you have any questions, please contact our team at enhance@rcsi.com.

Pathways to Improving Transition to Adult Services & Enhancing Self-Management in Young People living with Cystic Fibrosis The World Health Organisation defines adolescence as the period between 10-19 years. This is a time of change, both developmental and educational. Being an adolescent living with Cystic Fibrosis (CF) brings additional challenges due to complex and demanding treatment. Since the introduction of the new triple combination CFTR modulator therapies, the CF-clinical landscape is also changing. For some, there remains a requirement for in-patient care, for others there is a reliance on outpatient support and self-management.

How young people are adapting to these changes is an important area to consider.

As children transition into adolescence, their healthcare and support needs also change. In Ireland, many adolescents transition to adult care aged 16-18 years. For young people and their families, this is a much more than a logistic challenge. This is a move from a familiar environment and trusted clinical team to an

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Dr Caroline Heary is a developmental psychologist and senior lecturer at the School of Psychology, University of Galway. Her programme of research focuses on child/ adolescent health and wellbeing, the social context of health and wellbeing and the role of family, and school and peer support in fostering health & wellbeing. As Principal Investigator, Caroline leads the Pathways programme of research, having secured funding from the HRCI/ HRB Joint Funding Scheme 2022 in partnership with CF Ireland. Caroline is also co-investigator on YOULEAD, a youth mental health leadership doctoral training programme and a Co-Director of the PhD in Child & Youth Research at the University of Galway.

Dr Angeline Traynor, is a paediatric psychologist and postdoctoral researcher on the Pathways project. Angeline’s doctoral research focused on the psychological treatment of chronic pain in children and adolescents. Her research interests include the child/adolescent health and wellbeing, the psychological treatment of chronic conditions, transition readiness and public and patient involvement in research.

unfamiliar environment and clinical team. To make this move less daunting, the topic of healthcare transition should be discussed from at least early adolescence. A key task is to learn the skills needed to manage their health as they prepare to move to adult services. Fostering independence and self-management skills in these early years can help young people negotiate the various challenges that arise as they get older and move to an adult service. Keeping on top of the advice and recommendations of their health care team can be a challenge for many young people as this busy stage of their lives. Keeping up with friends, college and work are important to many. Understanding the needs of young people at this time is important as we move into a new era of treatments.

Pathways led by Dr Caroline Heary and Dr Angeline Traynor at the University of Galway, is a programme of research that includes three very different studies with a common goal, to explore and identify how to best support young people living with CF as they learn to manage their health and prepare to transition to adult care. Pathways is funded by the HRCI/HRB Joint Funding Scheme 2022 in partnership with CF Ireland. Collaborators include clinical and research teams from Children’s Health Ireland, St Vincents University Hospital, Sick Kids Children’s Hospital in Toronto, and the University of Galway. This research has two key areas of focus: the development of self-management skills and transition to adult care.

Pathways Interview Study The Pathways programme began with ainterview study that is ongoing in three paediatric sites and one adult site in Ireland.

This work seeks to understand the needs and experiences of The Pathways team are supported by young people in this new era of treatment regardless of their collaborators from Children’s Health Ireland at treatment plan. Young people from paediatric and adult sites in Crumlin, Temple Street and Tallaght University Dublin and their parents take part in one-to-one interviews to Hospital and at St Vincent University Hospital, share their experiences. In sharing their journey and what they the University of Galway and SICK Kids Children’s have learned over time, they are also identifying supports that Hospital, Toronto may be used to prepare for the future in what is a very changed clinical landscape.

While this study is ongoing, the response has been overwhelming positive from all who have generously given their time and experience. The overall findings from this research will be shared with research and clinical collaborators including Children’s Health Ireland and St Vincents University Hospital who have supported this work and are eager to give voice the patients and families they work with.

Pathways Survey Study – www.pathwaysresearchstudy.com Building on this work, the Pathways Survey was launched in the Summer of 2024. This is a nationwide, online, anonymous survey designed to overcome geographic barriers by capturing the supports needs of young people living with CF from across Ireland. Some find the move to greater independence and the move to adult services harder than others. Understanding these differences can help us make recommendations on

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the different needs young people might have. It is important to understand what are the barriers that now need to be addressed and are there additional or differing supports needed as young people move into adulthood and prepare for the future. The Pathways Survey is nationwide and open to young people aged 13-25 years living with CF. If you would like to know more about this, visit or scan below.

Peer Mentor Training The Pathways programme of research concludes with Peer Mentor Training. This study will focus on a frequently voiced need for peer-to-peer support. Young people and parents have identified a desire to be able to connect with peers who have more experience of health management or healthcare transition.

In response, the Pathways team are in the early stages of a peer mentor training programme where adolescents aged 13-16 years, living with CF may be paired with peer mentors aged 18-25 years living with CF, who have learned to manage their health and have experience of moving to adult care. Mentors will share their experience and offer support to young people preparing to move to adult care. Mentor training will be provided in early 2025. This training programme will be a first step toward the development of peer support resource where an adolescent preparing to move to adult care can avail of social support provided by young people with similar lived experience and whom they can relate. If you would like to know more about this, visit or scan below.

Youth Advisory Panel The Pathways programme of research has engaged with a youth advisory panel from the outset. This is a group of young people aged 16-25 years, living with CF, who offer guidance and feedback in every phase of research activity. The Pathways Youth Advisory Panel continue to guide the research team in the design of research materials, study recruitment and knowledge translation. The progress made to date could not have been achieved without the fantastic Pathways participants, our Youth Advisory Panel, our collaborators and our research teams. For this we would like to extend our sincere gratitude to everyone who has been involved up to this point and who continue in their support, helping ensure we address the issues that are most important young people living with CF and their families. If you would like to know more about this, visit or scan below.

If you would like to find out more about the Pathways Survey Study, visit www.pathwaysresearchstudy.com. If you would like more information on the

Pathways Interview Study

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Pathways Mentor Training

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Newsletter HIT-CF Europe September 2024

The HIT-CF Europe project aims to provide new treatment options to people with cystic fibrosis (CF) and ultra-rare genetic profiles. The project will evaluate the efficacy and safety of drug candidates provided by collaborating pharmaceutical companies in patients selected through preliminary tests in the laboratory on their mini-intestines – also called organoids.

Ultra-rare mutations

Rectal biopsy

Intestinal organoids

Drug testing/ selecting responders

Clinical trial

Enrollment in the CHOICES trial is in full swing We hope you all enjoyed a relaxing summer and were able to spend time with your loved ones. During the summer months, the CHOICES study team and the staff at the local study sites have been hard at work. At this moment, 11 people have been enrolled in CHOICES. Enrolled means that they are taking the active drug or placebo. An additional 5 have been screened. Screened means that all necessary exams have been done and participants are ready to start the trial. The left graph, or “pie” represents al 52 people that need to be enrolled in CHOICES. In blue you see the ones that are already enrolled, in orange the ones that are screened to be enrolled, and in grey the participants that are expected, but not yet screened. CHOICES is currently running in 10 study sites in 7 countries: Belgium, France, Italy, the Netherlands, Germany, Sweden and the UK. On the right “pie” you see how many participants are enrolled (shades of blue) and screened (shades of orange) per country.

Attention to all HIT-CF participants: reconsenting to organoid biobanking

At the beginning of the HIT-CF project, you have undergone a rectal biopsy. You have given your consent, or permission, to culture organoids from this biopsy, and to use and store these organoids for the duration of the project. Several drug compounds have been tested on these organoids, which enabled us to start the CHOICES study.

Worldwide, there is a growing number of people with CF who can be treated with new medications. However, still an important number of people with CF have no treatment opportunities yet. Therefore, further studies are done worldwide by researchers and pharmaceutical companies. Your organoids can be very important for testing new therapies in the future.

The consent you have given, is only valid for the duration of the HIT-CF project. By the end of 2024, the HIT-CF project in its current form will finish. This means that we would have to destroy your organoids if no further action is undertaken.

To learn more about the HIT-CF project, visit www.hitcf.org or send an e-mail to HITCF@umcutrecht.nl

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This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 755021

Newsletter HIT-CF Europe September 2024

The HIT-CF Europe project aims to provide new treatment options to people with cystic fibrosis (CF) and ultra-rare genetic profiles. The project will evaluate the efficacy and safety of drug candidates provided by collaborating pharmaceutical companies in patients selected through preliminary tests in the laboratory on their mini-intestines – also called organoids.

Fortunately, we have found a way to keep your organoids available for future studies by storing them in a new European Cystic Fibrosis Stem Cell Biobank. This does mean that you will need to sign a new informed consent; otherwise, your organoids will still be destroyed.

How do I reconsent? By simply sending an email to HITCF@umcutrecht.nl with either the message: - Yes, I am interested in keeping my organoids available, or - No, I am not interested, please destroy my organoids.

If you indicate you are interested, we will send you a letter with all necessary information, and a new consent form. After we have received your new consent form, we will store your organoids in the new biobank.

Otherwise, we will destroy your organoids according to your wishes. If you feel you first need more information to be able to make an informed decision, please write down your questions and send them to HITCF@umcutrecht.nl.

You might have received this information from your CF doctor as well. Please feel free to discuss this with him or her too.

Good news: ReCode mRNA trial is recruiting!

As we mentioned in the February newsletter, HIT-CF participants that were not selected for CHOICES, will have the opportunity to participate in another study. ReCode Therapeutics has developed an investigational inhaled therapy based on mRNA for people with CF with rare mutations, including nonsense/stop mutations. mRNA stands for messenger ribonucleic acid and contains instructions to tell our cells how to make a protein, such as the CFTR channel. mRNA is not able to alter or modify a person's genetic makeup (DNA). In people with CF, the CFTR protein is not functioning well or isn’t there at all. By delivering correct copies of CFTR mRNA to the lung cells, this problem can be bypassed, and the cells can once again start making functional CFTR channels.

The innovative inhaled mRNA therapy, which is called RCT2100 for now, is currently being tested in people with CF for the first time. This process is also known as a phase 1 study. A phase 1 study focuses primarily on finding the right dose of the drug and evaluating the safety. Participants to a phase 1 study may or may not have personal benefits from the treatment, but it is not the primary goal at that point. Moreover, it cannot be guaranteed that participants will automatically be able to take part in later phases of the study that do focus on investigating effectiveness. Nevertheless, this is a very important, if not the most important phase of the clinical research. Without sufficient safety data, the experimental drug cannot progress to the next phase. Therefore, it is very important to find enough participants.

During this study, the researchers want to determine how much RCT2100 can be administered, to find out whether RCT2100 is well tolerated, as well as how the body uses RCT2100 and how RCT2100 affects the body. To do so, the safety of several different doses of RCT2100 will be tested. Each participant is guaranteed to receive the experimental therapy; in this part of the study, there is no control group receiving a placebo or dummy drug.

This summer, ReCode obtained approval to perform the study in Europe, and they are now looking for participants.

Participating in this study means that you will have to visit the study centre about 17 times and requires blood draws, lung function testing and/or other measurements. The study will run in the Netherlands (Utrecht), France (Paris Necker, Toulouse and Montpellier) and the UK (Leeds, London King’s College Hospital, Nottingham, Southampton, Cambridge and Birmingham). If you don’t live in one of these countries, you can still participate if you are able and willing to travel to one of the study sites. For more information, you or your CF doctor can contact the HIT-CF team via HIT-CF@umcutrecht.nl.

To learn more about the HIT-CF project, visit www.hitcf.org or send an e-mail to HITCF@umcutrecht.nl

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This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 755021

My Legacy Would you consider including a gift in your Will for Cystic Fibrosis Ireland?

Cystic Fibrosis Ireland have teamed up with LawPlus Solicitors to provide subsidised wills for anyone who wants to take the time to draft one of the most important documents of your life.

According to recent research, only 27% of people in Ireland currently have a Will, significantly lower than in the UK, where 44% of people have already made their arrangements. This statistic highlights the importance of making your Will and the peace of mind this easy step will provide you and your family.

A Will not only ensures your loved ones are taken care of but also allows you to extend your support to a charity like Cystic Fibrosis Ireland. By including a gift in your Will, you leave a lasting legacy that will make a difference for years to come.

Whether large or small, legacy gifts in your Will can ensure support for the future of those living with CF. With a simple, affordable process, creating a Will can give you peace of mind knowing your wishes are carried out and by leaving a legacy gift, you can continue to make a difference long after you are gone.

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Why Make a Will?

Creating a Will is about more than just dividing up assets; it’s about ensuring your family is protected and your personal wishes are respected. For example, a Will allows you to: • • •

Provide for your loved ones, ensuring they receive what you want them to have.

Assign guardians for your children and ensure their future care is secured.

Protect your property and assets and reduce the impact of inheritance tax.

Most importantly, making a Will is often much simpler and more affordable than many people anticipate.

During November, we are encouraging you to schedule a consultation with Lawplus Solicitors LLP or your local solicitor, who can guide you through the process. Cystic Fibrosis Ireland is also proud to partner with MyLegacy.ie, which is backed by hundreds of legal firms across Ireland. These solicitors can provide expert advice on how to structure your Will, including the option of leaving a legacy gift to a cause that matters to you.

Considering a Legacy Gift to Cystic Fibrosis Ireland Including a legacy gift in your Will is an extraordinary way to ensure your values live on. Whether large or small, legacy gifts are tax-free and can make an immense difference to the lives of those living with CF. In Ireland, while 14% of people with a Will have included a charitable gift, there’s still a significant opportunity for growth, with 51% of people admitting that they simply hadn’t considered this option before. Legacy gifts are a chance to give far more than you might ever be able to in your lifetime, and they provide the security that allows charities like Cystic Fibrosis Ireland to plan for the future.

Why People Leave Legacy Gifts Recent research indicates that the most common reason people leave a legacy gift is their desire to support a cause they care deeply about. 39% of respondents say this is their motivation, while 27% leave a legacy gift to honour a loved one. For many, it’s an opportunity to make a lasting impact, ensuring their values live on through charitable work.

Including a legacy gift in your Will can help Cystic Fibrosis Ireland continue its mission, funding groundbreaking research, specialised medical care and essential support services for people with CF. By leaving a gift, you will directly contribute to: • • • • •

Advancing research towards finding a cure for Cystic Fibrosis.

Funding specialist CF multidisciplinary roles in hospitals across Ireland.

Supporting Cystic Fibrosis units around the country, including inpatient, outpatient, and day care centres.

Directly assisting individuals with CF through grants for exercise, transplants, fertility counselling, and bereavement support.

Offering counselling, education, and advocacy services to improve the lives of people living with Cystic Fibrosis.

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Subsidised Wills in November To help make the process easier and more accessible, we have teamed up with LawPlus Solicitors LLP during November to offer subsidised Wills. Throughout November, they will write your Will at a reduced cost of €140 for an individual or €195 for a couple. This offer is available for a standard Will, ensuring that you can take this crucial step without delay or unnecessary expense. Contact LawPlus Solicitors LLP at info@lawplus.ie or 01 276 5226 to arrange your appointment.

Leaving a Legacy is a Personal Decision We understand that leaving a legacy gift is deeply personal. Some people prefer to notify us in advance that they have included Cystic Fibrosis Ireland in their Will, while others may choose to keep it private until

1. Get expert advice

2. Look after loved ones first

3. Consider a legacy to a charity

the time comes. Whatever your preference, rest assured that your gift will have a lasting impact on the lives of future generations living with Cystic Fibrosis in Ireland.

Take the First Step – Make an Appointment 1. Get expert advice: Taking that first step toward making your Will can seem daunting, but with the right guidance, it can be a smooth and empowering process.

2. Look after loved ones first: A Will ensures your family and friends are cared for, your property is protected, and inheritance tax is minimized.

3. Consider a legacy to charity: Whether large or small, including a legacy gift to Cystic Fibrosis Ireland can be life-changing, allowing you to continue supporting a cause you care about long into the future.

Your Legacy Can Change Lives For more information on leaving a legacy gift to Cystic Fibrosis Ireland, please contact us for our advice leaflet, “Your Legacy Could Be Life-Changing”.

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Spectrum / Summer 2024

FUNDRAISING: For further details on any of our fundraising events, visit our website www.cfireland.ie, or contact the CFI Fundraising Team on (01) 496 2433 or email fundraising@cfireland.ie.

October 27th - Irish Life Dublin Marathon For all the marathon runners, don’t miss the Irish Life Dublin Marathon on Sunday, 27th October.

If you have secured a place in the Marathon and would like to take part on behalf of the Cystic Fibrosis Ireland Team, please register your interest NOW on our website at www.cfireland.ie. We will contact you and provide you with your Cystic Fibrosis Ireland shirt for the day and fundraising pack!

We are also delighted to announce that we have recently secured 25 guaranteed places for the Irish Life Dublin Marathon in 2025. To secure your place, please contact us ASAP by email fundraising@cfireland.ie or call (01) 496 2433.

November 3rd - TCS New York City Marathon Best of luck to all of our runners who are taking on the New York City Marathon in November. We look forward to seeing all the lovely photos.

We will have a limited number of places available for the 2025 TCS New York City Marathon once registration opens. Keep a close eye on our Social Media and make sure to register your interest on our website www. cfireland.ie as soon as details are announced to secure your place.

For further details please email fundraising@cfireland.ie.

www.cfireland.ie

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December 6th Christmas Jumper Day 4 CF

At last, a good reason to wear your favourite Christmas Jumper to work!

On Friday December 6th, Cystic Fibrosis Ireland are asking organisations big and small to support Christmas Jumper Day 4 CF by wearing their favourite Christmas Jumper to work. Love them or hate them, they are here to stay! Whether you’re in the ‘Seasonal Christmas Jumper Club’ or the ‘Bah Humbug Brigade’, we ask you to please support people with Cystic Fibrosis in Ireland on Friday, 6th December – Christmas Jumper Day 4 CF! To take part, all you have to do is follow five simple steps; 1. Ask your staff or colleagues to wear their most seasonal looking Christmas Jumper to work on Christmas Jumper Day 4 CF - Friday 6th December, in return for a €5 donation to support people with Cystic Fibrosis in Ireland.

2. Bah Humbug Donation! If someone does not wear a Christmas Jumper on the day, ask them to make a donation of €10 in return for our coveted Bah Humbug badge 3. Ask your company if they are willing to match the monies raised!

4. On the day, make sure to take lots of photos!

5. Tag us in all your social media posts using @cfireland don't forget the hashtag #ChristmasJumperDay4CF. Don’t be left out in the cold and red nosed this Christmas Jumper Day 4 CF. You can register now by scanning the QR code or visiting our website CFIreland.ie.

Once registered, we will contact you to organise delivery of your Christmas Jumper Day 4 CF essentials pack. For further information, please call Hazel on (01) 4962433 or email hdoran@cfireland.ie

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Spectrum / Summer 2024

April 11th, 2025 – 65 Roses Day A date for your diaries - 65 Roses Day takes place on Friday 11th April, 2025. Keep an eye on our website and social media over the coming weeks, where we will be announcing details of how you can get involved.

This could involve volunteering to help us with our collections nationwide on the day, taking part in a 65 Roses challenge or simply donating online to support our largest fundraising campaign.

This year we raised an amazing €305,471.28 and with your support would love to make 65 Roses Day even bigger and better next year!

April 27th, 2025 – TCS London Marathon We have secured a limited number of places for the TCS London Marathon in April next year.

To secure a place register your interest now on our website at www.cfireland.ie.

For further details please email fundraising@cfireland.ie.

June 1st, 2025 – Vhi Women’s Mini Marathon The Vhi Women’s Mini Marathon takes place on Sunday, 1st June next year. Why not join us and take part in this very special event while raising vital funds for Cystic Fibrosis Ireland! Mark the date in your diary now and keep an eye on our website and social media for details of how to register.

For further details, please contact us on 01 4962433 or email fundraising@cfireland.ie

Tax Reclaim You can maximise your donation to Cystic Fibrosis Ireland at no extra cost. If you have donated €250 or more during any year over the past 5 year all you have to do is sign and return the CHY3 form. We can then claim an extra 45% on top of your donation from revenue.

Please contact us at fundraising@cfireland.ie or 01 4962433 if you have any questions!

www.cfireland.ie

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September 11th to 14th, 2025 – 10th Annual Malin2Mizen Cycle4CF We are absolutely delighted to announce that our 10th Annual Malin2Mizen Cycle4CF will take place on Thursday 11th to Sunday 14th of September next year.

Malin2Mizen Cycle4CF will begin at Malin Head on Thursday 11th, September and finish in Mizen Head on Sunday 14th, September 2025. This amazing event has raised over €1m since it started in 2016 and you will be supported every ‘pedal’ along the way, by an incredible support team.

Register your interest now to secure your place for our 2025 cycle on our website at www.cfireland.ie. For further information please call Hazel on (01) 496 2433 or email fundraising@cfireland.ie.

Pictured here: The Malin2Mizen Cycle4CF committee presenting a cheque for €1,165,476 to Sarah Tecklenborg, Interim CEO Cystic Fibrosis Ireland. R-L: Sarah Techlenborg, Lucy Hanlon, Caroline Heffernan, Bernie Priestley, Fergal Smyth, Hazel Doran

All Year Round International Treks For the really adventurous looking for the challenge of a lifetime, International Treks are available across the year to climb Kilimanjaro, Machu Picchu and Everest Base Camp.

Register your interest to take part on our website www. cfireland.ie or email fundraising@cfireland.ie for more information.

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Spectrum / Summer 2024

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:

Head2Head Walk Cystic Fibrosis Ireland would like to say a huge thank you to everyone who took part in the 12th Annual Head2Head Walk on Sunday 8th September. It was a fantastic day with 400 people taking part.Lunch was provided at the half way point in Sandymount courtesy of our wonderful sponsors Around Noon Foods, Fyffes and Spar Leeson Street. We would also like to say a huge thank members of the BNI Grosvenor Group for sponsoring this year’s energy bars. The walkers then continued on their way towards the finish in Bray where they received their finishers medal.

A huge thank you to Luke Kennedy, Glen Mc Donnell, Jem & Lorraine Downes, Mary McCarroll, Denise O’Brien and their team of volunteers who put an enormous amount of work into organising this event with CFI and ensuring it ran smoothly on the day. Special thanks also to the Gardai, the bus drivers, Daniel Kennedy our van driver and The Order of Malta Ireland – Blackrock Branch who provided medical support on the day. We would especially like to thank all our walkers for turning out yet again in huge numbers.

www.cfireland.ie

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We hope you had a great day and your fundraising makes an enormous impact on the ability of CFI to support people with CF in Ireland. If you have sponsorship money to lodge from the event, you can do so using the bank account details below. Please remember to include your name and ‘H2H’ as a reference so we can receipt accordingly.

IBAN: IE59 AIBK 931071 08578593 BIC: AIBKIE2D We look forward to seeing you all at our 13th Annual Head2Head Walk on Sunday 7th September, 2025!

Malin2Mizen Cycle4CF Huge congratulations to everyone involved in Malin2Mizen Cycle4CF 2024 which took place in September and raised an unbelievable €135,114.18 to date.

Over €1m has now been raised by the Malin2Mizen Cycle4CF since the event started in 2016 as a follow on from a cycle in memory of Triona Priestley which took place in 2015. A huge thank you the members of the Malin2Mizen Cycle4CF committee for organising the event each year, all the cyclists for their amazing fundraising, and the wonderful support team. It is your commitment and dedication year on year that makes this event the success it is today.

Thank you to our fantastic sponsors including Drive You, Maxol, Dunnes Stores, Spar, Donegal LSP, Todd’s Drinks, Clonakilty Food Company and our Dublin West, Mayo, Galway and Cork Branches.

Registration of interest is now open for our Malin2Mizen Cycle4CF 2025 cycle which takes place from Thursday 11th to Sunday 14th September at www.cfireland.ie Sign up NOW for this adventure of a lifetime.

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Spectrum / Summer 2024

CFI International Walk in Andalusia Cystic Fibrosis Ireland would like to say a huge thank you to each of the walkers who took part in the 2024 CFI International Walk to Andalusia.

A whopping total of €126,472 has been raised to date! We look forward to seeing you all for the international walk in 2025!

CFI would also like to say a special thank you to the Walk Committee who work all year round to ensure the CFI International Walk takes place each year!

Keep an eye on our website and social media channels over the coming weeks for more info on our 2025 walk and if you have any questions drop Hazel an email at hdoran@cfireland.ie or call 01 496 2433.

Thomas Henry’s Money Tree Massive thanks to Thomas Henry who ran a novel fundraiser as his 65 Roses Challenge, a money tree which raised €2544.50 for Cystic Fibrosis Ireland.

His online raffle included amazing prizes with 1st prize being a money tree, yes exactly what is says, a tree covered in money.

Well done Thomas on coming up with such a creative idea for a 65 Roses Challenge and the huge amount of money raised.

Frankie Leonard’s Captain’s Prize at Ballinasloe Golf Club A huge thank you to Frank Leonard for choosing Cystic Fibrosis Ireland as his chosen charity for the Captain’s Prize event on July 13th in Ballinasloe Golf Club. Frank and the participants raised a total of €1,620 for Cystic Fibrosis Ireland.

www.cfireland.ie

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Spectrum / Summer 2024