Research

Research

  • 18th National Cystic Fibrosis Clinical Meeting

    The 18th National Cystic Fibrosis Clinical Meeting took place in Killarney, Co. Kerry on the 1st and 2nd February 2018.

    Over the course of the two day meeting, leading CF clinicians and researchers from around the world outlined their work to address a particular issue in CF care. Topics discussed included: the treatment of aspergillus fumigatus infections and advances in fertility care in CF. An update was also provided on the progress of the National Clinical Programme which has set up six sub-groups each dealing with a different area of CF care including post-transplant care and CF and Fertility.

  • Information Evenings

     

    CFI, in partnership with the Midlands Branch, held two information evenings for Parents & Carers in Portlaoise (October 21st) and Dublin (November 4th). The evenings primarily focused on new treatments in the pipelines for people with Cystic Fibrosis.

    Dr Ed McKone, CF Consultant from St Vincent's University Hospital, Dublin, was keynote speaker and shared his expert insight and knowledge regarding the treatment pipeline for people with CF in Ireland.  

    Click play on the videos below to view the presentations and discussions from the Portlaoise Information Evening, which was live streamed on the night. 

    Welcome Address by Midlands Branch

     

    Progress Update by Philip Watt, Chief Executive, CFI

     

    New Therapies in CF by Dr. Ed McKone, CF Consultant, St. Vincent's Hospital

     

    The Approval Process for New Therapies by Katie Murphy, Research & Development Officer, CFI

     

    Question & Answer Session

     

    More Information Evenings are planned for 2016, with details to be announced soon.

     
     
     
     
  • Channel 4 Report on New Potential Treatment Option for PWCF

    research2

    On the 11th of November, Channel 4's Jon Snow reported about recent Phase 1 trial results of potential treatment option for people with CF. The research project, which is being undertaken by Dr Anil Mehta from the University of Dundee, was described as a 'Eureka moment' that offered the hope of a 'cystic fibrosis cure'. Understandably there was a stir of excitement amongst the CF community after the report was released. However, we have been having a closer look at this promising research and would like to take this opportunity to highlight some important points about this research news.

    According to the University of Dundee's press statement this research is an early stage clinical study being undertaken in Italy, which has trialled the combined use of two drugs already licensed for use in other conditions – Cysteamine, and epigallpcatechin gallate (EGCG) – involving 10 people with CF, with two copies of the DF508 gene. This statement from the university says that the study found that the combination of the two aforementioned drugs reduced inflammation in 9 out of 10 patients' airways and also reduced sweat chloride levels Ñ these promising results certainly warrant further investigation but it is important to remember this is an early stage trial with a very small sample.

    The CF Trust, who provided funding to the earlier stages of this research, have also spoken out around the report and have said "talk of a potential cure was misplaced", to which Dr Mehta (lead researcher) also agreed.

    What happens next?

    Dr Mehta is now hoping to bring this work to a larger Phase 2 research study involving 120 patients. The CF Trust have also told us that Dr Mehta is planning to submit a funding application to the UK Government's Medical Research Council for support, however, there are a great number of regulatory hurdles the work will have to overcome first.

    Although this initial research indicates positive results, it is important to remember that drug development is an incredibly slow and expensive endeavour and positive results at the early stages of development does not guarantee success in clinical terms further down the line.

    In summary, the potential outcomes from this project are exciting, however it is clear that the media's portrayal of this research project as a possible cure for Cystic Fibrosis is premature and misleading.

    For more information:

  • Drug Iceberg 2.0 Published

    In February 2018 the Irish platform for patient organisations, science and industry (IPPOSI) and the Medical Research Charities Group (MRCG) announced the publication of the second “Drug Iceberg” Report. This report lay out key recommendations including a call for the development of a new national strategy on access to medicines. 

     

     

     

  • Education and Conference Grant

    The purpose of the Cystic Fibrosis Ireland (CFI) Education and Conference Grant is to provide financial assistance to CF health care professionals, researchers and patient representatives to attend and participate in Cystic Fibrosis specific conferences and training courses.

       

    To find out more about this grant and to access the application form, please click on the following link: Education and Conference Grant 2016.

    For any queries regarding this grant please contact Daniel Costigan on 01 496 2433 or email This email address is being protected from spambots. You need JavaScript enabled to view it.

  • Extension of Orkambi to children aged 6-11

    The recent decision of the European Medicines Agency (EMA) to extend the CF drug “Orkambi” to children aged 6 to 11 years old, on the basis of clinical research emerging from two Phase III studies (NCT02514473 and NCT01897233), means that this ground breaking drug which had previously only been available to those aged 12 years and older with two copies of the F508del mutation, will now also be available to children aged 6-11 (subject to the advice of their Consultant).

    Orkambi treats the most common CF gene alteration/ mutation in Ireland (and the world) that is people who have two copies of the F508del alteration. Orkambi was approved for reimbursement by the Irish Government in April 2017. As part of that agreement, in a unique “pipeline deal”, it was agreed that future extensions of Orkambi or Kalydeco (including this EMA decision to extend Orkambi to children aged 6-11), would be made available to people with CF in Ireland.

    Commenting on the extension of Orkambi to children aged 6-11, the Minister for Health Simon Harris said:
    “It’s very positive news that Orkambi is now licenced and available for reimbursement in Ireland for CF patients aged 6 to 11.”

     

  • Factors Predicting Psychological Distress in Caregivers of Children Diagnosed with Cystic Fibrosis

    Family-Support-Services 0

    Are you interested in helping researchers in Ulster University to understand more about the challenges parents experience in caring for a child with cystic fibrosis?

    Are you…

    • over 18 years of age and under 65?
    • the parent of a child who has received a diagnosis of Cystic Fibrosis?
    • living in Ireland?
    • interested in giving up 30 minutes of your time to answer a confidential survey?

    Your assistance will help us to understand more about parental levels of distress, coping strategies, supports, and quality of life which will help in identifying support requirements for parents caring for children with cystic fibrosis. 

    An information sheet is included with the survey, which outlines:

    • The Purpose of the Study
    • Potential benefits and risks in taking part
    • What you will be asked to do if you take part
    • What will happen the results of the study
    • Who is being invited to take part
    • Funding and payment in relation to the study
    • Your rights regarding taking part and withdrawing from the study
    • Who has reviewed the study
    • Confidentiality of your answers - they will be anonymous!
    • What to do if you have a comment, question or complaint about the study

     

    How do I take part in this study?

    If you would like to take part, three options are available:

    1. Do the survey online now by clicking on this link – the information sheet about the study is also available on this link.

    or

    2. You can ask in your CF centre for a paper version of the questionnaire (a limited number of copies have been made available to some centres)

    or

    3. You can contact Greg Stynes on This email address is being protected from spambots. You need JavaScript enabled to view it.

  • Focusing on Patient Participation; from Drug Development to Patient Access

    perspective

    November was certainly a busy month, perhaps it is an unofficial 'conference month', as there were numerous exciting meetings and conferences happening throughout Europe, two of which primarily focused on access to new therapies. First up on the agenda was the European Congress for the International Society for Pharmacoecomonics and Outcomes Research (ISPOR), which was held Amsterdam. Last year Dublin played host to this impressive event which sees over 4,000 delegates gathering from across the globe to discuss the latest in health research, drugs/therapy development, health economic assessments, etc. In 2013 the breakthrough CF drug Kalydeco was certainly a hot topic throughout the event, which was reflected in the programme of events. The opening plenary session in 2013 was 'The Patient and Health Technology Assessment: Challenges and Opportunities', using Kalydeco as an example. It was remarkable how surprised the delegates were to hear a patient speaking about this topic and even more startling how few patient representatives were in attendance. Of the 4,000 delegates in attendance, an expert-patient round table discussion of about 20 people was established to promote the patient perspective in all aspects of medicines development. One year later and the 2014 agenda certainly had a greater focus on the need to capture more 'real-life', 'patient-centric' evidence and to have it included in Health Technology Assessment (HTA) when assessing the effectiveness of new therapies. It is reassuring to hear the discussion about patient involvement is happening from the top down, with senior members of organisations like the European Medicines Agenda (EMA), the FDA and other regulatory bodies advocating for greater involvement of patients in all aspects of medicines development. 

    "Growth in the number of new therapies in the pipeline for CF means it is more important than ever to keep up-to-date on current practices in health assessments and also ensure that patient views are constantly represented with all stakeholders; industry, regulators, and payer," Katie Murphy, Research and Development Officer, CFI.
    Similarly, the World Orphan Drug Congress 2014 took place last month which starkly reminds us of the difficulties facing rare and ultra-rare diseases with regards to drug development, pricing and patient access. Speaking with patient representatives from ultra-rare disease groups is a reminder just how far we have come in the treatment and management of CF. Many illnesses have no treatment options available for patients, with little or no funding available or being invested in research/drug development. The Cystic Fibrosis Foundation's model of venture philanthropy model, through which the organisation provides upfront funding for pharmaceutical companies to help reduce the financial risk of developing drugs to treat CF, is a great example of patient groups leading the way in medicines development – but for some patient groups, representing as few as 20 patients in a country, this is not a feasible model to adopt. So although there has been a growing focus on cost-effectiveness accessing new therapies for patients, for many rare disease groups, accessing new therapies is not currently on their agenda. 

     

    Growth in the number of new therapies in the pipeline for CF means it is more important than ever to keep up-to-date on current practices in health assessments and also ensure that patient views are constantly represented with all stakeholders; industry, regulators, and payers. 

     

    For more information:

  • Joint North-South Rare Disease Conference

    To celebrate International Rare Disease Day (28th February 2018), a Joint North-South Rare Disease Conference took place in Riddel Hall, Belfast on Monday 5th March 2018. The Conference offered participants the opportunity to be part of a global call on policy makers, researchers, companies and healthcare professionals to increasingly and more effectively involve patients in rare disease research.

    The following is the opening speech delivered by Philip Watt, Chair of the Rare Disease Task Force; Chair of the Medical Research Charities Group and CEO of Cystic Fibrosis Ireland on the occasion of the North/South Rare Disease Conference, 5 March 2018 in Belfast.

    Good morning everyone. Thanks to everyone who made it today, especially for those of you had to overcome an added level of adversity due to the combination of disability, health and weather.

    I am very proud to be here today as a member of the Rare Disease Taskforce to be part of International Rare Disease Day.

    Our key message today remains ‘If you or a loved one has a rare disease, you are not alone’.

    We hope that today will in time be seen as an important step on the long and often desperate journey for equal rights for people with rare diseases in Ireland, North and South.

    Today we are once again in Belfast as we were in 2017 in Dublin to renew both important friendships and joint working that has been feature of patient groups on the island of Ireland. I particularly would like to acknowledge our friendship with Fiona McLoughlin and the NIRDP. We also take this opportunity to congratulate Christine Collins who was recognised in the 2018 New Year’s honors list for her work on rare diseases in Northern Ireland over many years.

    My name is Philip Watt and my presence here today is as Chairperson of the rare disease taskforce, where I represent the Medical Research Charities Group and Cystic Fibrosis Ireland where I work. The Task Force is also inclusive of Rare Disease Ireland and IPPOSI and the Irish Platform for Patients, Science and Industry

    This taskforce, established 6 years ago, has brought together patient groups and key stakeholders from industry, academia and clinical services who have shared a vision of a better deal for people with rare disease and their families.

    For too long many parents in Ireland North and South have had to cope with a sick child with a rare disease but with little information as to what the disease is, let alone where they might find treatment.

    For too long GP’s have been frustrated by being unsure where the best center of expertise is to treat a particular rare disease.

    For too long there has been an insufficient sharing of health resources North and South on our small Island. There had been some signs of change in cooperation in respect of children’s heart surgery, lung transplants and cataract operations for example.

    In recent years there have also been several important rare disease health research partnerships North and South that were funded through EU programmes such as Horizon 20/20.

    We urge that these beginnings of cooperation and joint research projects will not be up-scuttled or side-tracked by Brexit and the continuing lack of an administration in Northern Ireland. These are very worrying times for patient groups on both sides of the border.

    I am delighted to see the support of the Northern Ireland Human Rights Commission for today’s conference. The participation of Chief Commissioner Les Allamby sends out an important message on both the importance of PPI- Public and Patient Involvement in health policy and the need to locate rare diseases more robustly within a human rights and equality framework.

    In this context, we take this opportunity for the Minister for Health responsible for disability in the South, Finnian McGrath TD, to ensure that Ireland fulfils its oft repeated promise to ratify the UN Convention on the Rights of Disabilities that was signed by Ireland in 2007.

    A key part if this convention (article 19) is concerned with the ambition for Independent Living for all adults living with a disability or chronic disease. Ireland remains the only county in Europe not to have ratified this vital Convention, which sends out poor message nationally and internationally.

    In organizing today’s conference we once again draw attention and celebrate the fantastic work of rare disease patient groups in Ireland North and South.

    There are some 7,000 rare diseases in Ireland

    The fortunate few – perhaps around 30 or 40 - have a patient group to represent them.

    Many patient groups are reliant on volunteers or one or 2 staff members at most. They are understaffed and overworked but they do an amazing job. In many cases they are run by the mothers or fathers of someone with a rare disease. Please show your appreciation for the work of these voluntary groups.

    Today we also call on the Irish and British government to bring in urgently needed reforms to make drug therapies for those with a rare disease more accessible in Ireland North and South. For example, it is very unfair that the border can decide your access to a ground-breaking life enhancing drug. For example, the decision by the Irish government to approve the Cystic Fibrosis Drug Orkambi is in stark contrast to the British Government which continues to deny this vital drug to England, Scotland, Wales and Northern Ireland. Vice-versa the decision of Stormont to approve the drug Translarna for those with Muscular Dystrophy Duchenne is in stark contrast to the Irish Government which continues denies this drug to two patients.

    Today we also take this opportunity to call on the British and Irish Governments for better support for health research and clinical genetic services that have such an impact on people living with a rare disease. Clinicians and researchers on the island of Ireland have been increasingly been involved in fantastic research focused on rare diseases – this work must continue to be fostered in spite of recent political challenges. 

    Today as we enter the final year of the of the operation of the existing rare disease plans north and south we acknowledge progress to date but we also call for the many remaining promises in these plans to be fully implemented and we call for second plans to be developed, including a focus on North/South and East/West sharing of the limited existing resources for those living with a rare disease.

    Finally we call on both the Irish and the British Governments to tackle the often unstated or unintended carelessly though through mindset that ‘it is better to treat the many rather than the few’. For example - that it is better to give a million people a new drug at a lower price than provide funding for an expensive rare disease drug.

    We say again today ‘We must continue to treat the many and the few equally’ and to do otherwise is a breach of human rights.

    Philip Watt
    Chairperson Rare Disease Taskforce

    Medical Research Charities Group (MRCG)
    CEO Cystic Fibrosis Ireland.

     

  • Keeping up with Clinical Trials!

    There has been a very welcome surge in the number of clinical trials being conducted globally over the past decade – and Cystic Fibrosis is very much a part of this growth. Due to increasing awareness and interest in new therapies and clinical trials we have seen an increase in the number of queries around these studies.

    We are always delighted to provide support and information but another great way to keep up-to-date and to read about on-going clinical trials is to visit https://clinicaltrials.gov. ClinicalTrials.gov is a registry and results database of publicly and privately supported clinical studies of human participants conducted around the world.

    Red Blue Pill

    If you log on to the above website and search 'Cystic Fibrosis' – you will see detailed descriptions of status of trials, which phase they are in, where they are taking place, results (if any), etc. It is a great tool for interested people to stay informed and up-to-date.

  • Launch of CFI “Independent Living” Report

    On Wednesday 4th April 2018 CF Ireland launched a new and groundbreaking report on “Independent Living” which highlights major improvements in the quality and the length of lives of people with CF over two decades (1998-2017). The report shows that the sustained investment in better CF services and medications in recent years has made a very significant difference to those with CF in Ireland – but there is also much more that can and should be done.

    At the core of the Report ‘Independent Living and Cystic Fibrosis’ are two surveys undertaken by CFI in 1998 and 2017. Around one quarter of the adult CF population participated both surveys. Corroborative data was also drawn from other sources such as the CF Registry of Ireland. 

    Amongst the key points noted in the report are:

    o   In 1998 adults with CF were not expected to be in any employment. In 2017, a remarkable 54% of those surveyed were in full-time or part-time work. 

    o   In 1998, 74% of respondents lived with their parents in their parents’ house. By 2017 this figure had reduced to 43%. 

    o   In 1998 adults with CF were not expected to have children of their own. In 2017 (thanks to better health and the introduction of IVF) 26% of the adult CF population aged 21 or over surveyed had children

    o   In 1998 only 8% of those surveyed were married, compared with 26% in 2017

    o   The percentage of those obtaining third-level qualifications has increased from 38% in 1998 to 49% in 2017

    o   The median age of death in Ireland was only 17 years of age in 1998. By 2015 this had increased to 30 years for the first time.  


    Philip Watt CEO of CFI stated:

    “The findings are in many ways remarkable and send out a strong message of hope. The main message is that adults with CF are living much more fulfilled and independent lives in 2017 compared with 1998. They are much more likely to have a job, have better education attainment, to have their own home and to even have a family of their own.

    “The improvements in length and quality of life are closely linked to improvement in services such as dedicated CF hospital centres which we have fought for; improved rates of double lung transplantation and more recently access to new ground-breaking drugs such as Kalydeco and Orkambi. The report shows that with sustained investment over a number of years people with a chronic and fatal disease such as CF can lead longer and better lives.” 

    “However key gaps remain. The Government promised a dedicated CF in patient unit in Beaumont Hospital commencing in 2017. There has been no progress on this commitment in the Programme for Government and understaffing in major CF centres remains an issue.” 

    The ‘Independent Living and Cystic Fibrosis’ Report covers a number of key issues:

    Relationships and Housing
    In 1998, 74% of respondents lived with their parents in their parents’ house. By 2017 this figure had reduced to 43%. 

    In 1998, 87% of adults surveyed were ‘single’ compared with 39% in 2017. 

    In 1998 adults with CF were not expected to have children of their own. In 2017 thanks to better health and access to IVF, 26% of the adult CF population aged 21 or over have children. 

    Employment 
    In 1998 adults with CF were not expected to be in employment because life expectancy was much shorter. However, by 2017, a remarkable 54% of those surveyed were in full-time or part-time work. These jobs range from journalism to the caring professions, from IT to accountancy. 

    However, major challenges remain. 14% of all respondents in the 2017 survey remain  ‘too ill to work’. Many others said the potential loss of a medical card or sudden loss of Disability Allowance when entering a job remains a major deterrent to employment and independent living as their health comes first. 

    There is also evidence of improved understanding by employers about the potential contribution of people with CF in the workplace. In 2017, 92% of people with CF surveyed were prepared to let their employer know they had CF compared with only 44% in 1998. However, the report also shows those in lower paid employment can still be treated callously by employers. 

    Education and Mobility:
    In 2017, people with CF are still missing out on school and college because of their illness. However, the percentage of those obtaining third-level qualifications has increased from 38% in 1998 to 49% in 2017. 

    In 1998, 82% of those surveyed were reliant on public transport. By 2017 only 19% of journeys made by people with CF were on public transport. This reflects a much greater access to/ownership of cars and an increased wariness of the risk of infections in confined places, such as buses. 

    Foreign travel: 
    For leisure is increasingly the norm for people with CF, through travel insurance can be difficult to access at times.

    Starting a family: 
    The report shows that almost all men with CF and some women with CF will need IVF treatment to commence a family.  However, there are presently no state supports for the needed IVF treatment that costs about €5-6,000 a time.

    The publication of the report comes ahead of the national fundraising day for Cystic Fibrosis Ireland, 65 Roses Day, on Friday 13th April. With funds raised helping people with cystic fibrosis to live independently in Ireland, the public is being urged to lend its support by buying a purple rose for €2 or donating online at 65rosesday.ie.

    To download a copy of the "Independent Living and Cystic Fibrosis" Report, click on the image below.

     

  • Patient Experience on Ivacaftor (Kalydeco)/Lumacaftor

    writing letter
     

    Cystic Fibrosis Ireland is circulating a survey (link below) to enable us to gain an insight into the experiences of people with Cystic Fibrosis who are currently receiving either Ivacaftor (Kalydeco) or the Combination Therapy of Ivacaftor (Kalydeco) & Lumacaftor as part of their CF treatment regime.

    We are asking:

    People with CF, taking either Ivacaftor (Kalydeco) and/or Lumacaftor, over the age of 16 to complete the surveyParents of people with CF, taking either of the above treatments, aged 15 years and under to complete the survey on their child's behalf.

    If you/your child are not in receipt of Ivacaftor (Kalydeco) and/or Lumacaftor please feel free to ignore this survey.

    Why is your participation so important?

    Growth in the number of new therapies in the pipeline for CF means that it is more important than ever to keep up-to-date on current practises in health technology assessments and to ensure patient views are constantly represented with all stakeholders; industry, regulators and payers. This survey is a tool which can help to ensure we are representing your views and your real-life experiences throughout all stages of health assessments.

    All information received will be treated with the utmost care and respect and all participants have the opportunity to remain entirely anonymous.

    Click here to take this Survey

    Your time and participation is greatly appreciated

     

  • Patient Experience on Ivacaftor (Kalydeco)/Lumacaftor

    writing letter
     

    Cystic Fibrosis Ireland is circulating a survey (link below) to enable us to gain an insight into the experiences of people with Cystic Fibrosis who are currently receiving either Ivacaftor (Kalydeco) or the Combination Therapy of Ivacaftor (Kalydeco) & Lumacaftor as part of their CF treatment regime.

    We are asking:

    People with CF, taking either Ivacaftor (Kalydeco) and/or Lumacaftor, over the age of 16 to complete the surveyParents of people with CF, taking either of the above treatments, aged 15 years and under to complete the survey on their child's behalf.

    If you/your child are not in receipt of Ivacaftor (Kalydeco) and/or Lumacaftor please feel free to ignore this survey.

    Why is your participation so important?

    Growth in the number of new therapies in the pipeline for CF means that it is more important than ever to keep up-to-date on current practises in health technology assessments and to ensure patient views are constantly represented with all stakeholders; industry, regulators and payers. This survey is a tool which can help to ensure we are representing your views and your real-life experiences throughout all stages of health assessments.

    All information received will be treated with the utmost care and respect and all participants have the opportunity to remain entirely anonymous.

    Click here to take this Survey

    Your time and participation is greatly appreciated

     

  • Cystic Fibrosis Ireland-funded research to study the effect of exercise in the management of patients with cystic fibrosis undergoing lung transplantation

    Patient-centered research will examine the role of exercise in the management of patients with mild, moderate and severe cystic fibrosis

    With 65 Roses Day upon us, Cystic Fibrosis Ireland today announced an exciting new initiative within MedEx, the chronic illness rehabilitation programmerun by Dr. Noel McCaffrey in Dublin City University (DCU), following the recommendation of the Head of the Mater Lung Transplant Unit, Professor Karen Redmond.   This will be co-funded by the Mater Foundation with funds raised by family and friends of the late Becky Jones.    

    We are delighted to announce that Nicola Hurley has been selected for a four year PhD to carry out pioneering research into the role of exercise and its effects on physical fitness, muscle strength, bone health and quality of life in patients with cystic fibrosis (PWCF). 

    In Ireland, 1 in 10,000 people live with CF, while 1 in 19 people carry the altered CFTR gene. Current treatment options for people with CF include drugs, physiotherapy, nutritional advice, exercise and subsequent lung transplantation.

    High levels of cardiovascular fitness increase the lifespan of PWCF. Regardless of disease severity, an association between cardiovascular fitness and survival has been shown in both children and adults with CF. Participation in regular exercise also has the potential to slow the annual rate of decline in lung function, maintain or increase bone mineral density and increase health-related quality of life in PWCF.

    Nicola’s research will be carried out in the School of Health and Human Performance at DCU, in collaboration with the Mater Misericordiae University Hospital, Beaumont Hospital and St. Vincent’s University Hospital, Dublin. Nicola’s research will aim to develop an optimal exercise training program that incorporates both supervised and home-based elements. 

    Describing the research, Nicola said:

    “Although the message of ‘exercise is medicine’ is not new, the challenge to increase the use of exercise as a clinical management tool for PWCF still remains. This research grant will allow me to conduct high-quality, evidence-based work that will inform patient-care pathways with regard to the role of exercise in the management of CF, particularly in patients scheduled for lung transplant. I hope that the outcome of this research can positively impact the quality of life of patients living with CF and improve transplant outcomes. I am very excited to get started!”

    Professor Niall Moyna, supervisor of Nicola’s research and Head of the School of Health and Human Performance and a member of the Centre for Preventive Medicine, both located at DCU, added:

    “This is an exciting research opportunity for Nicola and Cystic Fibrosis Ireland. Exercise has an important role to play in the management of CF, particularly for patients scheduled for lung transplantation. This research will give Nicola the opportunity to enhance the patient care pathway and improve patients’ overall health related quality of life and post-transplant outcomes. I am looking forward to working with Nicola on this research”.

    Professor Karen Redmond, Consultant Thoracic and Lung Transplant Surgeon at the Mater Misericordiae Hospital, Dublin, another supervisor of Nicola’s research, added:

    Developing easily accessible state of the art exercise programmes that incorporates newer technology and practice methods will enhance quality of life and hopefully outcomes”.

    Philip Watt, CEO of Cystic Fibrosis Ireland, co-funder of Nicola’s research, added:

    CFI is proud to be one of the funding partners and to provide the funding for this very important research project. The importance of exercise in CF care is crucial, but to date there has been a limited focus on research on pre and post transplant patients with CF. We are confident that the outcomes of this study will help us understand the challenges but will also provide some of the practical tools which will help our patients”.

    Aisling Jones, mother of Becky Jones, said:

    We are delighted to be supporting this very important initiative and see great possibilities for CF patients as a consequence of the work that Nicola and the team at DCU will undertake.  Ireland has a long way to go in comparison with other countries worldwide, in optimising exercise and physical activity as therapeutic interventions and we see this as the first step towards creating a world class programme that will deliver great benefits to the CF population”.

    Dr. Noel McCaffrey, director of MedEx Wellness at DCU, added:

    “A large proportion of the disability, unwellness and poor quality of life in many chronic illnesses, including CF, is actually caused by physical deconditioning, which itself is due to becoming inactive. This progressive inactivity is very damaging because, in addition to leading to poor levels of physical function, it is also associated with poor psychological wellness and social isolation. There are particular challenges with designing and implementing an exercise intervention for CF patients, especially in a group environment like MedEx, and solving these challenges will be an important part of Nicola’s research. Our hope is that the output of the research in due course will be of practical benefit to CF patients”.

    ENDS


    What is cystic fibrosis (CF)?
    CF is Ireland’s most common life-threatening inherited disease. CF is a genetic disorder.Approximately 1 in 19 people are carriers of the CF gene and when two carriers have a child there is a one in four chance of a child being born with it. CF affects the regulation of absorption and secretion of salt and water in various parts of the body including the lungs, sweat glands, pancreas, and gastrointestinal tracts. This defect inhibits the flow of salt and water through the body’s cells, causing a build-up of thick, sticky mucus, which can clog airways and harbour harmful bacteria. Symptoms reported by people with CF vary significantly in severity from mild to debilitating. The most common symptom is recurrent chest infection, which results in lung damage, with the majority of deaths occurring through respiratory failure. There is a high prevalence of CF in Europe, with the highest prevalence in Ireland, which is almost three times the average rate in other EU countries and the United States.

    About Cystic Fibrosis Ireland
    Cystic Fibrosis Ireland, established by parents in 1963, is a leading national organisation dedicated to improving the quality of life of people with CF and their families across Ireland. It does this through providing information and education, advice and advocacy to people and their families, offering grant assistance, undertaking research, funding state-of-the-art dedicated CF health facilities and CF clinical staff, and advancing the development of lung transplantation in Ireland.

    About MedEx Wellness

    MedEx Wellness is a novel community-based chronic illness rehabilitation programme located at Dublin City University. It offers medically supervised exercise classes and educational workshops to patients with a range of chronic illnesses. Currently, programmes are provided for patients with cardiovascular disease, pulmonary disease, diabetes, and cancer. MedEx also encourages peer support through social activities after class. Since its establishment in 2006, MedEx has grown exponentially to become one of the largest centres of its kind in Europe. The programme, led by Dr Noel McCaffrey, has gained the confidence of local GPs and hospital physicians and has a strong and steady referral base. Currently, MedEx hosts over 600 patient visits per week. It has become a significant resource to the surrounding community and truly transforms the lives of individuals (and their families) living with the burden of chronic illness.

    MedEx offers the following classes:

    ·        HeartSmart - a cardiac rehabilitation programme.

    ·         BreatheSmart - a pulmonary rehabilitation programme.

    ·         SmartSteps - a vascular rehabilitation programme.

    ·         Diabetes Health Steps - a programme for people with diabetes.

    ·         Move On - a 12 week programme for people recovering from cancer treatment.

    ·         Movement To Music - a 12 week dance class for individuals with Parkinson’s.

    ·         Cancer Prepare – exercise programme for patients who are preparing for cancer surgery or treatment.

     For information or queries regarding the MedEx programmes please contact DCU Sport Complex on 01 700 5797.

  • Progress on Potential new CF drug

    The Food and Drug Administration in the United States (FDA) advisory committee, made up of independent medical experts and community representatives, voted 12 to 1 to recommend that the FDA approve the ivacaftor/lumacaftor combination therapy (Orkambi). 

    While the committee’s recommendation is an important part of the FDA’s review process, the decision is not binding. The FDA has said it will make its final decision by July 5, 2015.

    Orkambi targets the underlying cause of the disease in people with two copies of the F508del mutation. Currently, the FDA is considering whether to approve the drug for people with CF ages 12 and older who have two copies of the F508del. 

    New-Drug-for-Cystic Fibrosis

    In clinical trials, Orkambi was shown to improve lung function in people with CF and significantly reduce the rate of pulmonary exacerbations, which can lead to frequent hospitalizations and worsening lung function.

    Orkambi has still to be approved by the FDA (July 2015) and then it will be considered by the European medicine’s Agency (EMA) and then by individual countries have to consider reimbursement of the drug, should it be approved by the EMA. CFI will continue to keep our members updated on the progress of this and other potential therapies developed for use in CF care.

  • Publication of the Orkambi Clinical Trial Data 

    On 17th of May 2015, the The New England Journal of Medicine (NEJM) published results on a new treatment combining ivacaftor (Kalydeco) with another compound called lumacaftor. This combination is now known as Orkambi and it targets the underlying cause of the CF in people with two copies of the F508del mutation, which represents 56.5% (654) people with CF in Ireland1.

    Orkambi clinical trial data

    Published results show a significant increase in FEV1 in patients of between 2.6 - 4% (p<0.001). Further analysis also revealed that the rate of pulmonary exacerbations was 30 to 39% lower in patients who were in receipt of the combination therapy compared to those who received placebo. The rate of events leading to hospitalisation or the use of intravenous antibiotics was also reduced for people being treated with the combination therapy.

    The publication of the study comes on the heels of a recommendation last week from an advisory committee of the U.S. Food and Drug Administration (FDA) that the FDA approve the drug for people with CF ages 12 and older with two copies of F508del. While the committee’s recommendation is an important part of the FDA’s review process, the decision is not binding. The FDA has said it will make its final decision by July 5, 2015.

    The drug is currently being considered for licencing by the European Medicines Agency, with a decision expected in the autumn. If there is a positive decision from the EMA then individual countries have to consider reimbursement of the drug, should it be approved by the EMA.

    CFI will continue to keep our members updated on the progress of this and other potential therapies developed for use in CF care.

    You can listen to This email address is being protected from spambots. You need JavaScript enabled to view it., Research and Development Officer from CFI, speak about these results on RTE Radio 1 news.

    1Cystic Fibrosis Registry of Ireland (2013) Annual Report

  •  

    Check out past issues of the Research e-Zine by clicking on the issue number below.

    Issue 2: August 2018

    Issue 1: May 2018

  • Survey on Mental Health, Well-being and Quality of Life in Adults with CF


    If you have Cystic Fibrosis, and are over 18 years of age, we would like to invite you to take part in a study about mental health, well-being and quality of life in adults with Cystic Fibrosis.

    writing letter

    This study is being conducted by researchers from University College Cork, and is funded by Cystic Fibrosis Ireland and the Health Research Board. We know very little about the well-being of individuals with CF in Ireland, but by doing this research we hope to bring attention to this issue and hope it may contribute to improvements to services in the future.

    We would encourage you to take part in this valuable study. All information you provide will remain strictly confidential, however, there is one exception to this. If you indicate on your questionnaire you are experiencing symptoms of depression or anxiety, we are obliged to inform your CF consultant so that you can be followed up with the appropriate support.

    If you would like more information on the study, please read this information sheet or contact Jennifer Cronly This email address is being protected from spambots. You need JavaScript enabled to view it..

    The questionnaire takes approximately 15-20 minutes to complete. We would be grateful if you could complete the questionnaire by Tuesday 12th May.

    Take the Survey Now!

  • Vertex Announces Data from 12-Week Phase 2 Safety Study of VX-661 in Combination with Ivacaftor in People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation


    The primary objective of this Phase IIb trial was to assess and ensure patient safety when taking the combination of VX-661 and Ivacaftor. The study evaluated two doses of VX-661 (100 mg once daily or 50 mg every 12 hours) in combination with Ivacaftor (Kalydeco) (150 mg every 12 hours). The study showed that the combination regimen was generally well tolerated, and all patients completed 12 weeks of treatment. The most common adverse events were pulmonary exacerbation, and cough.

    test-tube

    Secondary endpoints evaluated the effect of the combination on lung function. The patient group who received the 100 mg dose of VX-661 plus Kalydeco, patients reported a 4.4% improvement in lung function at 4 weeks, and a 3% improvement at 12 weeks. Consistent with prior Phase 2 studies that evaluated 4 weeks of treatment with VX-661 in combination with Ivacaftor, this study showed a rapid improvement in lung function within four weeks of treatment, and after patients completed treatment, lung function returned to baseline.

    These safety and efficacy data, together with other data from multiple previously completed Phase 2 studies of VX-661, support Vertex's ongoing Phase 3 program of VX-661 in combination with Ivacaftor. The Phase 3 program is evaluating VX-661 (100 mg once daily) in combination with Ivacaftor (150 mg every 12 hours) and consists of four Phase 3 studies, including a study in people with two copies of the F508del mutation that began enrolment in February. The other three studies will enroll people with CF who have one copy of the F508del mutation and a second mutation that is either a gating mutation, residual function mutation or a mutation that results in minimal CFTR function.

    To read the full release, please click here.