Towards more personalised treatments for people with haemophilia
iPATH, the Irish Personalised Approach to the Treatment of Haemophilia, brings together academic and industry researchers and patient partners to explore the biology of haemophilia and how it impacts upon affects individuals' lives. The findings will help to fine-tune treatments and management plans for people living with severe haemophilia, and improve their quality of life.
The Consortium is part-funded by SFI under the SFI Strategic Partnership Programme, and is co-ordinated by the National Coagulation Centre at St James’s Hospital, Dublin.
The iPATH consortium also includes Children’s Health Ireland Crumlin, Takeda, The Irish Haemophilia Society, the Royal College of Surgeons in Ireland and Trinity College Dublin. The combined funding commitment from industry, charity and SFI is €4 million over the four years of the programme, which launched in 2017.
How do industry partners benefit from the partnership?
Industry partners benefit from the time, focus and expertise that academic scientists bring to the discovery process, which generates the new diagnoses and treatments that industry can further develop and validate for the benefit of patients.
Dr Peter Turecek, Senior Director, Global Medical Affairs, Takeda, says: “Takeda is committed to driving innovation and advancing standards of care in haemophilia. Through the iPATH study, we hope to uncover new solutions that build on and maximise the role of factor therapy and further personalise care for haemophilia patients.”
How do academic partners benefit from the partnership?
By working with industry, academic partners can design and develop their research to maximise the possibility of the findings being able to help patients.
iPATH Director, Professor James O' Donnell, says: “In Ireland we excel at providing treatment for people living with severe haemophilia, and we have rich data about the clinical phenotype, or how haemophilia manifests in patients. As academic researchers involved in iPATH, the project will enable us to fundamentally improve how we understand haemophilia and its treatment and put Ireland on the map for improving global approaches to the management of severe disease in patients.”
How do patient partners play an active role in this partnership?
iPATH has a strong patient focus, to ensure the research is meeting patient needs and that patients can play an active role in the development of new therapies.
Brian O'Mahony, CEO Irish Haemophilia Society, says: “A culture of research fosters a culture of clinical excellence and formal patient involvement. The iPATH research programme benefits people with haemophilia by researching fundamental biological issues which impact the community. A real example are the findings on BMI and physical activity level, which have resulted in the Society focus on healthier lifestyle and led directly to our new physiotherapy/exercise programme. We view the iPATH programme not as abstract research but as a gateway to improved understanding, to improved and targeted clinical care and to a better future for people with haemophilia in Ireland.”
Currently, treatment for haemophilia is for patients to receive frequent injections of the blood-clotting factors that they are missing. This means their blood can clot appropriately. The dose of clotting factors each patient receives is generally based on their weight, but it is a crude measure. For example, some people with severe haemophilia may clear the injected clotting factors out of their bodies quickly, so they need a higher dose than their weight suggests. iPATH is looking at the biological and behavioural factors (such as exercise) that can be used to tailor treatment for a patient, and manage their haemophilia more effectively.
iPATH will develop of personalised approaches for patients with severe haemophilia. The multi-disciplinary team of academics, doctors, nurses, physiotherapists and dentists are already presenting the work at international conferences and winning awards, and iPATH will strengthen the spotlight on Ireland as a world leader in research on bleeding disorders.
iPATH projects span from fundamental research on the biology of what happens in severe haemophilia and how factors within the body could affect a person’s experience of it, right through to how to manage the disease better in the clinic. iPATH projects include:
- Full genome analysis of patients with severe haemophilia and linking genomic features with clinical manifestations
- Looking at biological processes that affect how rapidly the body clears injected clotting factors, to better understand why and how some patients can maintain the injected blood-clotting factors for as long as 30 hours, while other patients use them up in just 6 hours.
- Tracking how body mass index (BMI) and physical activity affect a patient’s wellbeing and clinical progress.