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COST Innovator Grant (CIG) Information

COST Innovator Grant (IG16125): Open ILD: An Open Access Repository of Pluripotent Stem Cells from Children and Adults with Interstitial Lung Disease

Interstitial lung diseases (ILD) are a large group of lung diseases which are charactierised by scarring 9fibrosis) or inflammation of the lung. It can occur in children (chILD) or in adults. Children’s interstitial and diffuse lung disease (chILD) is a group of rare lung diseases ranging from infancy to adolescence. It is a heterogeneous group of non-neoplastic disorders resulting from damage by varying patterns of inflammation and fibrosis with the interstitium as the primary site of injury. Although there are a large number of different diseases (over 200) the presenting symptoms and signs are very similar with the main feature being a derangement of gas exchange and restrictive lung physiology.

Adult interstitial lung disease is often different from chILD although some of these diseases can present from childhood and persist into adulthood. The rarity of individual ILDs in particular chILDs contributes to a lack of randomized control trial data on effectiveness of treatments. Management strategies derive from other diseases or are based on physicians experience and remain controversial.

Open-ILD is a network of multidisciplinary clinicians (paediatric and adult), scientists, and patients and their families funded by COST Innovator Grant (IG16125). The network is coordinating pan-European research from basic science to clinical care with the ultimate goals to will foster a growing body of research across Europe to develop a robust preclinical model of ILD that can a) explore pathogenesis b) identify new patient-specific druggable targets and c) test novel compounds and inhaled modes of delivery for patients in vitro before clinical studies, leading to shortening of drug development pipelines and reduction of toxic side effects and improved quality of life for children and adults with ILD.

The COST Action's Memorandum of Understanding is available for download here.

Objectives

We are multidisciplinary clinicians, scientists and patients and their families that have united in this network. The network promotes research from basic science to clinical care, with the ultimate goal to develop new treatments that lead to improvements in the long term outcome of patients with ILD. The CIG aims to develop an operational, legal, ethical and regulatory framework which will facilitate the generation of a highly phenotyped sharable repository of induced pluripotent stem cells (iPSC) from children and adults with ILD. These cells will be generated from patients samples collected across Europe and in turn will be used to generate patient-specific alveolar epithelial cells (AECs) and macrophage enabling the development of a paradigm-shifting personalised model of disease pathogenesis. Open-ILD is strengthening the infrastructure for research on ILD, building on and unifying the expert networks of research in ILD and providing targeted training for Early Stage Researchers through Training Schools and on Short Term Scientific Missions, in the clinical and basic science aspects of ILD.

Description

Interstitial lung disease (ILD) in adults and children (chILD) is a group of devastating fatal lung diseases leading to scarring of the lung and death often within 3 years of diagnosis. While new antifibrotic drugs offer hope of slowing disease progression in adults, no effective treatments are available for children and lung transplant is the only effective cure. Genetic factors may cause or contribute significantly to the risk of developing ILDs and those patients with inherited forms of ILD may have a worse prognosis than sporadic-ILD, respond poorly to current treatments and some can have serious adverse reactions to immunosuppression after transplantation. Across Europe diverse mutations including those in genes related to telomere homeostasis, and more rarely in surfactant homeostasis can lead to abnormal cell aging and surfactant protein processing in lung alveolar epithelial cells respectively. The rarity of individual mutations contributes to a lack of basic mechanistic studies and randomised control trial data on effectiveness of treatments. Consequently, management strategies derive from other diseases are based on physicians experience and remain controversial. Furthermore, the current-state-of-the-art preclinical models fail to accurately recapitulate the diverse genetic causes of ILD. Therefore, there is a lack of effective preclinical tools that can be shared with researchers across Europe to study these diseases in order to identify and personalise treatments for patients. Members of the CIG have already employed patient-derived geneedited induced pluripotent stem cells to generate alveolar epithelial cells and macrophage and successfully model ILD and other chronic lung diseases in vitro. The CIG has already established nascent collaborative relationships to use these cells to study this group of diseases and to test novel treatments. The European Network for Translational Research in Children’s and Adult ILD (ENTeR chILD) a network of multidisciplinary clinicians (paediatric and adult), scientists, and patients and their families has already initiated pan-European research with the ultimate goals to deliver accurate and early diagnosis with structured, personalised, management and therapies. In partnership with patient organisations, we have co-designed a groundbreaking proposal which aims to leverage this existing transdisciplinary network to establish a first-of-a-kind Europe-Wide open-access resource of patient-derived iPSC linked with highly phenotyped patient data. We believe the establishment of this resource will foster a growing body of research across Europe to develop a robust preclinical model of ILD that can a) explore pathogenesis b) identify new patient-specific druggable targets and c) test novel compounds and inhaled modes of delivery for patients in vitro before clinical studies, leading to shortening of drug development pipelines and reduction of toxic side effects.

Scientific programme

The scientific programme is delivered by three highly integrated Working Groups (WG), supported by a programme of conferences, workshops, Training Schools and Short Term Scientific Meetings (STSM). Collaborations between WGs provide a truly innovative environment for this scientific programme. ENTeR-chILD remains flexible to enable the implementation of new ideas and perspectives not foreseen during the planning stage, and to enable inclusion of scientists who did not participate in the early stages.

How to join the COST

https://www.cost.eu/cost-actions/participate-in-an-ongoing-action/