Research Coordination Objectives
The specific, measurable, achievable, relevant, and timely objectives of Neutro-NARPS are as follows:
a) The identification of genetic causes of unclassified congenital CNP and elucidation of the role of inflammation in their transformation: The Action will coordinate the (a) identification of undiagnosed congenital CNP patients, (b) establishment of a diagnostic pipeline integrating NGS patients’ data to identify variants of interest, AND c) functional validation models to study their implication in granulopoiesis and/or MDS/A These rare CNP entities could not be collectively analysed without an international collaboration.
b) The elucidation of the pathogenetic basis of novel CNP entities: The Action will coordinate the (a) identification of patients with likely-acquired, idiopathic and CNP related to LEA and (b) GWAS, WES/WGS data using advanced bioinformatics analysis to identify germline mutations related to immune dysregulation and/or novel molecular mechanisms affecting neutrophil productio Without an international collaboration these patients remain undiagnosed.
c) The clarification of mechanisms of CNP associated with novel therapies: The Action will coordinate the (a) identification of patients with CNP following treatment with signal transduction inhibitors, immunomodulatory agents, novel antibodies or CAR-T therapies, (b) collection of patients’ PB and/or BM samples for the investigation of the effects of the immune system in the production and survival characteristics of neutrophils following these treatme
d) The development of ML models for the precise identification, classification and prognostication of CNP that will facilitate clinical practice: The Action will coordinate the (a) collection of clinical, laboratory and -omics data from different CNP patient Registries, (b) use of data for the design, development and implementation of ML tools aiming to: i) predict the likelihood of development of MDS/AL or other disease entities such as malignancy, autoimmunity, CVD; ii) unravel patterns indicating new disease subcategories; iii) identify biomarkers that can enhance diagnostic accuracy, provide insights into disease mechanisms, and guide the development of targeted therapies.
e) The organization of the regulatory framework for the introduction of clinical trials for the treatment and potential cure of patients with severe CNP using novel, orphan or repurposed drugs and gene ther The Action will build on the experience of existing partners on the development and pre-clinical application of novel therapies for severe CNP and boost the participation of expert clinicians, representatives of clinical trials centres, patient Registries, patient Organizations, and pharmaceutical companies to set-up the implementation of novel therapies from bench to bedside, as well as their communication to the scientific and patient communities.
f) The development and implementation in the clinical practice of electronic tools for PROs to improve CNP patients’ QoL, based on the Action’s multidisciplinary experts including bioengineers, digital health engineers, clinicians from different centres/countries, patient/family representatives and SMEs. Such tools will also give the potential for go-to-market exploitation for a wide use in patients with other BM failure syndromes.
COST Members
COST Intl. Partner (UoW,USA)
Participating Organization (EHA)
1
+Months
Deliverables
Capacity-building Objectives
The Action builds upon a network of multidisciplinary approaches and interdisciplinary co-operations, relevant to the COST missions that have advanced the diagnosis and homogenized the management of CNP patients across and beyond Europe. The Neutro-NARPS network is enriched with interactions between: clinicians-experts in CNP; researchers with expertise in biomedicine, bioinformatics and computer sciences; physician-scientists able to implement state-of-the-art technologies and novel therapies in the clinic; patient registries offering availability and access to biological material and clinical data; patient organizations contributing to the better recognition of patient needs and their willingness to accept novel therapies; pharmaceutical companies and representatives from the European Clinical Research Infrastructure Network (ECRIN) for clinical trials development; small and medium-sized enterprises (SMEs) aiming to promote research, technology and entrepreneurial innovation under the scopes and missions of the European Research Area (ERA). Prestigious scientific communities such as the EHA, the EHA’s relevant Specialized Working Groups (EHA-SWG) (e.g. Granulocytes & constitutional marrow failure syndromes, G&CMFS; QoL), the Severe Chronic Neutropenia International Registry (SCNIR) [40], as well as representatives from National Haematology Associations and Competent Authorities, will contribute to the realization and dissemination of the Action’s aims by providing expertise in scientific, ethics and regulatory issues, opportunities for interactions with patient Organizations and partnership with clinical trials centres.
The Action involves a critical mass of Young Researchers and Innovators (YRI) and commits to further increase their numbers particularly from COST Inclusiveness Target Countries (ITC), keeping the balance between genders, to become the next generation CNP experts. The Action will also give the opportunity to outstanding researchers or “pockets of excellence” who are not yet well integrated in European Research Networks to collaborate, enhancing the capacity building at the European level. Overall, Neutro-NARPS gives unique opportunities for cooperation between researchers, physicians, patient Organizations and industrial stakeholders to pave the way for cutting-edge research and transfer of knowledge from bench-to-bedside for the treatment and support of CNP patients. The capacity building objectives of the Action are to:
a) strengthen, by using the COST tools, the interactions among experts in the field of CNP at the European level and beyond exchanging knowledge on orphan or novel CNP entities and on the application of novel therapies in the clinic that will improve the survival and QoL of these patients;
b) bridge, for the first time in the field of CNP, clinicians and biomedicine researchers with bioinformaticians, computational biology scientists and bioengineers for the development of innovative ML models and PRO tools towards the benefit of CNP patients and families, physicians treating these patients and the European healthcare system as whole;
c) facilitate collaborations between stakeholders for the entrepreneurial exploitation of innovative diagnostic/prognostic pipelines, ML models and PRO tools, for CNP and potentially other BM failure syndromes, increasing individuals’, Institutional, European research competitiveness.
d) act as a stakeholder platform with expertise in all aspects of CNP entities, including orphan and novel types, from basic research to patients’ treatment and support, to cover the needs of the scientific and patient community and disseminate the novel knowledge within these communities.
e) create the new generation of experts in CNP research, from the bench-to-bedside, by fostering the active involvement of YRIs, particularly from ITC and regions with less research capabilities, keeping the gender balance and promoting the inclusion of newly established research groups.
Action Of European Cooperation in Science and Technology
This website is based upon work from COST Action Neutro-NARPS, CA24124, supported by COST (European Cooperation in Science and Technology).
COST (European Cooperation in Science and Technology) is a funding agency for research and innovation networks. Our Actions help connect research initiatives across Europe and enable scientists to grow their ideas by sharing them with their peers. This boosts their research, career and innovation.
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