The main aim of NEURONET is to set up an efficient platform to boost synergy and collaboration across the IMI projects of the Neurodegenerative Disorders (ND) portfolio, assisting in identifying gaps, multiplying its impact, enhancing its visibility and facilitating dovetailing with related initiatives in Europe and worldwide. This will be achieved through the following specific objectives: 1) creation of an overall platform for efficient collaboration, communication and operational synergies among present and future IMI ND projects; 2) designing systems to map and analyse information regarding actions, initiatives and partnerships, assessing impact of the individual projects, remaining gaps and global value of the programme for stakeholders; 3) supporting the management of the programme (timelines, dependencies, synergies and key results across projects); 4) proactively detecting needs, opportunities and transferable best practices of projects and to connect them; 5) providing support to the projects by organising tools, services, expert advice and guidelines/recommendations on common issues; 6) promoting enhancement and coordination of communication across the IMI neurodegeneration projects, increasing programme visibility, outreaching to key stakeholders and establishing relationships with initiatives in the field; and 7) preparing and securing the long-term sustainability of NEURONET itself. Achievement of these objectives will necessarily rely on buy-in from existing and future projects in the IMI ND portfolio, and effective connections with other programmes and initiatives in Europe and beyond, including stakeholder representation. For this, the Consortium has been constituted by very active partners in a variety of IMI ND projects and related initiatives with specialists in areas like complex/project management, data sharing & re-use, drug development, patient engagement, communication, sustainability and regulatory/HTA interactions.
The GetReal Initiative brings together partners from the IMI GetReal project to drive the adoption of tools, methodologies and best practices from IMI GetReal and increase the quality of real-world evidence (RWE) generation in medicines development and regulatory/HTA processes across Europe. We will establish, in Work Package (WP) 1, a Think Tank, a number of Task Forces and a RWE Research Community. The Think Tank will consist of international thought leaders and will discuss, assesses and give recommendations on the opportunities and barriers to the generation, use and acceptability of RWE. They will act as ambassadors for the use of RWE during the project and beyond, enagaging with key stakeholder groups to drive policy debate and facilitate the uptake of the outputs of IMI GetReal and the GetReal Initiative. The GetReal Taskforces will drive the focused development of tangible solutions to the key challenges identified in IMI GetReal and the Think Tank. The initial three task forces will be: (i) Pragmatic Trials (design, operational feasibility and analysis/the GetReal PragMagic tool), (ii) network meta-analysis and benefit risk assessment (incl. the GetReal ADDIS tool) and (iii) Statistical Approaches for enriching RCTs with real-world data. The GetReal Research Community will consist of researchers and organisations active in the field of RWE generation, regulators, HTAs, physicians and patients. The Community will review and comment on any GetReal Initiative guidelines, recommendations or white papers ahead of their finalisation, will have access to all the GetReal tools and outputs, receive regular newsletters and receive invitations to attend stakeholder events. The project will also invest in long term sustainability of the GetReal Initiative and the tools on a not-for-profit basis (WP2). The project is supported by professional experienced consortium management (WP3) and an ethics work package (WP4).
There is an urgent need for novel approaches assessing functional decline in early AD. The main goal of the RADAR-AD project is to develop a digital platform to detect subtle functional deficits in early Alzheimer’s disease (AD) individuals by integrating a meaningful combination of smartphone, wearable and/or home sensor based parameters. The system developed will be suitable for future longitudinal studies, including trials. The objectives are to 1) Identify the most relevant functional domains and the most promising remote measurement tools (RMTs) for these domains based on reviewing the literature and piloting of RMTs in small studies; 2) Optimise the RADAR-CNS platform for use in AD studies; 3) Test the platform and selected RMTs in a real world environment clinical study with 240 participants across the AD spectrum ranging from the preclinical AD to the dementia stage; 4) Perform statistical modelling to estimate longitudinal predictions; 5) Discuss results with regulatory agencies in order to obtain guidance about how to develop a path for formal qualification as outcome measurements in future therapeutic interventions. Considering the limited budget and project duration, we will rely upon already available technology platforms and on available longitudinal datasets where possible. The consortium includes experts in clinical dementia studies, computer science, bioinformatics, regulatory policies, ethics, and patient and public involvement (PPI). Additional strengths of the consortium are the deep and broad interface with RADAR-CNS and related IMI projects, and the access to large amount of patient-level data from key European cohort studies for modelling purposes.
EBiSC2 builds on the achievements of the European Bank for iPSCs (EBiSC1) in centralising existing capacities across Europe in a unique banking and distribution infrastructure for research use in response to the increasing demand for human induced pluripotent stem cells (iPSC). Significant progress towards this aim has been made by EBiSC1; further resources, however, are required to ensure self-sustainability. Key partners of EBiSC1 who have delivered major assets of the current bank, join efforts to establish EBiSC2 as self-sustainable, central bank. Based on a gap analysis of the EBiSC1 endeavours towards sustainability, and focussing on user demand, scientific excellence and productivity, EBiSC2 will deploy a business strategy for a sustainable, non-for-profit bank providing access to disease-relevant and quality-controlled iPSCs, along with comprehensive data and freedom to operate for academic and commercial use. To meet evolving requirements from industry and academia, the cell catalogue will be constantly enriched through on-demand generation of new iPSC lines, including gene-edited lines and isogenic controls, iPSC-derived and progenitor cells. EBiSC2 will distribute cell lines and develop a range of additional cell services (incl. screening panels of disease-relevant iPSC and control lines in ready-to-use-formats) to extend its offer, while reducing operational costs through state-of-the-art upscaling and automation enabling bulk production of standardised high-quality cells. Proof-of-concept studies performed jointly by academia and industry will demonstrate the reliability and robustness of the lines for disease modelling and screening and enrich the EBiSC2 catalogue with extensive data. To bundle resources, EBiSC2 focuses on collaboration with iPSC programmes and aims to serve as central hub for EU-funded projects to bank their iPSC lines, and thus, enable long-term access by the research community to the results of European investments.
Immunotherapies have emerged as effective treatments for immune system-related diseases, such as cancer and inflammatory diseases. Although immunotherapeutics are effective not all patients benefit and side effects vary greatly. Thus, there is an unmet need for a personalized approach. Blood and tissue biomarkers have played only a modest role up until now in assessing immune status in vivo and we believe that non-invasive whole body imaging will make a significant contribution in the future. Although the field of imaging (PET, MRI, Optical Imaging) is flourishing, its application to patient immune status measurement and monitoring has barely been explored. Within Immune-Image we aim to develop new immunotracers for imaging specific immune cell subsets, develop quantitative imaging workflows, validate the immunotracers pre-clinically in relevant disease models, produce the tracers in a Good Manufacturing Practice (GMP)-compliant way, and conduct smart clinical trials, including with novel tracers. To this end, insights will be gained into the optimal use of drugs and a more efficient development of immunotherapeutics. Moreover, the success rate of immunotherapy development will be enhanced since our molecular imaging platform will provide novel insights into patient immune status, which will support drug development and treatment decisions. To achieve our goals, we have assembled an experienced international multi-disciplinary consortium composed of chemists, biologists, immunologists, physicists, pharmacists, information technologists, and medical specialists with diverse and essential backgrounds who will join forces to design, synthesise, evaluate and validate immunotracers and create sustainable molecular imaging techniques that can be broadly applied in the assessment of the immune status and immune modulation of patients. There will be input from patients and regulators in order to assure optimal design and execution of immunotracer clinical trials.