There is a very high need for improving the management of pain. Acute and persistent pain of different origins represent a common medical, social, and economic burden, and its pharmacotherapy is often inadequate. To advance management of pain patients and support decision making in clinical practice, more predictive assessments of treatment success are needed. The development of analgesics is onerous because promising preclinical data often do not translate into the clinic. Improved pharmacodynamic biomarkers could define whether nociceptive signalling is adequately modulated by a new drug, so increasing the chance of successful translation and greatly reducing the risk in initiating clinical development. Further, the pathophysiology of chronic pelvic pain indications is poorly understood and no adequate preclinical models are available, precluding focused preclinical research and leaving affected patients with little hope of relief. IMI-PainCare aims at making advances in these three pain areas in a complementary manner. Three subprojects will address specific scientific challenges. Subproject PROMPT will identify Patient Reported Outcome Measures as tools to standardise assessments of treatment success of acute and chronic pain in Real World conditions and controlled trials, and so improve its management; subproject BioPain will validate the translatability of pharmacodynamic biomarkers and PK-PD modelling in pain pathways of healthy subjects and preclinical species, thereby offering tools to improve drug development; subprojectTRiPP will identify biomarkers and novel therapeutic pathways of clinical phenotypes of patients with chronic pelvic pain, which after back-translation, can improve how preclinical models reflecting human diseases. The goal of IMI-PainCare is to improve the care of patients with acute or chronic pain by providing a toolbox to streamline the development process for novel analgesic drugs and to improve treatment quality in clinical practice.

Alzheimer’s disease (AD) and Parkinson’s disease (PD) are common neurodegenerative conditions, posing a major societal burden. There is a lack of treatments to slow disease progression, and therapeutic development has been impeded by a lack of biomarkers that can detect individuals early in the disease, measure treatment effects, and stratify patients. European cohorts recruited for research on aging and neurodegeneration provide a huge potential for biomarker discovery and validation by providing bio-samples along with deep clinical and imaging phenotypes. However, these cohorts are difficult to access. An overview of the availability of data and samples is lacking, and protocols and regulations for data and sample collection, storage, and sharing vary. The European Platform for Neurodegenerative Diseases (EPND) will tackle the above issues by developing a self-sustaining European-based platform to facilitate discovery and access of relevant bio-samples and data. EPND will be built on an existing informatics infrastructure, the AD Workbench, which EPND will adapt to support resource- and participant-level discovery, data harmonisation, central and federated data and sample storage, and data analysis. The sample and data discovery tools will be connected to a network of over 60 cohorts on AD, PD, and related disorders. Together, these cohorts will facilitate access to data and samples of over 120,000 research participants including CSF (n=30,000), plasma (n=120,000), stools (n=6,000), urine (n=27,000), saliva (n=17,000) and digital biomarkers (n=2,000). Prospective data collection will also occur during the project. This approach provides the community with a new and powerful environment for collaborative cross study analysis of harmonised biomarkers, datasets and samples. EPND will provide visibility into the quality and standardization of the data and samples available in the platform from the cohorts available and will also provide protocols for ongoing data and sample collection. This will guarantee quality of samples available, an important factor for validation and regulatory approval for biomarkers. EPND will be guided by ethical, legal and regulatory experts, patients, and other stakeholders to ensure responsible practices and processes underpin all discovery, sharing and access of data and samples, whilst simultaneously ensuring the platform is self-sustainable by the end of the project. Thereby, EPND will provide the community with a long-term, powerful environment to aid biomarker research for neurodegenerative disorders, enabling critical advances in the development of treatments for AD and PD.

THERACAT is an international and multidisciplinary consortium aiming at the training of 13 ESRs on the innovative topic of novel bio-orthogonal catalysis-based tools for cancer therapy. This ETN comprises 6 academic partners, 3 industrial partners active in the pharmaceutical market (1 large pharmaceutical company, Teva, and 2 SMEs, BiogelX and Tagworks) and 3 partners with focus on science communication (Cancer research UK), gender and minorities (UAB-Observatory for Equality) and management and entrepreneurship (ESADE business school). The combination of academic, private and society-involved organisations will provide a broad training for the 13 ESRs recruited, equipping them with the necessary skills to succeed as scientists, industrial researchers and entrepreneurs. The development of novel cancer therapies is a major challenge for academic research and pharmaceutical industries. THERACAT aims to establish a training programme focused on the development of THERApeutic CATalysts. In this strategy, materials bearing a catalytic unit are delivered to the tumour and subsequently non-active prodrugs are administered. The prodrugs are non-toxic and therefore generate limited side effects. Only at the tumour site the catalytic particles convert the prodrugs into active compounds that generate a therapeutic effect. This approach presents several advantages on the classical drug delivery paradigm including limited side effects and prolonged efficacy. This multidisciplinary research programme will be the setting for the training of 13 ESRs. The combination of the research expertise, the cutting-edge facilities and the complementary skills present in the consortium holds a great promise for the advancement of their careers, as well as of the knowledge of catalysis-based anticancer therapies and the development of marketable technologies and products.

Autism Spectrum Disorder (ASD) is a heterogeneous neurodevelopmental condition affecting over five million people in the European Union. The combination of core symptoms (deficits in social-communication and repetitive and restricted behaviours and interests) and common comorbidities (e.g. epilepsy and depression) significantly reduces the quality of life and life-span of affected individuals. Currently there are no effective drug treatments for the core symptoms. Key factors that have hampered progress include; 1) limited understanding of the underlying pathophysiolog(ies); 2) lack of successful translation from animal models to humans; 3) testing of drugs with specific actions in biologically heterogeneous populations; 4) limited expertise of many European ASD centres in running large-scale clinical trials; and 5) trial designs (e.g. placebo effects). Our vision, therefore, is to apply a precision medicine approach to ASD and improve patient outcomes by tailoring treatments to a patient’s biological profile. Our efforts will build on the achievements of 5 other IMI initiatives, 4 Horizon 2020 networks, and 6 SMEs for the first time to; 1) align global resources to validate and qualify stratification biomarkers from infancy to adulthood; 2) develop objective outcome measures that can be used in trials; 3) create a European-wide clinical trials network that reliably carries out studies able to support filings to the EMA/FDA; 4) carry out better targeted clinical trials linked to other international efforts – including quick wins or “fast fails” of ineffective agents; 5) translate molecular mechanisms and drug effects between preclinical models and particular subtypes of ASD. Together we will bring Europe to the forefront of clinical research in ASD. Also we will provide a sustainable legacy that is accessible by others across the world, attracts industry into ASD, and helps transform healthcare.

RealHOPE will create an understanding of the real-life handling of protein drugs in hospital pharmacy, clinics and in the hands of patients by applying smart tag technologies. Different parameters will be logged and combined with protein characterisation at different stages, as well as with information from EFPIA partners on their drugs in use. Statistical evaluation of the data will be used to identify patterns in handling that are linked to protein destabilisation occurrence and type of protein degradation. Focus interviews with personnel in hospital pharmacies, clinics and with patients/care givers will be used to understand current handling practice and what the desired handling instructions and limitations are. These insights will be used to design in-use mimicking stability protocols for the protein drugs in the project. Ultra-scaled-down devices for stability assessment will be designed to be used in early phase for efficient development cost effective and safe future protein therapies. Protocols and devices will be validated towards the collected handling data base. Interventions in hospital pharmacies using e.g. compounding robots will be investigated. Techniques to assess e.g. aggregate formation in the final drug preparation situation will be evaluated together with SMEs producing analytical tools and hospital pharmacists. The collected data and interviews will form the base of development of teaching materials directed towards different target groups: hospital pharmacists, nurses and patients/care givers. App developers will be active in this part to design attractive and efficient apps for teaching and collecting therapy performance data.