Research & Innovation - Participant Portal

TOPIC : Discovery and characterisation of blood-brain barrier targets and transport mechanisms for brain delivery of therapeutics to treat neurodegenerative & metabolic diseases

Topic identifier: IMI2-2017-12-06
Publication date: 19 July 2017

Types of action: IMI2-RIA Research and Innovation action
DeadlineModel:
Opening date:
two-stage
19 July 2017
Deadline:
2nd stage Deadline:
24 October 2017 17:00:00
16 May 2018 17:00:00

Time Zone : (Brussels time)
  Horizon 2020
Topic Description
Specific Challenge:

Several challenges have yet to be addressed to better understand the role and alterations of the Blood Brain Barrier (BBB) and transport mechanisms in health and diseases. This is particularly critical for neurodegenerative diseases (e.g. Alzheimer and Parkinson’s diseases, Amyotrophic Lateral Sclerosis (ALS)), vascular dementia, multiple sclerosis and metabolism-related central diseases (diabetes and obesity). It will be also important to understand the mechanisms of neurotropic virus-mediated BBB and brain penetration, and to be able to apply this knowledge for the development of innovative drug delivery systems, especially for biopharmaceuticals, and the identification of novel drug targets. Furthermore the various transport mechanisms and potential drug delivery systems that have been suggested so far need validation by independent researchers and the further understanding of challenges to advancing into clinical drug development by biotech/pharma.

Scope:

The objectives of the project to be delivered from this topic are:

  1. establishment and characterisation of BBB models relevant for healthy and disease conditions for evaluation of disease-modifying agents (human in vitro cell based, in particular iPSC or progenitor-derived cells, and in vivo);
  2. identification of translational readouts closer to the pathogenesis of neurodegeneration and mimicking altered BBB under disease conditions;
  3. in-depth understanding of the biology of the BBB and characterisation of various transport mechanisms across the BBB (including virus-mediated BBB and Central Nervous System(CNS) penetration);
  4. discovery and development of innovative and efficacious brain delivery systems.

These objectives could be attained through the milestones shown hereunder. Each of them could represent an independent work package and will be described later in the topic text:

  1. select specific genes and pathways expressed in endothelial cells of normal and/or diseased human brains or preclinical models;
  2. validate in vitro and in vivo that these genes or pathways are responsible for normal/deficient/altered transport at the BBB and the impacts of disease development and progression on these genes or pathways;
  3. this will enable the generation of improved BBB models for neurodegenerative/metabolic diseases predictive for the disease situation with optimized in vitro-in vivo correlation compared to established models; develop in silico models for predicting BBB penetration and pharmacokinetics of therapeutics in CNS;
  4. identify and validate novel targets for brain delivery;
  5. understand the mechanisms of neurotropic virus-mediated BBB and CNS penetration to inform innovative ways of brain-selective delivery.
Expected Impact:

The use of ‘healthy’ and patient-derived specimens, inducible pluripotent stem cell (iPSC) clones and other types of progenitors offers compelling approaches due to the direct connection to patients with the underlying disease. The impacts of these new models could include: (1) yielding novel insights into currently identified BBB transport mechanisms for drugs, especially biopharmaceuticals, (2) allowing to use comparative assessment between ‘healthy’ and ‘diseased’ BBB, including in silico models, to prioritise some approaches for specific disease(s) because the transport mechanism is modified in the disease state, (3) leading to the identification and characterisation of novel transport mechanisms that are unaffected or upregulated in the disease or neurotropic virus-mediated, making them even more interesting, and (4) facilitating the discovery and characterisation of novel targets addressing the vascular aspect of neurological disorders like AD and thus open up novel routes for therapy.

Topic conditions and documents

Please read carefully all provisions below before the preparation of your application.

The IMI2 12th Call for proposals topic text as well as the Call Conditions are available here

1. List of countries and applicable rules for funding: described in article 10(2) of Regulation N° 1290/2013 of 11 December 2013 laying down the rules for participation and dissemination in Horizon 2020 and in article 1 of the Commission Delegated Regulation (EU) N° 622/2014 of 14 February 2014.

2. Eligibility and admissibility conditions: described in the IMI2 Manual for evaluation, submission and grant award See also the Commission Delegated Regulation (EU) N° 622/2014 of 14 February 2014.

Proposal page limits and layout: Please refer to Part B of the standard proposal template.

3. Evaluation

Submission and evaluation process, including evaluation criteria and procedure, scoring and threshold are described in the IMI2 Manual for submission, evaluation and grant award. See also the proposal templates for your specific action in section 5, below.

4. Indicative timetable for evaluation and grant agreement:

Notification of outcomes of stage 1 evaluations: Maximum 5 months from deadline for submitting proposals.
Notification of outcomes of stage 2 evaluations: Maximum 5 months from deadline for submitting full proposals.

Signature of grant agreements: maximum 3 months from the date of informing successful applicants.

5. Provisions, proposal templates and evaluation forms:

IMI2 Research and Innovation Action (IMI2-RIA) and (IMI2-IA):

Proposal templates are available after entering the submission tool

Standard evaluation form 

IMI2 Model Grant Agreement

Clinical trial template – the Clinical Trial template is compulsory at stage 2 only !

6. Additional provisions:
Open access must be granted to all scientific publications resulting from Horizon 2020 actions, and proposals must refer to measures envisaged. Where relevant, proposals should also provide information on how the participants will manage the research data generated and/or collected during the project, such as details on what types of data the project will generate, whether and how this data will be exploited or made accessible for verification and re-use, and how it will be curated and preserved.

This topic participates per default in the open access to research data pilot which aims to improve and maximise access to and re-use of research data generated by projects:

- The pilot applies to the data needed to validate the results presented in scientific publications. Additionally, projects can choose to make other data available for open access and need to describe their approach in a Data Management Plan (to be provided within six months after the project start).

- Note that the evaluation phase proposals will not be evaluated more favourably because they are part of the Pilot, and will not be penalised for opting out of the Pilot.

- Projects can at any stage opt-out of the pilot.

The legal requirements for projects participating in this pilot are in the article 29.3 of the Model Grant Agreement.
Further information on the Open Research Data Pilot is made available in the H2020 Online Manual: http://ec.europa.eu/research/participants/docs/h2020-funding-guide/cross-cutting-issues/open-access-dissemination_en.htm 

7. Additional documents:

Summary of the most relevant provisions for participating in IMI2 actions

IMI2 Annual Work Plan 2017 

IMI2 Regulators Guidance tool for researchers 

IMI JU derogation to H2020 Rules for Participation  

Horizon 2020 Rules for Participation 

Horizon 2020 Regulation of Establishment

Horizon 2020 Specific Programme 

 

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To access existing draft proposals for this topic, please login to the Participant Portal and select the My Proposals page of the My Area section.

Type of Action Research and Innovation action [IMI2-RIA]
Topic Discovery and characterisation of blood-brain barrier targets and transport mechanisms for brain delivery of therapeutics to treat neurodegenerative & metabolic diseases - IMI2-2017-12-06
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