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MCDS-Therapy SIGNED

Repurposing of carbamazepine for treatment of skeletal dysplasia

Total Cost €

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EC-Contrib. €

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Partnership

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 MCDS-Therapy project word cloud

Explore the words cloud of the MCDS-Therapy project. It provides you a very rough idea of what is the project "MCDS-Therapy" about.

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Project "MCDS-Therapy" data sheet

The following table provides information about the project.

Coordinator
UNIVERSITY OF NEWCASTLE UPON TYNE 

Organization address
address: KINGS GATE
city: NEWCASTLE UPON TYNE
postcode: NE1 7RU
website: http://www.ncl.ac.uk/

contact info
title: n.a.
name: n.a.
surname: n.a.
function: n.a.
email: n.a.
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 Coordinator Country United Kingdom [UK]
 Project website https://mcds-therapy.eu/
 Total cost 5˙697˙390 €
 EC max contribution 5˙697˙390 € (100%)
 Programme 1. H2020-EU.3.1.3. (Treating and managing disease)
 Code Call H2020-SC1-2017-Two-Stage-RTD
 Funding Scheme RIA
 Starting year 2017
 Duration (year-month-day) from 2017-12-01   to  2022-11-30

 Partnership

Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    UNIVERSITY OF NEWCASTLE UPON TYNE UK (NEWCASTLE UPON TYNE) coordinator 2˙175˙161.00
2    ISTITUTO ORTOPEDICO RIZZOLI IT (BOLOGNA) participant 782˙175.00
3    UNIVERSITAETSKLINIKUM FREIBURG DE (FREIBURG) participant 657˙569.00
4    SCIOMICS GMBH DE (HEIDELBERG) participant 604˙030.00
5    FINOVATIS FR (LYON) participant 320˙937.00
6    MURDOCH CHILDRENS RESEARCH INSTITUTE AU (PARKVILLE) participant 318˙625.00
7    FINDACURE FOUNDATION UK (CAMBRIDGE) participant 309˙235.00
8    ASSISTANCE PUBLIQUE HOPITAUX DE PARIS FR (PARIS) participant 211˙479.00
9    GUYS AND ST THOMAS' NHS FOUNDATIONTRUST UK (London) participant 197˙428.00
10    UNIVERSITAIR ZIEKENHUIS ANTWERPEN BE (EDEGEM) participant 120˙750.00

Map

 Project objective

Genetic skeletal diseases (GSDs) are an extremely diverse and complex group of rare genetic diseases that affect the development the skeleton. There are more than 450 unique and well-characterised phenotypes that range in severity from relatively mild to severe and lethal forms. Although individually rare, as a group of related genetic skeletal diseases, GSDs have an overall prevalence of at least 1 per 4,000 children, which extrapolates to a minimum of 225,000 people in the 27 member states and candidate countries of the EU. This burden in pain and disability leads to poor quality of life and high healthcare costs. Metaphyseal chondrodysplasia, type Schmid (MCDS) results from mutations in collagen X and affects <1/100,000 of the population. Mutant collagen X molecules miss-fold during synthesis and are retained within the endoplasmic reticulum (ER) of hypertrophic chondrocytes, thereby causing ER stress. Our extensive pre-clinical studies have shown that carbamazepine (CBZ) can alleviate ER stress caused by the expression of mutant collagen X and restore bone growth in a validated mouse model of MCDS. CBZ is an FDA approved drug used for the treatment of epilepsy and bipolar disorder and received orphan drug designation by the European Commission for the treatment of MCDS in September 2016. MCDS-Therapy is a 5-year collaborative project comprising world-renown clinical centres and SMEs to advance the repurposing of CBZ for MCDS (up to the Marketing Authorization Application dossier) through a multicentre and multinational (EU & AUS) clinical trial (Phase1, Phase2/3). MCDS-Therapy also encompasses biomarker development and health economics assessment studies to deliver by 2022 an innovative and affordable (CBZ already exists in a generic form) repurposed therapy for MCDS along with the diagnosis/prognosis tools to personalise the treatment strategy.

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The information about "MCDS-THERAPY" are provided by the European Opendata Portal: CORDIS opendata.

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