Bioimage-neuromuscular diseases

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Project Title: Bioimage-neuromuscular diseases
Project Number: CORDIS-110240
Project web address: Follow on CORDIS
Organization: University Of Newcastle Upon Tyne, United Kingdom, Newcastle Upon Tyne
Collaborators: University College London, GB
Katholieke Universiteit Leuven, BE
Università Cattolica Del Sacro Cuore, IT
Academisch Ziekenhuis Leiden, NL
Scito S.A., FR
Association Institut De Myologie, FR
Consultants For Research In Imaging And Spectroscopy Scrl, BE
Biomarin Nederland Bv, NL
Principal Investigators (PI): Helen Gallon, GB
Elke Lammertyn, BE
Filippo Leone, IT
Raymond Gilles, BE
Christelle Gaultier, FR
Gosia Kielbasa, GB
Jan Verschuuren, NL
Isabel Ferreira, NL
Arno Klaassen, FR
 
Project Description:
BIOIMAGE-NMD will develop and apply imaging technology to monitor response to novel therapies in neuromuscular diseases (NMD) and will use Duchenne muscular dystrophy (DMD) as an exemplar disease. DMD is well characterised genetically and clinically but to date a disease modifying treatment is not available. One of the most promising developments for future treatment of NMD is RNA modulation through antisense oligonucleotides (AON). In DMD, AON are used for exon skipping and this is a genuine example of personalised medicine, where patients are treated according to their specific gene mutation.Quantitative Magnetic Resonance Imaging (MRI) and Magnetic Resonance Spectroscopic Imaging (MRSI) are used to assess structural and metabolic muscle pathology in NMD but their effectiveness in monitoring therapy is yet to be shown. The project will apply a simultaneous MRI/MRSI protocol in multi-centre clinical trials of AON therapy in DMD with the aim of establishing a clinical proof of principle that these imaging measures are effective biomarkers of therapeutic response. To enhance the imaging protocol, novel Diffusion Tensor MRI (DTI) methods will be developed and optimised to assess muscle microstructure and applied in these trials. The project will also develop methods for radiolabelling of AON and demonstrate the use of pre-clinical Positron Emission Tomography (PET)/MRI to assess the tissue targeting, bio-distribution and pharmacokinetics of AON in vivo.BIOIMAGE-NMD will deliver PET/MRI and MRI/MRSI technologies for both drug development and clinical evaluation roles which will significantly contribute to bringing personalized therapeutic interventions in rare and common diseases to the market.
 
Project Terms:
life sciences
Project Title: Bioimage-neuromuscular diseases
Project Number: CORDIS-110240
Project web address: Follow on CORDIS
Organization: University Of Newcastle Upon Tyne, United Kingdom, Newcastle Upon Tyne
Collaborators: University College London, GB
Katholieke Universiteit Leuven, BE
Università Cattolica Del Sacro Cuore, IT
Academisch Ziekenhuis Leiden, NL
Scito S.A., FR
Association Institut De Myologie, FR
Consultants For Research In Imaging And Spectroscopy Scrl, BE
Biomarin Nederland Bv, NL
Principal Investigators (PI): Helen Gallon, GB
Elke Lammertyn, BE
Filippo Leone, IT
Raymond Gilles, BE
Christelle Gaultier, FR
Gosia Kielbasa, GB
Jan Verschuuren, NL
Isabel Ferreira, NL
Arno Klaassen, FR
 
Project Categories:
Natural Sciences > Clinical Trials & Therapy > Treatment
 
Other Information:
Fiscal Year: 2013
Project Start Date: 1 October 2013
Project End Date: 31 March 2017
Project program: FP7-HEALTH
 
Project Funding Information:
Funding Mechanism: CP-FP - Small or medium-scale focused research project
Year Funding Organization Total Funding, $
2013 European Research Council $8,658,726