Evaluation of serum MMP-9 as predictive biomarker for antisense therapy in Duchenne View Full Text


Ontology type: schema:ScholarlyArticle      Open Access: True


Article Info

DATE

2017-12-20

AUTHORS

A. Lourbakos, N. Yau, P. de Bruijn, M. Hiller, K. Kozaczynska, R. Jean-Baptiste, M. Reza, R. Wolterbeek, Z. Koeks, B. Ayoglu, D. de Klerk, G. Campion, I. Zaharieva, V. D. Nadarajah, P. Nilsson, C. Al-Khalili Szigyarto, F. Muntoni, H. Lochmüller, J. J. Verschuuren, N. Goemans, M. Tulinius, E. H. Niks, S. de Kimpe, A. Aartsma-Rus, Peter A. C. ’t Hoen, P. Spitali

ABSTRACT

Duchenne Muscular Dystrophy (DMD) is a severe muscle disorder caused by lack of dystrophin. Predictive biomarkers able to anticipate response to the therapeutic treatments aiming at dystrophin re-expression are lacking. The objective of this study is to investigate Matrix Metalloproteinase-9 (MMP-9) as predictive biomarker for Duchenne. Two natural history cohorts were studied including 168 longitudinal samples belonging to 66 patients. We further studied 1536 samples obtained from 3 independent clinical trials with drisapersen, an antisense oligonucleotide targeting exon 51: an open label study including 12 patients; a phase 3 randomized, double blind, placebo controlled study involving 186 patients; an open label extension study performed after the phase 3. Analysis of natural history cohorts showed elevated MMP-9 levels in patients and a significant increase over time in longitudinal samples. MMP-9 decreased in parallel to clinical stabilization in the 12 patients involved in the open label study. The phase 3 study and subsequent extension study clarified that the decrease in MMP-9 levels was not predictive of treatment response. These data do not support the inclusion of serum MMP-9 as predictive biomarker for DMD patients. More... »

PAGES

17888

Journal

TITLE

Scientific Reports

ISSUE

1

VOLUME

7

Identifiers

URI

http://scigraph.springernature.com/pub.10.1038/s41598-017-17982-y

DOI

http://dx.doi.org/10.1038/s41598-017-17982-y

DIMENSIONS

https://app.dimensions.ai/details/publication/pub.1099719839

PUBMED

https://www.ncbi.nlm.nih.gov/pubmed/29263366


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