Prof. dr. J.H. Veldink
UMC Utrecht
Netherlands
PhenotYpe Research for ALS ModIfyer Discovery (PYRAMID)
ZonMw
250,000
01/06/2013
3
ALS causative mechanisms remain only partially understood and therapeutic strategies based on these mechanistic insights are largely ineffective. The only drug available- Riluzole- extends the lifespan of ALS by 6 months, presumably by targeting disease modifying rather than disease causing targets. Therefore, we hypothesize that targeting disease modifying factors is important to accelerate the development of novel, alternative therapeutics, that will complement the search for causative mechanisms and therapies already covered in other projects. In this project, using unbiased genetic, proteomic, epigenetic and gene-expression techniques, we aim to identify modifiers of disease progression. We want to exploit the phenotypic heterogeneity of patients by comparing ALS patients with very fast disease progression to patients with slow disease progression. Using this original study design we aim to identify the factors that protect a subset of patients from an aggressive disease.
http://www.zonmw.nl/nl/projecten/project-detail/phenotype-research-for-als-modifyer-discovery-pyramid/samenvatting/