Focusing on disease-modifying rather than etiologic mechanisms may accelerate the development of novel therapies and complement current research initiatives on causative mechanisms and curative therapies.
Bron
Verkorte titel
Aandoening
ALS, MND, amyotrophic lateral sclerosis
Ondersteuning
Vesalius Research Center, VIB, Leuven, Belgium
University Hospital Jena, Germany
Onderzoeksproduct en/of interventie
Geen registraties gevonden.
Uitkomstmaten
Primaire uitkomstmaten
1. Report on key molecular drivers in transcriptomic data related to disease progression (month 35);<br>
2. Report on key molecular drivers in methylation data related to disease progression (month 35);<br>
3. Report on key molecular drivers in proteomic data related to disease progression (month 35);<br>
4. Report on longitudinal MRI and MUNIX changes related to disease progression (month 35).
Achtergrond van het onderzoek
Amyotrophic lateral sclerosis is a relentlessly progressive neurodegenerative disorder of motor neurons, leading to death of the patient on average only 36 months after disease onset. ALS is characterized by heterogeneity at the clinical and genetic level. Causative mechanisms of motor neuron degeneration are studied in several European studies, in particular Euro-MOTOR. Notwithstanding these efforts, 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. Data collection and sample management will be done according to harmonized European ALS sampling and data management standards developed through the EU funded project SOPHIA. In addition, the detailed prospective clinical monitoring of patients will allow us reliably delineate the parameters that predict fast disease progression already in patients early in the disease. Insight into disease modifying factors will enhance our understanding of motor neuron degeneration and reveal potential therapeutic strategies.
Doel van het onderzoek
Focusing on disease-modifying rather than etiologic mechanisms may accelerate the development of novel therapies and complement current research initiatives on causative mechanisms and curative therapies.
Onderzoeksopzet
Month 35.
Onderzoeksproduct en/of interventie
N/A
Publiek
Dept Neurology, G03.232<br>
Heidelberglaan 100
Petra Berk
Utrecht 3584 CX
The Netherlands
+31 (0)88 7551221
P.A.Berk@umcutrecht.nl
Wetenschappelijk
Dept Neurology, G03.232<br>
Heidelberglaan 100
Petra Berk
Utrecht 3584 CX
The Netherlands
+31 (0)88 7551221
P.A.Berk@umcutrecht.nl
Belangrijkste voorwaarden om deel te mogen nemen (Inclusiecriteria)
All patients with ALS.
Belangrijkste redenen om niet deel te kunnen nemen (Exclusiecriteria)
N/A
Opzet
Deelname
Opgevolgd door onderstaande (mogelijk meer actuele) registratie
Geen registraties gevonden.
Andere (mogelijk minder actuele) registraties in dit register
Geen registraties gevonden.
In overige registers
Register | ID |
---|---|
NTR-new | NL3704 |
NTR-old | NTR3902 |
Ander register | ZonMw : 40-42900-98-1008 |
ISRCTN | ISRCTN wordt niet meer aangevraagd. |