|PERT diagram (Click to zoom)|
MIROCALS aims to perform a novel intervention study with the potential to:
- halt disease progression
- test the proof of concept that biomarkers can accelerate future intervention studies
- provide unique insights on disease pathogenesis identifying novel targets for intervention.
The workprogramme has been structured in 7 workpackages (WPs):
WP2 and WP3 relate to the performance of the clinical trial
Work package 2: Clinical trial implementation
WP2 aim at designing, getting approval and organizing the clinical trial.
Work package 3: Clinical trial conduct
In this WP, the clinical trial will be carried out in compliance with GCP/GCLP rules, to demonstrate the efficacy and safety of ultra-low-dose IL-2 for patients with recently diagnosed ALS.
WP4, WP5 and WP6 relate to the specific laboratory investigations using CSF and blood samples collected during the trial to get better insights into the effects of riluzole and low dose IL-2 in ALS patients.
Work package 4: Deep immuno-phenotyping
WP4 aims to establish the effect of low dose IL-2 on key effectors and regulators of the neuro-inflammatory process and establish their relationship with treatment responses and disease activity.
Work package 5: Brain Biomarkers
The main focus of this WP will be on neurofilaments, whose dynamic of release into biological fluids reflects the progressive cytoskeletal break-down of neurons and axons.
Work package 6: Genomics and Transcriptomics
The main objective of WP6 is to establish the changes in transcriptomic profiles in response to both riluzole and IL-2 treatment and to characterise the gene expression signatures of responders to IL-2 versus those of non-responders.
WP1 and WP7 will ensure the project management as well as organizing the dissemination of the project’s results to patients and other stakeholders.