PRECISION ALS-an integrated pan European patient data platform for ALS

Area of Science:

• Neurodegenerative Diseases
• Precision Medicine
• Health Informatics

Background:

• Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disorder with a significant unmet need for effective therapies.
• Translational challenges in ALS drug development are partly due to human disease heterogeneity.
• A precision medicine approach is increasingly recognized as crucial for overcoming these translational hurdles.

Purpose of the Study:

• To develop a sustainable, precision medicine-based approach for Amyotrophic Lateral Sclerosis (ALS) drug development.
• To address key clinical, computational, data science, and technological research questions in ALS.
• To create a modular, transferable pan-European ICT framework for multimodal data collection and analysis in ALS.

Main Methods:

• Established PRECISION-ALS, an academic-industry collaboration involving diverse experts.
• Utilized a General Data Protection Regulation (GDPR) compliant framework for data collection across nine European sites.
• Integrated and analyzed multimodal data including clinical, patient journey, remote monitoring, imaging, neuro-signaling, genomic, and biomarker datasets using AI and machine learning.

Main Results:

• Generated a research-quality, multimodal, multi-sourced dataset for ALS research.
• Developed a seamless data collection, processing, and analysis framework.
• Created a first-in-kind modular and transferable pan-European ICT framework for ALS.

Conclusions:

• The PRECISION-ALS framework facilitates a precision medicine approach to ALS drug development by addressing data heterogeneity.
• The developed ICT infrastructure is adaptable for use in other regions facing similar challenges in multimodal data management.
• This initiative represents a significant step towards improving the translation of research findings into effective ALS therapies.