New Approaches to Neurodegenerative Disease Research

Due to increasing life expectancy, the prevalence of neurodegenerative diseases such as Alzheimer's and Parkinson’s disease is increasing, while our ability for effective treatments is still limited. Despite enormous recent progress, a concerted approach of translational research is necessary:

1. Genetic approaches have provided important clues to the cause of many neurodegenerative diseases. However, much of the total heritability is still unexplained, and our understanding of gene-gene or gene-environment interactions, epigenetics or gene regulation and their role in disease is still scarce.

2. Transcriptomic, proteomic and metabolomic networks linked to genetic or non-genetic causal and modifying factors must be further deciphered in a systematic and quantitative way (systems biology)

3. Even if disease causes were completely understood, the development of treatments would be hampered by the lack of early (presymptomatic) diagnosis and of reliable biomarkers for disease state and progression.

4. There is still a dearth of validated animal and cellular models to identify drug targets, lead compounds and drugs for phase I and II trials.

5. Finally, for the successful implementation of novel treatments, a clinical study infrastructure, which includes well characterized patient cohorts with longitudinal follow-up has to be developed.

Rational treatment and prevention in complex neurodegenerative disorders will likely be based on the recognition of a network of disease causing and modifying factors (personalized medicine). Twenty-three European countries have initiated a process to identify priorities for research and to develop the necessary tools for implementation in order to meet this extraordinary challenge.