SlowSCA

Therapiestudie SlowSCA

01.09.2015 bis 01.09.2017

In many neurodegenerative diseases, including spinocerebellar ataxias (SCA), large populations of neurons are already lost and compensatory resources exhausted at time of clinical diagnosis. This calls for early intervention strategies aiming to slow down disease progression already at the premanifest stage of the disease. Here we propose the world-first interventional study aiming to delay onset in a rare genetic neurodegenerative disease. Specifically, we propose a piloting study using continuous motor training provided via whole body-controlled video games (exergames) to establish proof-of-concept evidence that such training leads to motor and neural changes in pre-manifest SCA subjects. The subclinical effects will be unravelled within an intraindividual control study design by elaborated quantitative VICON®-based movement analysis and structural and functional 3T magnetic resonance imaging. This will provide unique insights in underlying motor and neural networks and compensatory strategies. If successful, this piloting trial will provide the basis for a rigorous international multi-center large-scale study in a larger SCA population. Moreover, it will stimulate complementary tandem projects on effects of motor training on neural functioning and molecular pathways in premanifest SCA mouse models.