Central nervous system (CNS) diseases and injuries are one of the most devastating for patients. In particular, Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disorder with no cure. Actually, it is estimated that the number of ALS cases across the globe will increase 69% from 2015 to 2040. So, there is an urgent need on a detailed knowledge of the pathophysiology of CNS disorders, but also for more specific and effective delivery strategies. In this context, we proposed to use a novel nanomedicine approach using targeted cell delivery vehicles to the CSF that can be guided/monitored by MRI. This novel one-package system allows providing a therapeutic effect by control delivery of drugs and cells and monitoring in vivo their biodistribution. Advances in micro-/nano-biomaterials field will provide new opportunities for an effective treatment of neurological diseases.