Multifaceted nanoparticles: emerging mechanisms and therapies in neurodegenerative diseases

Developing drugs that can cross the blood-brain barrier is a major challenge for the treatment of neurodegenerative disease. Mistretta et al. discuss the potential of nanoparticle-based systems to overcome this difficulty and review their use in Friedreich's ataxia as a test case. Neurodegenerative diseases are a major global health burden particularly with the increasing ageing population. Hereditary predisposition and environmental risk factors contribute to the heterogeneity of existing pathological phenotypes. Traditional clinical interventions focused on the use of small drugs have often led to failures due to the difficulties in crossing the blood-brain barrier and reaching the brain. In this regard, nanosystems can specifically deliver drugs and improve their bioavailability, overcoming some of the major challenges in neurodegenerative disease treatment. This review focuses on the use of nanosystems as an encouraging therapeutic approach targeting molecular pathways involved in localized and systematic neurodegenerative diseases. Among the latter, Friedreich's ataxia is an untreatable complex multisystemic disorder and the most widespread type of ataxia; it represents a test case to validate the clinical potential of therapeutic strategies based on nanoparticles with pleiotropic effects.

Automated summary

Developing drugs that can cross the blood-brain barrier is a challenge for treating neurodegenerative disease. The use of nanoparticle-based systems shows potential in overcoming this difficulty, with the example of Friedreich's ataxia. Neurodegenerative diseases are a global health burden, and traditional interventions have limitations in reaching the brain. Nanosystems offer a promising therapeutic approach by delivering drugs and improving bioavailability. This review highlights the use of nanosystems in targeting molecular pathways involved in neurodegenerative diseases, with Friedreich's ataxia as an example.