Novel Tissue-Specific Multifunctionalized Nanotechnological Platform Encapsulating Riluzole Against Motor Neuron Diseases
Year: 2025
Type: article
Abstract: Background: Motor neuron diseases are neurological disorders characterized by progressive degeneration of upper and/or lower motor neurons. Amyotrophic Lateral Sclerosis (ALS) is the most common form of motor neuron diseases, where patients suffer progressive paralysis, muscle atrophy and finally death. Despite ALS severity, no treatment is safe and fully effective. In this area, Riluzole was the first drug approved and it constitutes the gold-standard for this pathology. However, to obtain suitable therapeutic efficacy, Riluzole requires high doses that are associated with severe adverse effects in other tissues. To attain Riluzole therapeutic efficacy avoiding other organs side-effects, new therapeutic strategies to enhance the delivery of Riluzole specifically to motor neurons constitute an unmet medical need. In this area, we have developed a novel multifunctional nanostructurated carrier to selectively deliver Riluzole to motor neurons. Results: This work develops and characterizes at in vitro and in vivo levels a tissue-targeted formulation of peptide and PEG-labelled PLGA nanoparticles encapsulating Riluzole. For this purpose, pVEC, a cell penetrating peptide able to increase transport through the blood-brain barrier, was attached to the nanoparticles surface. The multifunctionalized nanoparticles show suitable characteristics for the release of Riluzole in the central nervous system and were detected in motor neurons within 1 h after administration while significantly reducing the concentration of Riluzole in non-therapeutic organs responsible of side effects. Conclusion: A novel drug delivery system has been developed and characterized, demonstrating enhanced CNS biodistribution of riluzole, which shows promise as efficient therapeutic tool for motor neuron diseases, including amyotrophic lateral sclerosis.
DOI: 10.2147/ijn.s479819
Institutions: Consejo Superior de Investigaciones Científicas, Institute of Advanced Chemistry of Catalonia, Institut de Nanociència i Nanotecnologia de la Universitat de Barcelona, Universitat de Barcelona, Biomedical Research Networking Center on Neurodegenerative Diseases, Institut de Recerca Biomèdica Catalunya Sud, Universitat Autònoma de Barcelona, University College Dublin
Language: English
Subfield: Biomaterials
Field: Materials Science
Domain: Physical Sciences
Open Access status: gold
Estimated APC paid: $2,836
Awards: G1175115578, G1197054721, G1855990336, G2262748287, G2304992279, G2309174360, G2436713165, G279363373, G2855141111, G300979063, G3031149416, G3071963729, G34346518, G3480869486, G3518538256, G4042007251, G410571670, G4170490609, G425668148, G430777504, G451917667, G5080475149, G5208757957, G5421974903, G5967599077, G7029449665, G7434936867, G7815222579, G8339870507, G8468985988, G8599964989, G8920660035, G897484643, G904308495, G929308097, G99296377
OpenAlex ID: W4407828782