Cocktail of sonosensitive droplets for BBB-opening and brain therapy. – Sono-Cocktail

The Central Nervous System (CNS) is a therapeutic target for many brain disorders including Amyotrophic Lateral Sclerosis (ALS) that is the focus of our project. Although a leakiness of the blood-brain barrier (BBB) is admitted during the progress of some brain pathologies, crossing the BBB remains a key challenge to overcome in the treatment of most CNS diseases including ALS. Our project consists of creating a tool causing the transient opening of the BBB and then the controlled release of a drug in the CNS. To do so, we intend to use a smart US-triggered formulation composed of a cocktail of large and small perfluorocarbon droplets sensitive to different acoustic sequences. Large droplets will transiently open the BBB while small droplets will ensure the drug delivery and controlled release in the brain compartment.