The Blood-Brain Barrier (BBB) is a specialized and dynamic membrane that protects the brain from the external blood-flow. The blood, in fact, contains and transports a number of molecules that can be toxic to the brain. The BBB is also essential in selecting which molecules can enter the brain and the spinal cord and these include therapeutic drugs, thus affecting patient treatment.
BBB dysfunction is involved in several neurodegenerative pathologies and by creating a model in the laboratory we may be able to explore it and identify alterations involved in disease. A BBB model will be like an open window to explore the connection between the brain and the rest of the body.
Because of its complex function, different layers of cells, all with their characteristics and specialised roles, form the BBB. As part of the EuroNeuroTrophin Consortium, we use cells donated by patients affected by motor neurone disease/amyotrophic lateral sclerosis (MND/ALS), to reproduce the most accurate BBB model, so that we can better understand its impairment in disease, and explore how these alterations affect disease and the transport of drugs from the blood to the brain. We will use this laboratory model to identify the most potent neurotrophin mimetics that are also efficient in crossing the BBB, thus reaching the brain and spinal cord. This model is an essential step in our translational research programmes.