As the Center for Rare Neurological Diseases (CRND) pursues the development of targeted therapies for the treatments for rare lysosomal diseases, we also must create successful screening and delivery methods for these drugs.
Therapies for neurological disease need to successfully reach the brain and often times it is the blood-brain barrier that prevents novel drugs from entering the brain. This semi-permeable barrier prevents potentially harmful materials in the blood (such as bacteria, viruses, chemicals) from entering the brain. Although the blood-brain barrier is meant to protect the brain, it also represents a barrier for therapeuic agents delivered intravenously or orally. Such therapies need to pass the blood-brain barrier in order to reach sufficient concentrations in the brain. Our nanotechnology-based approaches (technology used to build tiny structures on a molecular level), innovative screening tools and state-of-the-art medicinal chemistry offer great promise for meeting this need.
We believe that our collaborative efforts, bringing together Northwestern University experts in neurology, biomedical engineering, nanotechnology, neuroproteomics, and disease modeling, will allow us to approach our research in a more holistic way and will lead to innovative and novel solutions that have eluded traditional biomedical research models.