The development of a therapeutic carrier

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Neurological diseases associated with cancers, genetic diseases, infectious diseases and aging create a significant social and economic burden. Despite advances in technologies that have spawned new drug targets and therapeutic candidates for neurological diseases, we have yet to find successful clinical therapies, due in part to disease complexity and the individual variability within the human population. However, the most serious barrier to treatment is often inefficient delivery of drugs to the disease-affected brain tissue. This is due to restrictions dictated by the existence of the blood-brain barrier (BBB). Designing efficient 'vectors' (antibodies, protein carriers, viruses, nanoparticles) to deliver therapeutics across the BBB in a controlled and non-invasive manner remains one of the key goals of drug development for brain diseases. We have demonstrated that by using a patented carrier molecule, chemotherapeutic drugs can be delivered to the brain and be effective against cancer. There also exists preliminary data in the patent that this carrier may be useful by acting as a conjugate to treat diseases of the central nervous system (CNS). The purpose of this proposal is to suggest experiments that will extend the life of the patent in order to allow this technology to be produced and therefore become useful in a therapeutic setting. This proposal outlines experiments that will provide a model the carrier protein to act as a delivery vehicle when conjugated to a therapeutic enzyme, as well as to begin to elucidate the specific interactions that take place which allow the carrier protein to transport into and across a physical cellular barrier such as the BBB.