SBIR Phase I: A Novel Platform to Enable Directed Delivery of Therapeutics into Brain Injuries

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The broader impact/commercial potential of this Small Business Innovation Research (SBIR) Phase I project is to enable transformative advances into the treatment of traumatic brain injury (TBI) and positively impact the lives of injury victims, such as soldiers, their families and the society in general by reducing the degree of disability and increasing independence. With over 10 million people worldwide sustaining TBI annually, it is the leading cause of death and disability in the most active segment of the population. The enormous financial and human suffering burden of TBI provides a compelling rationale for the development of novel and innovative agents and approaches to TBI management. Despite an obvious need, there are currently no clinically proven, effective drugs to limit secondary injury or enhance repair in TBI. Precision-guided delivery of drugs specifically to injured areas in the TBI brain may result in a paradigm shift in the clinical management of TBI. The potential for clinical application of such an approach is high, as the targeted delivery technology can be used for delivering any therapeutic or diagnostic agent. Such a targeting technology has not previously been available for treatment of brain injury and therefore there exists a huge commercial opportunity. The proposed project focuses on developing a targeting platform for site-specific delivery of drugs to brain injury. One of the reasons for the failure of many neuro-protective agents in clinical trials is due to the dose limiting effects of these drugs. Incorporating a targeted delivery strategy can circumvent this. We have identified a novel targeting peptide that specifically recognizes TBI. Using animal models, we propose to demonstrate that combining therapeutics with this peptide can improve delivery of drugs to brain injury. In this proposal, we will test two different types of drugs (a protein therapeutic and antisense nucleic acids) in conjunction with our targeting peptide to demonstrate the validity of the principle of therapeutic targeting. We expect that an intravenous administration of targeted drugs will lead to a higher accumulation in brain injury than untargeted drug. This strategy can be expected to improve therapy, and also diagnosis, of brain injuries. It may also advance the understanding of the molecular changes associated with brain injuries and may provide new therapeutic targets. With this platform technology, transformative advances in brain injury treatment in the form of increased efficacy, reduced side effects, and ease of administration are expected to ensue