There currently are no commercially available systems for specific delivery of pharmaceuticals to motor neurons. Many drugs that have potentially beneficial uses in treating disorders of the cholinergic nervous system are unavailable for use in motor neuron diseases due to drawbacks such as insolubility problems, short systemic half-lives, and high systemic toxicity. The drug delivery system described in this proposal could be a valuable tool for treatment of neuronal disorders due to the fact it specifically targets and delivers pharmaceuticals directly to the site of action (motor neurons). This therapy system circumvents undesirable systemic side-effects and toxicities. Our research will demonstrate the feasibility of delivering compounds such as peptide inhibitors of enzymes and low molecular weight organic enzyme inhibitors to motor neurons.
