A variety of therapies have been attempted to treat Multiple Sclerosis. Such experimental therapies have included the administration of myelin basic protein, administration of peptides derived from myelin basic protein, injection of the myelin basic protein peptide motif mimic, glatiramer acetate, and most recently, treating with DNA encoding myelin basic protein. The early clinical trials using such therapy have showed no, or little, clinical benefit. Based on these past clinical trials, it is clear that more efficacious therapies for Multiple Sclerosis patients are needed. The overall goal for the SBIR Phase I studies proposed here is to develop a novel formulation for use in treating Multiple Sclerosis patients. To begin to accomplish this task, we will express a novel protein in transgenic soybean seeds. The successful expression of this novel protein will allow soy formulations to be tested for efficacy in treating Multiple Sclerosis. To accomplish this task, the following milestones are proposed: Milestone #1: A novel fusion protein for treating Multiple Sclerosis will be expressed in transgenic soybeans. In milestone #1, we will establish soybean lines and produce soybean seeds in bulk quantities that can be used to make soy formulations for characterization (Milestone #2) and for use in efficacy studies (Milestone #3). Milestone #2: We will characterize the novel soybean-derived fusion protein and demonstrate that this protein retains it normal properties and functionality. Milestone #3: Demonstrate that soy formulations made from seeds expressing the fusion protein shows efficacy in treating a model of Multiple Sclerosis. Despite the disappointments and limited success of human clinical trials aimed at treating Multiple Sclerosis, the potential for such therapies is too great to ignore. In this Phase I SBIR, we propose to express a novel fusion protein in transgenic soybeans so that soy formulations can be assessed for their efficacy in treating a model of Multiple Sclerosis. Success with this Phase I SBIR studies will set the stage for future SBIR studies which will demonstrate safety and efficacy of such therapy in Multiple Sclerosis patients.
Based on past clinical trials, it is clear that more efficacious therapies for Multiple Sclerosis patients are needed. The overall goal for the SBIR Phase I studies proposed here is to develop a novel formulation for use in treating Multiple Sclerosis patients. The successful expression of this novel formulation will allow testing for efficacy in treating Multiple Sclerosis.
