An oral medication for multiple sclerosis may rest in a drug originally intended for cancer
Today's protein-based therapies for multiple sclerosis (MS) are only partially effective. They are expensive and difficult to manufacture, and unlike small molecule drugs, these biologics cannot be taken orally. This represents an additional burden for patients with this chronic disease. One of the current challenges in MS care is developing new, orally available therapies.
Yuhong Yang, MD, is working on this problem at The Ohio State University Wexner Medical Center by focusing on inhibitors of the cytokine IL-6 and the transcription factor Stat3 in T cells. This pathway sits at a crossroads in the pathology of MS: It regulates processes in T cells that cause them to become pathogenic, and it also suppresses cells that control that pathogenic immune response.
Coincidentally, Dr. Yang's colleague Chenglong Li, PhD, in Ohio State's College of Pharmacy had been developing new small molecules to target this pathway in cancer. When he saw Dr. Yang's work studying this pathway in EAE (an MS model disease in mice), he proposed that they join forces to work on the improved therapy together.
In 2013, Dr. Yang began to test lead compounds developed in Dr. Li's laboratory in EAE with a pilot grant from the National Multiple Sclerosis Society. Promising results from that initial research have led to a formal collaboration and an R01 drug development grant from the National Institutes of Health.
Over the next three years, Dr. Yang will be studying the drug candidates in mice with EAE and using peripheral blood cells from patients with MS as Dr. Li continues to optimize these compounds to maximize their stability and potency. Their ultimate goal is to develop a compound that could be tested in a future clinical trial with MS patients.