Researchers at NIH and Sequella, a biotechnology company based in Rockville, MD, are working together to produce the first new drug to treat Tuberculosis in over 30 years

Tuberculosis (TB) is a disease of mammoth proportions both in human lives lost and economic impact. Although preventative measures in this country have left the US relatively unscathed, TB is the second leading cause of death in adults worldwide. It disproportionately affects populations with poorer access to healthcare, particularly those in developing nations. Because TB is transmitted through the air, it is highly contagious. Furthermore, the World Health Organization (WHO) expects that the number of people stricken with TB will increase in the coming decades. According to the Global Alliance for TB Drug Development, more than eight million new people develop active tuberculosis every year and 2 million people die from TB, yet no new TB drugs have been introduced in over 30 years. The dearth of new TB therapeutics has bestowed TB with the dubious distinction of being a 'neglected disease'. Current therapeutic regimens require that drugs be taken continuously for 6 to 9 months. The difficulty of ensuring patient compliance over such a long period is compounded by the fact that current therapies also have very unpleasant side effects. These factors contribute to high mortality rates as well as the emergence of new drug-resistant TB strains. Therefore, a new drug that could treat TB in a shorter amount of time and/or with fewer side effects is highly desirable. Research aimed at producing novel TB therapies was initiated by Dr. Clifton Barry, III of the National Institute of Allergy and Infectious Diseases (NIAID) within NIH. He screened over 100,000 compounds that are chemical variants of an anti-TB drug called ethambutol in order to find molecules that are more effective than ethambutol itself. This pool was narrowed down to three compounds that were subsequently tested in animals by the NIH researchers. It is believed that these new compounds may be effective against ethambutol-resistant TB and be less toxic than ethambutol. This work by the NIH researchers was performed in collaboration with researchers Marina Protopopova and Elena Bogatcheva from Sequella, a newly formed biotechnology company in Maryland. The company's primary focus is on translational aspects of basic research with the goal of developing novel therapeutics for some of the most acute and devastating illnesses. It is the only company that focuses exclusively on TB. In addition to TB therapeutics, Sequella is actively pursuing the development of diagnostics and vaccines that will stem the TB pandemic. The compound screening collaboration with Sequella was carried out under a Cooperative Research and Development Agreement (CRADA) and was funded by the NCI/NIAID Inter-Institute Program for Development of AIDS-related Therapeutics. Sequella's role in the CRADA was to develop a streamlined approach to synthesizing these compounds. A patent for these compounds issued in March 2006. The CRADA provided Sequella the first opportunity to obtain an exclusive license from the NIH for these compounds. Sequella exercised this option and negotiated the exclusive license through Robert Joynes, Esq. in the Office of Technology Transfer at the NIH. The NIH granted a worldwide exclusive license to Sequella in March 2006 to further development of this new drug candidate. Sequella expects to start Phase I human clinical trials in 2006. Often, research in the area of neglected diseases lags due to a perception that new treatments may not be economically fruitful. However, the Global Alliance for TB Drug Development carried out a study that indicated that the market for TB drugs could reach $700 million per year. This partnership demonstrates how the unique yet complementary strengths of public and private research institutions can be combined to target diseases for which there have been very few therapeutic advances.