On October 8, the FDA approved a regenerative medicine based on Louise Markert, M.D., Ph.D.'s, pioneering work as the only therapy for congenital athymia, a grouping of rare diseases such as complete DiGeorge Syndrome that are characterized by the lack of a functioning thymus. Without the gland, which trains the body’s T-cells to fight pathogens, seemingly harmless infections can be fatal. Without treatment, infants with congenital athymia conditions typically die by the age of three.
The newly approved therapy, which Duke licensed to Enzyvant Therapeutics, GmbH for development in 2016, uses thymus tissue that undergoes a series of intricate processes and then is implanted in babies with athymia, where it selects T-cells to recognize and attack pathogens.