Synergistic Combination of Gene Therapy and Umbilical Cord Blood Transplantation to Treat Krabbe Disease
Krabbe disease is an ultra-rare inherited lysosomal storage disorder that destroys the myelin of nerve cells in the brain and throughout the nervous system. It affects approximately 1 in 100,000 people and in most cases is diagnosed before 6 months of age, usually resulting in death before the second birthday. Children with the most severe form of Krabbe disease experience rapid neurodegeneration. The current treatment method, umbilical cord blood transplantation (UCBT), has been shown to be effective in preserving cognition and extending lifespan in individuals with early forms of the condition. However, while UCBT halts the progression of brain degeneration prior to the onset of neurological symptoms, it has not been effective in treating signs of peripheral nerve disease that cause significant motor disability for affected patients, and patients may only live until their late teens.
Description
Researchers at the University of Pittsburgh are poised to revolutionize the treatment of Krabbe disease and alter the standard of care for affected individuals by combining gene therapy with UCBT, resulting in vastly increased lifespan and rapid improvements to the nervous system. Pre-clinical studies have also shown that administering gene therapy shortly after transplantation of umbilical cord blood results in rapid and significant improvements in myelination in both the peripheral and central nervous system, suggesting that gene therapy and UCBT can combine in a synergistic manner to more effectively treat Krabbe disease. Importantly, researchers’ use of allogenic (unrelated donor) rather than autologous (self-donor) cord blood may significantly improve on results of previous attempts to combine gene therapy with UCBT.Applications
· Treatment of Krabbe disease· Treatment of other lysosomal storage disorders
Advantages
· Vastly increased lifespan over UCBT alone· Rapid improvement of myelination in both the central and peripheral nervous system