Phase 2 of JAR of Hope’s hyperbaric oxygen chamber treatment (HBOT) study will begin at Duke University in the first quarter of 2019, headed by Dr. Joanne Kurtzberg. The study, which originally aimed to examine the hypothesis of increased oxygen to enhance cardiac, respiratory, and skeletal muscle function, will now shift to stem cell research. Specifically, testing the hypothesis that HBOT will allow stem cells to live longer within a mouse model using the Duchenne phenotype. Stem cells, which are the body’s raw materials—cells from which all other cells with specialized functions are generated, offer considerable promise for a plethora of new medical treatments and can enhance our understanding of how diseases occur. By watching stem cells mature into cells in bones, heart muscle, nerves, and other organs and tissue, stem cells allow researchers and doctors to better understand how diseases and conditions develop.
HBOT is critical to Duchenne muscular dystrophy (DMD) research because the delivery of oxygen to the tissues is vital for sustaining life. In patients with DMD, this fundamental task is impeded through a variety of mechanisms, causing hypoxia (a deficiency of oxygen reaching the tissue) and other complications. DeWayne Townsend, DVM, PhD, who in 2016 spearheaded Phase 1 of the study at the University of Minnesota, had initially uncovered the adverse result of introducing mice to a hypoxic environment and was shocked by its consequences. Using otherwise healthy mice, Dr. Townsend placed them in an environment with decreased oxygen levels in their bloodstreams, and found a significant amount of scarring and damage after removing the mice weeks later.
Such findings demonstrated that hypoxia alone is sufficient in causing significant changes in dystrophic mice. This, and other data, indicates that dystrophic mice have an augmented susceptibility to hypoxia. The HBOT was intended to prove increased oxygen levels will enhance muscle performance in these mice, which it did. The theory was supported by the fact that Jamesy Raffone stands as one of the first-ever children with DMD to undergo the HBOT treatment. Jamesy experienced a marked improvement in his cardiac and motor functions after being administered oxygenated therapy, and catalyzed the mission to proceed with this groundbreaking endeavor.
Now, the next step—Dr. Kurtzberg will lead the team studying the second phase of the theory that intensified levels of oxygen will prolong the life of stem cells in dystrophic mice. If proven true, such results will significantly improve muscle function in DMD patients. This initiative was facilitated with the help of New Jersey Congressman Chris Smith, who is pushing to drive awareness through congress regarding DMD. “If our hypothesis is correct, then Duchenne patients will soon have access to a method that would slow the progression of the disease,” remarked Jim Raffone. “We believe that higher oxygen levels can preserve the lifecycle of stem cells further increasing the body’s ability to improve muscle function. We have the utmost confidence in the ability of Dr. Kurtzberg in leading this study and we look forward to positive results.”