Every Thursday since July 2009, Case has traveled to Vanderbilt Children’s Hospital to receive an infusion of a drug called Elaprase. So, by now, Case has had approximately 80 infusions (he’s only missed about 2 because of sickness), but he will have to continue these every week for the rest of his life.
Elaprase was approved by the FDA in 2006 and is made by Shire HGT, a division of Shire Pharmaceuticals. Elaprase works by acting as the enzyme Case is missing in his body (iduronate sulfatase) – it goes into the bloodstream and removes the GAG in many cells that his body is otherwise unable to remove.
Since starting Elaprase, Case’s walking, climbing, jumping, breathing, talking, etc. abilities have improved immensely. We are hopeful that the Elaprase will stabilize his heart valves, airway issues, and other serious complications of Hunter Syndrome, but there is too little data yet to know how well it will work on those issues.
Elaprase, however, does not cross the blood-brain barrier and thereby stop the brain decline in our boys. So, the intrathecal drug in the clinical trial that Case is involved in is hoping to address this. While we are at UNC for the clinical trial, Case also receives his Elaprase infusion there – depicted in this video.