Case’s Elaprase Treatment

Posted Saturday April 02, 2011 by Melissa

Hospital bed sleeping

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.

You may also like:

10 Responses to “Case’s Elaprase Treatment”

  1. This was so neat to see. I just stood here and watched it with Kathy Williams and she was commenting on how wonderful it is to see his progress in Sunday school.
    It is just neat to see a little picture of what he (and you!) are actually doing all these days!

    • Thanks Sarah! We are so thankful for his progress and that he is becoming more engaged with everyone around him – you and Kathy included!

  2. Thanks for sharing that. He is such a pro! I can definitely tell the treatments are making a difference. What a blessing to be able to get them. And…the skypping with Daddy just brought a tear to my eye. 🙂

    • Oh, he loves Skype! If you are on, you’ll have to friend us so we can call you some night we’re here. He would love that.

  3. Melissa this site is absolutely amazing! The videos are so clear… I feel so close almost there… You and Case are such a team… may you continue to be blessed and a blessing!!

    • Thanks Nana. I hope someone might be blessed by these videos. Case sure enjoys watching himself in them! The iPad makes taking and editing them incredibly easy.

  4. Melissa-thank you so much for sharing. I have worked in clinical research for 11 years but never know the persons participating in drug trials. It was important for me to see this part of the process and to remember that I’m not just doing a job. The reason I go to work every day to help people just like Case. May God continue to bless your whole family!

    • Thanks Jen! We so appreciate all the people that work hard to come up with drugs like these. We’re still hoping to get together here sometime soon while we’re here!

  5. i want to know about those doctors/manufacturers who can provide me elaprase.
    my two child are affected by mps 2 disease . so pls kindly reply me