Is There a Cure for MPS II?

No there is not a cure. But there is one approved treatment and another on the horizon.  On June 14, 2007, Health Canada approved an enzyme replacement therapy (ERT) called IV Elaprase, developed by Shire Human Genetic Therapies.  IV Elaprase is a weekly four hour infusion that infuses the enzyme people with MPS II are missing, into the body. It drastically reduces the physical effects of MPS II, but it cannot help the brain or central nervous system because the protein in Elaprase is too big to cross the blood brain barrier. Thus, people with MPS II, regardless of ERT, often need surgery to relieve the pressure in the brain or on the spine and may also continue to have brain involvement. Recently though, an intrathecal trial opened in North Carolina, that injects the missing enzyme right into the central nervous system. On August 19, 2011 Trey qualified for this trial and October 18, 2011, Trey received his first dose of monthly intrathecal (IT) Elaprase.

Although IV Elaprase has been approved in Canada and Trey is receiving the treatment (Trey was granted Special Access to use Elaprase through Health Canada’s Special Access Program before Health Canada’a approval; Health Canada Special Access Program), many other children with Hunter Syndrome in Canada have not been so fortunate. Although we are thrilled that Health Canada has approved Elaprase, this only means that is it available and approved for use in Canada.

Funding is decided on a province to province basis and many provinces are not supportive of funding expensive drugs for rare diseases. This is why Canada needs an Orphan Drug Policy so that lifesaving treatments can be granted to individuals with rare diseases as soon as they become available. For more information about the efforts to create an Orphan Drug Policy Click Here.

Meanwhile, researchers continue to work on a cure.  Their work is dependent in large part on private funding, which often takes the form of fundraising and donations. Click Here to read about the research we are funding.

Share and Enjoy:
  • Print
  • Digg
  • StumbleUpon
  • Facebook
  • Yahoo! Buzz
  • Twitter
  • Google Bookmarks

One thought on “Is There a Cure for MPS II?

  1. Do tablets of ” Soyfem ” or Genistein powder ” useful in delaying feects of MPS type 2 ( Hunter ) . Is there anything useful otherwise? Is Bone Marrow transplantation good for these types of patients ? We have a 7 years old son involved with desease . What can we do about it. Desperately waiting to hear from you.

    Nader and Nasrin Ghorbanpour

Leave a Reply

Your email address will not be published. Required fields are marked *

This site uses Akismet to reduce spam. Learn how your comment data is processed.