No there isn’t a cure. But there is treatment. On June 14, 2007, Health Canada approved an enzyme replacement therapy (ERT) called Elaprase, developed by Shire Human Genetic Therapies. A number of kids worldwide have been on a trial of Elaprase with remarkable results. Elaprase drastically reduces many of the effects of MPS II. It is not clear, however, how much Elaprase helps the Central Nervous System and because of this, people with MPS, regardless of ERT, often need surgery to relieve the pressure on theirs spines and may continue to have brain involvement.
Although 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: http://www.hc-sc.gc.ca/dhp-mps/acces/drugs-drogues/index_e.html), 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 on efforts to create an Orphan Drug Policy, go to: http://www.raredisorders.ca.
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. To read about the research we are funding, click here.
