NORTH VANCOUVER BOY FIRST IN CANADA TO RECEIVE BREAKTHROUGH DRUG
Vancouver, BC – On August 19th Deb Purcell’s Facebook status read “And now I wait. The longest four hours of my life.” Her status was no understatement. The North Vancouver mother of three posted from a North Carolina Hospital where her eldest son, 7-year old Trey Purcell, was under general anesthetic to determine whether or not he qualified for a ground-breaking MPS II Trial Treatment called IT Elaprase, a treatment which would halt and possibly reverse the effects of the rare, degenerative disorder with which he was born.
Trey Purcell qualified for the trial. He will be the first Canadian to receive the treatment when he begins the trial in North Carolina this fall, marking the beginning of another chapter in a young life that has been full of medical firsts.
Trey was born with MPS II, otherwise known as Hunter Syndrome. Although he appeared to be a healthy baby, a series of seemingly unrelated health issues in his first two years eventually lead to the diagnosis that he had Hunter’s, a progressive disease which would cripple his body before eventually shutting it down entirely. At the time of diagnosis in February of 2006, a life-saving treatment called IV Elaprase was on the brink of approval by the FDA in the United States. Upon FDA approval of the drug in July 2006, the Purcell’s successfully lobbied for Trey to access IV Elaprase, making him the first child in Canada to receive the treatment outside of the trial. Then in July of 2010, Deb became the first non-medical professional trained to administer the treatment at home and Trey became the first child to receive the drug in the home setting from a family member.
IV Elaprase is only effective in slowing the physical deterioration of a person with MPS II. It is not able to slow mental decline, a symptom that Trey and approximately 80% of other children with Hunter’s experience. That is the major difference between IV Elaprase and IT Elaprase. IT Elaprase, which stands for Intrathecal Elaprase, is the first MPS II treatment that is able to cross the blood brain barrier and slow, halt or prevent the progression of the disease in the brain. And so far the reported results have been incredible! It is for that reason that the Purcell’s have thrice traveled to North Carolina for the trial’s rigorous qualification process, a process which culminated in a four hour set of procedures under general anesthetic, testing the pressure in Trey’s central nervous system to ensure that it can withstand the IT treatment, which will be delivered through a port connected directly into his spine.
Trey was fortunate to not only qualify for the trial, but to be randomly assigned to receive the drug. Being assigned to the control group would have meant waiting for 6 monthes before going down for his pressure to be tested again. Instead, Trey will have his port placed in just over a month’s time and will receive his first IT infusion two weeks later. For the duration of the six-month trial and for the first three treatments post trial, Trey and Deb will travel to North Carolina for 10 days of every month. It’s a significant strain on the entire family, but one that they are only too happy to bear.
Between now and the first treatment, there’s a lot to be done, including final preparations for their Alice in Wonderland themed ‘Once Upon a Cure Gala’ to be held on September 24 at The Sutton Place Hotel. Special Guests will include actors from a number of local shows, including: Once Upon a Time, Sanctuary, Smallville, Stargate: Universe, Stargate: Atlantis, Stargate: SG-1, Riese, Fringe, Supernatural and Eureka. All proceeds will go to the MPS II Research fund. Tickets & information at: www.onceuponacure.com
The MPS II research fund, administered through the MPS Society of Canada, provides research grants to doctors working towards finding a cure for MPS II.
If you would like to know more about this topic, or to schedule an interview with the Purcell family, please email Deb Purcell at firstname.lastname@example.org. For more information about Trey and Hunter Syndrome, please visit: www.treypurcell.com.