Press Release

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FOR IMMEDIATE RELEASE

 
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 purcelldeb@gmail.com.

For more information about Trey and Hunter Syndrome, please visit: www.treypurcell.com.

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HOME INFUSION BREAKTHROUGH FOR RARE DISEASE

For three and half years the Purcell family spent every Monday at BC Children’s hospital, where their son Trey received Enzyme Replacement Therapy (ERT) infusions to treat MPS II (Hunter Syndrome), a rare degenerative disorder with which he was diagnosed at age two. No more! After six months of training, mom Deb Purcell is now the first ever parent in Canada qualified to do the infusions and has begun treating her son at home. This means their family has regained that day each week for living.

There were a number of hurdles that prevented the practice of home infusions in BC until now – the most challenging being a personnel issue. The BC health care system allows for nurses to make home visits to begin infusions and to de- access, but they aren’t able to stay for the duration of a long infusion like Trey’s, which lasts 4 hours. With the use of ERT in MPS II treatment, there is always a chance of an adverse reaction, so a physician or experienced health care provider should supervise. With the medication in place, the home supplies provided by the government’s “At Home Program” and the family more than willing, the manufacturer of the ERT hired Bayshore Home Health to train and supervise Deb in the complicated procedure, and continue to retain the home nurses as a reference, resource and emergency contact for Deb and family.

“Home infusions have given us our life back. Instead of having to cancel Trey’s baseball opening ceremony, a baseball game and a birthday party this past weekend, we were able to reschedule Trey’s infusion for a time when nothing was planned. We can now travel for more than 8 days at a time. Given the progressive nature of this disease, these extra moments mean everything to us.” – Deb Purcell

It’s fitting that Trey’s family is part of this medical advancement in Canada. When Deb learned in 2006 that Trey had a crippling disorder, she successfully lobbied the government & BC Children’s Hospital for Trey to be the first child in Canada, who was not participating in the trial, to receive ERT, which was newly approved in the US, but not yet approved by Health Canada. Remarkable results from this treatment have since been observed in Trey – many of his symptoms have been reversed and the progressions of other symptoms have been halted.

Health Canada has now approved the ERT and most provinces have made it available to Hunter patients, but ERT is a treatment – not a cure. So, the Purcell family has created a number of initiatives to raise the funds necessary to continue the research needed towards finding a cure to MPS II. To date these fundraisers have brought in $184,000 with all but 5% (used to cover basic admin. expenses) going directly to research grants applied for by doctors in the field of MPS II Research around the world.

With hopes to increase the yearly number of grants made available, this year’s fundraiser, ‘Once Upon a Cure, a Gala for Hunter Syndrome Research’, will be held at The Sutton Place Hotel, Vancouver, on September 24, 2011. Having ties to the film and television industry, they have attracted the attention of a number of Vancouver actors and VIP’s, who will be in attendance to experience the magical Alice in Wonderland themed cocktail reception, plated dinner, live & silent auctions, infamous Chocoholic dessert buffet, live entertainment and local celebrity DJ, to dance the night away. Not only will this event bring in the funding necessary to continue researching a cure, but it’s sure to become an annual event people will look forward to marking on their calendar. If you would like further details about this event or MPS II Please visit us at: www.onceuponacure.com.

If you would like to know more about this topic, or to schedule an interview with the Purcell family, please call Deb Purcell at (604) 980-0930 or email at dpurcell@telus.net.

Contact Info:
Jennifer Lo, Media Director

media@onceuponacure.com

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