Gene therapy gives hope to ‘boy in bubble’

Gene therapy gives hope to ‘boy in bubble’ TheStar.com – GTA – Gene therapy gives hope to ‘boy in bubble’

Parker DesLauriers and his parents return from Italy with renewed hope for the little boy with rare immune deficiency

September 28, 2007


STAFF REPORTER
When Parker DesLauriers’ mother pushed his stroller through the arrivals gate at Pearson International Airport yesterday, throngs of adoring relatives and friends crowded around the tot, whom they smiled and waved at, but could not touch.

Blowing kisses and laughing from behind a plastic shield around his stroller, the chubby-cheeked 20-month-old boy looked the picture of health despite having a seriously compromised immune system.

Parker had just returned from Italy, where he spent the last six months undergoing experimental gene therapy, which his parents say has given him a fighting chance to beat the rare condition with which he was born, dubbed “boy-in-the-bubble” disease.

“We’re definitely on our way to success,” exclaimed his mother, Tracy Kulas-DesLauriers, 32.

“I totally believe it will work out in the end. And if for some reason it doesn’t, then we’ll do whatever we have to do to make sure it works later,” she added.

The Ajax lad was born with ADA-deficient severe combined immunodeficiency. Each of his parents unknowingly carried a copy of a defective gene, which they passed to him. So rare is the disease that the Hospital for Sick Children has treated only 15 patients with it in the last two decades.

Without treatment, Parker would probably die before age 2.

The therapy he received in Milan is not available in Canada. It involved harvesting his bone marrow, extracting stem cells and genetically altering them. The cells were then re-injected into the boy.

The treatment is Parker’s best hope for long-term survival.

“It’s successful so far. It’s a long-term thing and it’s going to take a while for us to figure out how well it worked,” explained Parker’s father, Kevin DesLauriers, 35.

The gene therapy trial is being conducted by Dr. Alessandro Aiuti and the results will eventually be published in a medical journal. In the meantime, Parker’s family is not at liberty to discuss some details of his treatment.

They said Parker suffered a setback shortly after the genetically altered stem cells were re-injected and at one point it was feared the treatment had failed.

“Even though he didn’t have the greatest (initial) response, he had a lot of cells given back to him, which is definitely an advantage. He has engrafted,” his mother said.

Parker will have to visit the Hospital for Sick Children every two weeks for blood tests and next spring, he will return to Italy for additional testing.

At Terminal 1 yesterday, the family’s relatives and friends welcomed them home with balloons, flowers and toys.

“You can see it. He’s so loved. He’s never played with his cousins,” remarked his mother, noting the boy is allowed very limited contact with others because of his compromised immune system.

“He’s not out of the woods yet,” said a teary-eyed Roy Kulas, Parker’s grandfather.

Kulas scrubbed his daughter’s home from top to bottom with Lysol in preparation for Parker’s return. The boy has to live in extremely sanitary conditions with few visitors.

“It’s probably the cleanest house in the whole of Toronto. You don’t want to take a chance,” Kulas said.

Only Parker’s grandparents are allowed to pick him up.

His parents always carry a bottle of sanitizer with them and there are other bottles in every room of their home.

They got nervous during the nine-hour trip home whenever they heard someone cough or sneeze.

But they were full of praise for Air Canada, for giving them free flights.

DesLauriers and Kulas-DesLauriers have turned their lives upside down for their son.

Both teachers, Parker’s parents took leaves from their jobs – his mother two years ago and his father 18 months ago.

Kulas-DesLauriers plans to stay home with Parker, and her husband is considering a new career in the computer field.

“That’s the stress now,” she said. “It’s a financial stress.”

Italian fundraising covered the cost of the gene therapy and the family’s accommodation. But they’ve still had to make their monthly mortgage payments and cover their own expenses.

The family has a blog documenting Parker’s journey and providing information on making financial donations. Visit the blog at: www.scidada.blogspot.com

linkback url: http://www.thestar.com/article/261446

Advertisements

Comments are closed.

%d bloggers like this: