Boy’s best chance for life is in Milan
CHARLA JONES/TORONTO STAR
Parker DesLauriers, seen with mom Tracy, is off to Milan to take part in an experimental treatment.
Mar 12, 2007 04:30 AM
After an agonizing wait, 14-month-old Parker DesLauriers and his parents are heading off to Italy today so the infant can begin potentially life-saving, experimental gene therapy.
Parker suffers from a rare disease called ADA-deficient severe combined immunodeficiency (SCID), an illness he inherited from his parents, who both carried a copy of a defective gene.
“I started packing today and it’s crazy,” Parker’s elated mother Tracy told the Toronto Star last week. “I’ve got five suitcases. We are going from a nice-sized home to a tiny hotel room.”
The condition, also known as “boy in the bubble” disease after a famous case in the 1970s, is so rare that the Hospital for Sick Children has only treated 15 patients in the past two decades.
Parker spent most of his first five months of life at Sick Kids – isolated from others for fear he would catch an infection his body is unable to fight off. He was finally allowed to go home to Ajax last June while he is on ADA replacement therapy and his immune system is propped up.
Air Canada has donated the DesLauriers’ flights to Milan and is making every effort to ensure the family arrives safely, including whisking them through the airport check-in and security lines. “It’s perfect,” she says. “Five minutes before we get to the airport, we’ll call them and they’ll check us in and take us to the lounge.”
Air Canada will also assist when the family lands in Frankfurt,in order to catch their connecting flight to Milan. “I’ll bring my little Lysol wipes and wipe down his seat,” said Tracy, who always makes sure Parker is in the cleanest environment possible.
The disease means his body is missing the enzyme adenosine deaminase (ADA). Without this enzyme, which removes toxins from the body, toxins accumulate in the blood and organs and can lead to critical infection.
He currently gets ADA through enzyme injection therapy at Sick Kids twice a week and he’s thriving.
But few children survive more than three years on this therapy, known as PEG-ADA.
The experimental gene therapy in Milan is Parker’s best hope at long-term survival. The family will be in Milan for six months. There, Italian doctors, led by Dr. Alessandro Aiuti, will remove stem cells from his bone marrow and introduce a normal version of the ADA gene. These will be reinjected and it is hoped they will migrate to the bone marrow and begin making ADA.
Scientists believe gene therapy holds much promise for the future. A cell’s genetic material is stored on chromosomes in the nucleus on long molecules of deoxyribonucleic acid or DNA, which contain genes that dictate an organism’s characteristics.
“Parker has been doing so well, I don’t even think about this not working,” said Tracy of her son, who weighs 26.5 pounds.
After they arrive, Parker will go through one month of tests. Then he’ll be given chemotherapy and placed in isolation. After a period of time, the gene therapy treatment will begin.
There are 16 children participating in the clinical trial. Since this is a trial, the family doesn’t have to pay for the medical treatment.
But both Tracy and her husband Kevin have taken leave from their teaching jobs to be with their son full-time, so money will be tight.
The family is looking forward to a big homecoming in six months. “Our arrival home will be more fun than the departure,” she said. “My mom has been crying for two weeks.”
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