New hope for Ajax 'bubble boy'
RICK EGLINTON/TORONTO STAR
Two-year-old Parker DesLauriers loves to play with his grandfather
Roy Kulas, one of the few people allowed to have physical contact
with him.
Cutting-edge therapy done in Milan appears to be boosting child's
immune system
Mar 03, 2008 04:30 AM
Joseph Hall
Health Reporter
Tracy Kulas-DesLauriers holds her son firmly by the shoulders and
pleads with him, with exasperated bemusement, to please stop spinning.
"It will make you dizzy, Parker. It will make you fall down!"
But when she lets go, he's a human top again; twirling, twirling,
running toddler circles around his kneeling mother and around all
that medical expertise that said he likely wouldn't do what he has so
obviously and gleefully done.
Parker DesLauriers has ADA-deficient severe combined
immunodeficiency – a form of "bubble boy" disease.
With the frenzied energy of a 2-year-old bent on being naughty, he
has every appearance of brimming health.
But his immune system remains massively compromised and Parker, who
celebrated his second birthday in January, is a virtual prisoner in
his antiseptic Ajax home.
"We've been confined, basically for two years and it's taxing. I mean
it gets to you after a while," his mother says.
Basically, the only time he leaves the house is for twice-monthly
visits to the Hospital for Sick Children, she says.
The disease, a rare genetic ailment caused by one malfunctioning gene
on chromosome 20, leaves him with almost no working T and B blood
cells, which are essential to a body's immune system. Without these
white blood cell components, Parker is almost entirely unable to fend
off any harmful virus or bacteria he encounters.
But there's hope in his mother's voice now, and a rising confidence
that one day, maybe soon, her little boy will enjoy a normal
childhood.
Because, deep in Parker's bone marrow, a seeding of genetically
modified stem cells may be sprouting a new immune system.
"We have reason to believe that it's definitely working," Kulas-
DesLauriers says of the cutting-edge gene therapy her son received in
Italy.
The process, which took place in Milan last May, involved harvesting
Parker's bone marrow, from which doctors removed a number of the stem
cells that can produce every type of blood cell, including the
disease-fighting cells Parker lacks.
Then, using a virus as a transporting vector, they inserted a healthy
gene into the stem cells to counteract its defective counterpart.
That gene produced an enzyme known as ADA, which is critical in
protecting T cells from rapid destruction in the body. Without T
cells, antibody-producing B cells do not perform properly either,
leaving the entire immune system in shambles.
The genetically altered stem cells were reintroduced into Parker's
marrow. Now, some nine months later, his T and B blood cells have
substantially improved.
When he was born, Parker had a lymphocyte count of 80, his mother
says. Now it fluctuates between 700 and 900, she says. A normal count
would be about 2,000, doctors say.
"The main thing now is the fact that he is producing these cells,
something he was never able to do on his own before," she says.
Parker will return to Italy this May for a one-year follow-up.
Meanwhile, the quarantine of the outside world from the family's Ajax
home must be maintained.
"He can't play with his little cousins or anything ... little kids
are bacteria carriers," says grandfather Roy Kulas, who, with his
wife, Parker's fraternal grandparents and the boy's parents, are the
only people allowed to pick the toddler up.
"Still, he's a good happy lad, and a joy to be around," Kulas says.
Financially, the disease has been devastating for his parents, who
both had to quit their jobs as teachers – with their professional
obligation to be around kids and germs – after Parker was born.
They've been living off savings, fundraising events and support from
family and friends. "I had a nice house, I had a great job, I had
everything going for me and now all of a sudden, I'm in this position
where I need to ask for help," Parker's mother says. "I'm going to
lose this house. I'm going to lose everything that we've worked so
hard to get."
The family will be holding a fundraising golf tournament June 1 at
the 4 Seasons Golf Club in Claremont. Anyone wishing to buy tickets
or donate can email golfingforparker@
http://www.thestar.
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StemCells subscribers may also be interested in these sites:
Children's Neurobiological Solutions
http://www.CNSfoundation.org/
Cord Blood Registry
http://www.CordBlood.com/at.cgi?a=150123
The CNS Healing Group
http://groups.yahoo.com/group/CNS_Healing
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