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A mother's gamble pays off: Tot with rare disorder is healing

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flashl Donating Member (1000+ posts) Send PM | Profile | Ignore Tue Jun-03-08 05:49 AM
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A mother's gamble pays off: Tot with rare disorder is healing
Two-year-old Nate Liao has spent his young life swathed in bandages from head to toe.

Nate has a rare and deadly genetic disease that prevents his skin from attaching to his body. The slightest friction against his skin, such as the rubbing of the seam from his shirt, gave him blisters the size of water balloons. Swallowing anything but baby food tore his esophagus.

...

For the first time, doctors say, they appear to have cured the disease Nate has, called epidermolysis bullosa.

"Every now and then, you really feel like you've done something great," says John Wagner, a hematologist at the University of Minnesota Medical School who oversaw Nate's therapy, a bone marrow transplant from his healthy 3-year-old brother, Julian. Doctors did the transplant to give Nate a vital protein that he was born without. That protein, collagen VII, glues the outermost layer of skin to the underlying layer, Wagner says.

The goal was for stem cells from the transplanted bone marrow to travel to Nate's skin and begin to make collagen VII once they arrive, says Angela Christiano of Columbia University, who identified the gene for Nate's disease and collaborated on his therapy.

USA Today
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dipsydoodle Donating Member (1000+ posts) Send PM | Profile | Ignore Tue Jun-03-08 06:30 AM
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1. Nice to read some really good news now and then
Hope the poor little mite now recovers fully.
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midnight Donating Member (1000+ posts) Send PM | Profile | Ignore Tue Jun-03-08 06:30 AM
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2. This is wonderful..... But what was the gamble the mother made?
Using science to heal her child in a time period where we are only allowed to worship a God/Goddess/higher power who somehow does not get the credit for this miraculous field?
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flashl Donating Member (1000+ posts) Send PM | Profile | Ignore Tue Jun-03-08 06:34 AM
Response to Reply #2
3. More from article ...
The procedure, like all bone marrow transplants, was risky. Before they delivered the healthy bone marrow, doctors first administered intensive chemotherapy to kill off Nate's immune system and prevent his body from rejecting his brother's cells. A child like Nate, who has so many open wounds, would be left especially vulnerable to infection.

But Nate was lucky, Wagner says. His brother's blood was a perfect match, which cut the risk of rejection.
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Sadie4629 Donating Member (919 posts) Send PM | Profile | Ignore Tue Jun-03-08 07:04 AM
Response to Reply #2
4. Oh, good grief
Exaggerate much?

Very, very few people--even (gasp!) the evil Fundies--oppose using scientific research/advancement to treat illnesses. And, if they choose to credit God or the Flying Spaghetti Monster with leading to a successful treatment, who cares????

I read the article much earlier this morning, before I had tanked up with enough coffee, but if I recall, the mom in the article did mention God or faith or something a couple of times.

The "gamble" involved the destruction of the immune system of a child whose illness would make him particularly susceptible to potentially lethal infections.
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roguevalley Donating Member (1000+ posts) Send PM | Profile | Ignore Tue Jun-03-08 02:31 PM
Response to Reply #2
6. this is a nice and good thing. i am happy for him and so should
everyone on this thread without the rest of the decrying. jeez, just be glad a tortured little kid is healed and maybe helping advance others to be too.
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wildflowergardener Donating Member (863 posts) Send PM | Profile | Ignore Tue Jun-03-08 09:24 AM
Response to Original message
5. glad
very glad this has helped - I also read in the article that his older brother has the disease also and is getting a bone marrow transplant from a non relative - hope that works out as well.

Meg
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