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what you say Dr. Dave?
Sep 13, 2002

Hi Doctor Wohl,

I just simply wanted to share this with you. Im a bit curious as are others, that if defective genes that made a immunosuppressed system have been cured, is it possible that such an advancement as below can have any positive effect on using similiar procedures in treating hiv?


Dale McDonald

Dutch Boy Exits Bubble After Care

By ANTHONY DEUTSCH .c The Associated Press

AMSTERDAM, Netherlands (AP) - The remnants of lunch were on Wilco Conradi's rosy cheeks when the 2-year-old grabbed a large ice cream and ran back to the table.

``Let's see if this tastes OK,'' he said, climbing onto his mother's lap and drawing a giggle.

This summertime outing at the zoo once was unthinkable for the Dutch boy, who lived his first months in a germ-proof plastic enclosure after being born with severe combined immunodeficiency, or SCID. The plastic enclosure protected Wilco from infections that would have killed him.

But the blond, blue-eyed boy underwent a new gene therapy technique that apparently cured his disease and allowed him to leave his plastic bubble. He needs no medication or special treatment and eats a regular diet.

``He can go to school, go on vacation, and live like any other child,'' said his father, Theo Conradi. ``Just look at him!''

Wilco was peering disapprovingly at a boy sleeping in a stroller, apparently worn out by the heat.

Wilco is among the first four boys to undergo the new treatment - all with successful results - for the inherited disease that occurs in about one of every 75,000 births. The disorder, which is carried by women but afflicts only boys, has plagued Wilco's family for generations, killing one of his uncles and two cousins.

The illness renders the immune system ineffective against microbes ordinarily harmless to people with normal resistance. The best-known victim was David, Houston's famous ``bubble boy,'' who lived in a germ-proof plastic enclosure until his death at age 12 in 1984.

Many afflicted babies are saved by bone marrow transplants, but for the rest of their lives take monthly intravenous infusions of immune globulin, antibodies culled from donated blood.

Wilco was a baby when he received the experimental treatment at the Hopital Necker-Enfants Malades in Paris. After receiving a single injection of genetically modified stem cells, Wilco now has a normal immune system.

In April, the experimental procedure passed its first major test when Wilco got sick.

``He had chicken pox and recovered on his own,'' said Dr. Nico Wulffraat, an immunologist at the Wilhelmina Children's Hospital in Utrecht. ``Normally, that would have been lethal.''

The boy has no signs of complications - such as fever or infection - that could indicate problems, Wulffraat said.

``Everything looks right, but we are closely following his case,'' Wulffraat said. ``Several other children have now received the same treatment.''

Similar gene therapy might be used to fight other inherited disorders, Wulffraat said, ``but these children will have to be followed for a long time'' to know the long-term results.

The day Wilco was born, his father took a blood sample to Wulffraat, fearing the boy had inherited the disease first discovered in his family 45 years earlier. The diagnosis was confirmed hours later and the next day Wilco was admitted to the hospital.

Wulffraat then introduced the family to Paris immunologist Dr. Alain Fischer.

The four boys treated in France lacked an essential protein due to a genetic mutation. As a result, they could not produce two types of infection-fighting immune cells and a third type did not work, leaving their bodies vulnerable to infection.

To reverse that gene defect, doctors drew bone marrow from the boys. They culled stem cells from the marrow and mixed them with a harmless virus that contained a gene that makes the missing protein.

After the virus infected the bone marrow cells, millions of each boy's cells were injected into his bloodstream to give them healthy immune systems.

Wilco visits the hospital four times a year, twice in Paris and twice in the Netherlands. He does not remember anything about his groundbreaking treatment or about his infancy in isolation.

At the Artis zoo in downtown Amsterdam, Wilco scampered off with his 12-year-old sister, Petra, to play on the slide as his parents explained how the treatment lifted what seemed like a family curse. Their second child, Wilma, died of a brain tumor a year before Wilco was born.

``It feels like we have been given a second chance,'' said Wilco's mother, Roelien. ``But it is still difficult to treat him like a normal child.''

09/05/02 11:48 EDT

Copyright 2002 The Associated Press. The information contained in the AP news report may not be published, broadcast, rewritten or otherwise distributed without the prior written authority of The Associated Press. All active hyperlinks have been inserted by AOL.

Response from Dr. Wohl

Thanks for the email and sharing the article.

The major difference between the illness that this child had and HIV infection is that the boy was unable to produce a competent immnue system while those with HIV can make immune cells but they keep getting destroyed by the virus. Therefore, the exact therapy for SCID would not help in treating HIV.

However, could there be other genetic manipulations performed that could render someone better able to fight HIV and preserve their immune system? This is a serious line of research that hopefully will yield benefits in teh not too too distant future.

Thanks again- DW.

protease inhibitors should they be avoided?
Two weeks into a once-a-day study

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