Stories about: Dana-Farber/Boston Children’s Cancer and Blood Disorders Center

Gene therapy trial offers hope for Harry

Boy with Wiskott-Aldrich syndrome holds father's hand.
Harry and his father at Dana/Farber-Boston Children’s

In their Brookline home-away-from-home, 2-year-old Duy Anh “Harry” Le plays with blocks and pop-up toys on the floor with his mother, Thao Nguyen. He is lively and happy, and his skin is clear. He looks almost nothing like the sickly baby covered in eczema who arrived in Boston from his native Vietnam in November of 2016 to participate in a gene therapy clinical trial for Wiskott-Aldrich syndrome.

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Pulmonary vein stenosis: A clinical trial in Jack’s juice glass

Jack, who has pulmonary vein stenosis, is pictured sitting on the back deck at this family homeAt just 6 months old, Jack Marquis was suddenly given four weeks to live. After he was born with complex congenital heart defects, Jack’s doctors in California had performed two open-heart surgeries that they thought would save Jack’s life.

But just when they thought he was out of the woods, Jack’s condition suddenly began to deteriorate rapidly.

“On top of everything else, we learned he had a rare condition called pulmonary vein stenosis,” says Jack’s father, Andrew.

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Two life-threatening conditions.
One remarkable wish.

Lucas, who received a 5-organ (multivisceral) transplant looks over his mom's shoulderWhen Lucas St. Onge blew out the five candles on his birthday cake, he made just one wish. He didn’t ask for ninja turtles, a T-ball set or an Xbox — the only thing he wished was to be just like any other kid.

“It was a gift I couldn’t give,” says his mom, Heather.

On that same day, last May, he got his wish.

“He got a liver, stomach, pancreas, intestine and spleen — the five organs he needed to help him become healthy and happy,” Heather says.

Heather was 18 weeks pregnant when she and her husband, Anthony, learned they were about to face a myriad of medical challenges with their unborn child. To what extent remained unclear, until the day Lucas was born.

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Blood relatives: Family bound by love and rare blood disorder

Tracy Antonelli was 4 when she was diagnosed with thalassemia, a rare blood disorder that occurs often enough in Mediterranean countries like Italy that an old adage, uttered only partially in jest, warns Italian-Americans against marrying other Italian-Americans.

In 2002, Tracy wed Patrick Mooty, whose background is mostly Irish. Their three daughters — 7-year-old Emmilene, 6-year-old Rosalie and 3-year-old Francesca — all have thalassemia, but not through the accident of the couple’s genetics. Tracy and Patrick adopted the girls from China, specifically because they, too, have the potentially life-threatening disease, which, according to conservative estimates, occurs in about 10,000 pregnancies a year in China and about 600 a year in the United States.

“This is the most rewarding, perfect experience I’ve had in my life,” says Tracy.

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