Pinning their hopes on a Penn gene therapy

Posted: March 11, 2013 at 10:53 am

But after 30 months of testing in more than a dozen adults and children - patients with no conventional options left - worldwide excitement over the T cell therapy's unprecedented power continues to build. The treatment, developed at the University of Pennsylvania, has eradicated advanced blood cancers in mere weeks, and is being adapted to attack solid tumors including prostate, pancreatic, ovarian, and breast cancer.

Maddie's parents, who live in La Plata, Md., marveled that the unique therapy was a cakewalk compared with what she has been through since her diagnosis at age 3.

She has had thousands of doses of toxic chemotherapy. Head-to-toe radiation. Hundreds of blood transfusions. Life-threatening infections in her kidneys, liver, and brain. Months on life support in intensive care. An experimental cell therapy at the National Institutes of Health.

And still, "the beast," as her parents call Maddie's acute lymphoblastic leukemia, would not stay away.

During lunch in the cafeteria, Robyn Major said she was optimistic about the T cell therapy. She did not elaborate because Maddie - perked up and chowing down on pizza, spaghetti, and Fritos - knows more than a 7-year-old should about the limits of modern medicine.

"We've been to Children's National Medical Center in D.C., the NIH, and now here," Maddie said. "This is definitely the best."

She meant the food.

Acute lymphoblastic leukemia was a death sentence in the 1960s. Today, with potent chemotherapies and aggressive treatment of the cerebrospinal fluid, about 80 percent of the 3,000 children diagnosed annually in the United States are cured.

That still leaves many other kids like Madison Grace Major.

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Pinning their hopes on a Penn gene therapy

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