Marie McCullough, Inquirer Staff Writer Posted: Sunday, March 10, 2013, 5:22 AM

Still groggy from painkillers, Maddie Major, 7, clutched her stuffed Pooh Bear and laid her head on her father's shoulder as he carried her to the hospital cafeteria.

Maddie, dad Tim, mom Robyn, and big sister Candace spent that February morning at Children's Hospital of Philadelphia, where a doctor extracted samples of the child's spinal fluid and bone marrow.

In a few days, the biopsies would reveal whether Maddie's leukemia had been wiped out by an experimental gene therapy made from her own white blood cells - crucial disease fighters called T cells.

Medical science has been trying for 40 years to harness the immune system to cure cancer, or at least turn it into a docile chronic disease. Excitement has invariably been followed by disappointment.

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.

Originally posted here:

Pinning their hopes on a Penn gene therapy