A Childhood Cancer Patient Beats the Odds

I remember the day I met five-year-old Carley when she had come to the University of Chicago for a second opinion. Like 1,000 other US children each year, Carley had been diagnosed with neuroblastoma, an often-deadly type of childhood cancer of the peripheral nervous system. In addition to tumors in her chest and abdomen, Carley had tumor cells in her bones and bone marrow. When I met her, Carley had already undergone six cycles of intensive chemotherapy that left her tired and with a weakened immune system. She had weathered two aggressive surgeries to remove the tumors left behind after chemotherapy. Despite all of this, Carley’s scans and tests showed that she still had tumors in her bones and bone marrow. Carley’s doctors recognized that children who still possess that much disease following this therapy have a very poor prognosis, so they referred her to the University of Chicago Center for Childhood Cancer and Blood Diseases.

One of the reasons I remember meeting Carley so well, is that I had to relay to Carley’s parents how dismal her prognosis was. It is always heartbreaking to deliver such news, but to explain this to scared young parents is especially difficult. Carley’s parents were very well read about neuroblastoma, so they knew how serious her condition was. They came to Chicago to learn about other forms of therapy we were able to offer them.

The New Advances in Neuroblastoma Therapy (NANT) Consortium, which St. Baldrick’s helps to fund, was founded to provide access to new therapies, and as a participating center, we can offer these trials. Instead of recommending “standard” therapy for Carley, we offered her a form of intensive chemotherapy using the drugs buthionine sulfoximine (BSO) and melphalan. This form of therapy is being studied for neuroblastoma but is not yet a proven treatment. I remember telling Carley’s parents that these drugs can have serious and sometimes fatal effects. This therapy is so intense that children must receive stem cells afterwards to “rescue” their bone marrow that has been obliterated by the chemotherapy. With standard therapies, there was little chance of curing Carley, and I didn’t know if this experimental therapy would be any better. After hearing about the trial, Carley’s parents agreed that it was the best of the imperfect options available.

Carley did well with the therapy and her stem cell transplant. We were heartened to see that after treatment, the amount of tumor that remained was drastically reduced. Nevertheless, some still remained in her bone marrow.

We then offered to treat her with another experimental therapy using antibodies against neuroblastoma cells along with a drug to rev up her immune system. This therapy is also dangerous – causing severe muscle aches and fevers during the drug infusions. Carley endured six months of this therapy, suffering through many prolonged hospitalizations.

Following this, we had planned to enroll Carley on another trial using a radioactive tagged drug. However, we were surprised to find that Carley now showed no evidence of tumor anywhere. She was finally in remission.

That was two years ago, and we have cautiously watched Carley ever since. I’ve seen her grow into a beautiful little girl, who now can enjoy going to school and playing with her three younger siblings. We know that Carley is not out of the woods yet, but with each passing day, we are more and more hopeful.

Unfortunately, Carley’s story is not typical of children with high-risk neuroblastoma. About half of those children will die from this disease. Of those like Carley that have disease at the end of induction therapy, most will not be cured. New ways of diagnosing and treating children with neuroblastoma are sorely needed. Being able to tell at diagnosis which children have the most aggressive tumors is critical to curing these kids. The development of targeted therapies customized not only to treat neuroblastoma but also tailored to the patient will be absolutely necessary before every child can be cured.

Learn more about neuroblastoma, and how St. Baldrick’s donor funds can help.