When John and Pam Witt got the devastating news that their son Zach’s lymphoma had relapsed, they felt helpless and scared. But doctors at The Children’s Hospital of Philadelphia were optimistic about an experimental treatment that they believed could save the 5-year-old’s life.
It was September 2010 when John and Pam Witt noticed lumps on their son Zach’s neck. Worried about the swelling, the Witts took Zach to his pediatrician, who recommended that the family go to a CHOP Specialty Care Center for testing. The family was shocked to learn that Zach had anaplastic large cell lymphoma (ALCL). He was only 5 years old.
Chemotherapy began right away, the conventional approach for children with ALCL. Zach was on treatment through March 2011, and his cancer seemed to be responding well. But just as his chemotherapy was ending, the Witts noticed cold-like symptoms that quickly developed into frequent fevers. When it was clear that this was more than a cold, he was brought back to CHOP. After a 10-day hospital stay and multiple tests, doctors confirmed John and Pam’s worst fear — Zach’s lymphoma had relapsed.
“It’s an overwhelming and helpless feeling,” says John. “His cancer came back while he was on treatment, so now what? That’s pretty scary. And it was frightening how quickly everything was happening.” The Witts had a meeting with their doctors to discuss the next step in Zach’s treatment. This was when they learned that their son had a genetic marker that made him a candidate for a Children’s Oncology Group phase 1 clinical trial of a drug called crizotinib. The Children’s Oncology Group is a research consortium that unites scientists from many different hospitals. The crizotinib study was led by Yael Mossé, MD, pediatric oncologist and researcher at The Children’s Hospital of Philadelphia.
Richard Aplenc, MD, is Zach’s oncologist at CHOP. “Zach's lymphoma is characterized by changes in the ALK gene,” says Aplenc. “It's called an ALK-positive lymphoma or anaplastic large cell lymphoma. The changes in the ALK gene make the lymphoma cells grow out of control. This medication actually targets that change and turns off that driving signal.”
ALK has also recently been found to be important in a form of non-small-cell lung cancer and in a subset of neuroblastomas. The ALK-inhibiting drug crizotinib had already been developed to target the gene directly. For this reason, study leader Mossé and her colleagues were able to quickly move crizotinib into an initial pediatric trial.
The idea of an experimental treatment worried the Witts. “We were considering more severe standard chemotherapy,” says John. “But we had the meeting with the doctors and I really sensed some excitement as they were explaining what they hoped the drug would do. Pam asked one of them, ‘If this was your child, what would you do?’ He didn't even hesitate. He said ‘I would go on the study.’ That was very reassuring.”
Zach was enrolled in the phase 1 trial and began treatment in mid-April 2011. Doctors told the Witts that if the drug worked, they would likely see a quick response. In fact, less than 48 hours had passed when they saw a dramatic change in their son.
Pam recalled how weak Zach had been after his relapse. “He'd wake up in the morning and say ‘Mom, I want to go to the playroom.’ But he just didn't have any energy to get there,” she says. “The second day that he was on the drug, we decided to take a walk down to the playroom. He literally ran down the hallway. I mean, you just couldn't believe it was the same kid that was lying in bed all day. It was just a miracle.”
The new treatment gave Zach more than an energy boost. It gave his family hope that their son will live a long, healthy life. In Zach’s first year on the drug, scans have shown no evidence of lymphoma. He’s living the normal, happy, high-energy life of a 6-year-old and experiencing no side effects from his treatment, which he’ll continue for the foreseeable future.
“Zach's case is an outstanding example of the kind of therapy that we would like to have for every child with cancer,” says Aplenc. “We want to get to the point where we understand why the leukemia or the lymphoma happened, and then give medicines that specifically target what went wrong without affecting the normal cells. It's been so satisfying to play a small role in making a difference in Zach’s life. He's been able to go back to school and run around and just be a kid. That's been very wonderful to watch. We’re working very hard to be able to do this for all children with cancer.”
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