New treatment guidelines for children with a once-fatal type of leukemia

Fifty years ago, the most common pediatric cancer was invariably fatal. Now, 90 percent of children diagnosed with the disease, called acute lymphoblastic leukemia or ALL, are cured.

“This is among the most profound medical success stories in history,” said Patrick Brown, a children’s oncologist who directs Johns Hopkins University’s pediatric leukemia program.

But that phenomenal cure rate means most children with the blood cancer are no longer being treated in studies aimed at finding the best approaches. In response, Brown and other experts developed the first pediatric ALL treatment guidelines, released Thursday by the National Comprehensive Cancer Network (NCCN).

ALL progresses rapidly and, left untreated, can be fatal within months as immature white blood cells, called lymphocytes, grow out of control.

The intensive, complex treatment typically consists of about three years of multidrug chemotherapy divided into four parts: inducing remission; wiping out undetectable disease; preventing relapse with maintenance therapy; and eradicating disease hiding in the central nervous system. About 15 percent of children relapse despite this grueling regimen and typically undergo a stem-cell transplant. The guidelines also address the use of innovative treatments, including molecularly targeted cancer drugs, and Kymriah, the revolutionary T-cell therapy pioneered at the University of Pennsylvania and Children’s Hospital of Philadelphia and approved in 2017.

Emily Whitehead of Philipsburg became the world’s first child to receive the T-cell therapy when she was treated at CHOP in 2012. Now 14, she marked seven years cancer-free this month.

The guidelines also specify supportive care to minimize or prevent the toxic side effects of chemotherapy, which are fatal in a small percentage of cases.

The guidelines, which will be used internationally, include recommendations for treatment in countries with limited resources. “We know many, many children can be cured with inexpensive and widely available therapies,” Brown said.

In the U.S., ALL is diagnosed in about 3,000 people under age 20. ALL accounts for about a third of all cancers in that age group.

NCCN, a Plymouth Meeting-based nonprofit alliance of 28 leading U.S. cancer centers, develops cancer guidelines for clinicians and patients.