Survival rates improve for children with cancer – Pittsburgh Post-Gazette

First of two parts.

A diagnosis of cancer in childhood means something very different today than in the past. More intense and effective treatments are now increasing cure rates for many types of cancer, together with treatments tailored to be less troublesome to young patients.

In its annual report on cancer statistics, released Thursday, the American Cancer Society said cancer is still the second most common cause of death among children ages 1 to 14 in the country after accidents. It estimates that this year 10,380 children will be diagnosed with cancer, and 1,250 will die from it.

Survival rates continued their upward climb: For all cancers, survival at five years went from 58 percent for children diagnosed between 1975-77 to 83 percent for those diagnosed from 2005-11.

Many people don’t know that the cure rates are so high and that research is moving forward in both common and rare cancers, said pediatric cancer specialist Jean M. Tersak at Children’s Hospital of Pittsburgh of UPMC.

“There are advances [toward cures]. The majority will go on to be long-term cancer survivors,” she said.

Leukemia most common

According to the cancer society, leukemia accounts for 30 percent of the childhood cancers, with brain and other nervous system tumors at 26 percent. Three out of four leukemias among children and teens are acute lymphoblastic leukemia, known as ALL. The survival rate for the years 2005-11 was 91 percent. Most of the remaining cases are acute myeloid leukemia, or AML.

“AML requires more aggressive therapy,” Dr. Tersak said. “It has a lower cure rate than ALL — but there are advances there as well.” The cancer society reported an upward trend in survival rates for AML patients, reaching 67 percent in 2005-11.

“Individually, cancer is so rare. That’s why it takes so long to advance,” Dr. Tersak explained. That is why Children’s and other children’s research hospitals are part of the Children’s Oncology Group, coordinating research and treatment guidelines for all children’s cancers. Quality clinical research trials are done, with patient groups randomized and treatment changes compared to standard treatment.

“The goal is we need hundreds of children [to get good results],” Dr. Tersak said. There are two kinds of trials, she explained: those that intensify therapy to bring about a cure, and those that pull back on therapy (with lower doses, for example), to reduce health problems that show up months or years after treatment has ended.

Gabe’s story

Gabriel Aguirre, 6, of Kittanning, is being treated for ALL, and he’s part of a clinical trial testing lighter doses of medication in “favorable risk” patients. Before Christmas, Gabe visited Dr. Tersak at Children’s with his grandmother, and he was a bundle of energy.

Now in kindergarten, Gabe first came to Children’s at the end of October 2014 complaining that his whole body hurt. After about three weeks of inpatient treatment, he’s now on daily oral chemotherapy at home. He also comes to the hospital clinic at specific intervals for re-evaluation and IV chemotherapy.

He’s back to running fast — “100 miles an hour,” he said. And he told Dr. Tersak he doesn’t have any aches or pains now.

“What happened to your front teeth?” Dr. Tersak asked. As they discussed the tooth fairy, the doctor examined Gabe, telling him to “blow out your breath like you’re blowing out your birthday candles.”

After praising the boy for learning to swallow his pills — “That was a really big accomplishment” — Dr. Tersak concluded the exam with “I would have to say you pass with flying colors!”

New paths of treatment

Advances since the 1960s have been credited to the toxic chemotherapy agents that kill malignant cells. But together with surgery and radiation, they can cause the health problems known as “late effects.” They’re a growing concern because more patients are living longer.

More than 90 percent of those who had childhood cancer have a chronic health condition as adults and have a higher risk for severe problems, such as another cancer, organ damage and thinking/learning disabilities.

Two new developments that promise higher cure rates and lower toxicity are targeted therapy and immunotherapy. In the first, analyzing the genetic characteristics of a tumor can help identify specific drugs to target it.

In the second, modifying a patient’s immune system’s T-cells so that it can recognize and reject tumor cells is being tested in both adults and children.

Children’s Hospital is one of 27 pediatric institutions nationally in which these novel therapies are being tested. The phase 1 program evaluates new therapies for children who have had their cancer return following more standard treatments. Dr. Tersak is the principal investigator at Children’s.

These immunotherapy drugs include monoclonal antibodies (mabs, for short), manmade versions of immune system proteins that can be designed to target certain antigens like ones found on cancer cells. A number of these compounds are being evaluated in the phase 1 program at Children’s.

“The drugs we’ve used for decades attack rapidly dividing cells [which children have a lot of],” Dr. Tersak said. “Mabs target specific cancer cells.”

Cancer vaccines are another type of immunotherapy. As leukemia treatments achieve success, brain cancer has become the leading cause of cancer death in children and teens, according to the cancer society.

Children’s Hospital is conducting a vaccine study for life-threatening brain tumors known as gliomas and ependymomas, Dr. Tersak said. “The option is not available anywhere else.”

Other clinical trials investigate treatments for neuroblastoma, another type of solid tumor in young patients, which accounts for 6 percent of all children’s cancers.

“High risk neuroblastoma has been devastating in the last few years,” said Dr. Tersak, who called it a “very aggressive disease.” “Now there is an antibody with an extended treatment time. Now it has an improved cure rate: by 20 percent. Children live three years from diagnosis — the new cure rate is over 80 percent. … More patients with stage IV high risk neuroblastoma are long-term survivors.

“In utero, there’s an excellent chance of a cure, more than 90 percent. Neuroblastoma in children under 18 months of age have an excellent prognosis.”

Mason’s story

The new medication crizotinib is being used at Children’s as an experimental treatment for 4-year-old Mason Gaetano, whose rare cancer returned about 1½ years after his first treatment.

Playing peekaboo behind his grandfather, Anthony Gaetano, at the beginning of his December appointment, Mason’s energy level is noticeably higher than during his last visit, Dr. Tersak noted. Mason, of Monroeville, was first diagnosed with an extremely rare inflammatory myofibroblastic tumor in December 2013.

The tumor was found around the heart, and the boy had open heart surgery to remove it, followed by six rounds of chemotherapy. The cancer was gone until this past June when something showed up on a scan. Since then, because the cancer returned after more standard treatments, Mason has been on a new chemotherapy medication, crizotinib, which targets cancer cells specifically. 

Supportive care

Dr. Tersak said there is a special effort to work with parents at Children’s: “We help them understand the seriousness of the disease. Cancer is life-threatening.”

“Supportive cancer care has improved … It can make a difference in their quality of life,” she said.

As far as cancer treatment, she said, “We won’t be satisfied until the cure rate is 100 percent.”

NEXT TUESDAY: A focus on survivorship for patients cured of childhood cancer.

 Jill Daly:, 412-263-1596.