How CRI-Funded Scientists are Reducing and Guarding Against Childhood Cancers 

A cancer diagnosis is always extremely difficult news for a patient and their family. It can be even more challenging for a vulnerable subset of the population: children, adolescents, and their families.

Childhood cancer has an outsized impact on a global scale. Worldwide, youths under the age of 20 receive a cancer diagnosis every other minute, directly impacting approximately 300,000 patients annually. For younger children under the age of 15, the four most common cancers are acute lymphocytic leukemia, brain cancer, neuroblastoma, and non-Hodgkin lymphoma. For adolescents between the ages of 15 and 19, the most common cancers are brain cancer, Hodgkin lymphoma, thyroid carcinoma, and testicular germ cell tumors.

Within the U.S., cancer is the deadliest disease for young people under 20 years of age. Approximately one in every 285 people in the U.S. will receive a cancer diagnosis before age 20. Unfortunately, the average age for youths with a cancer diagnosis is six years old.

CRI is proud to have helped pave inroads in immunotherapy treatment for childhood cancer patients. Today, nearly 80% of childhood cancer patients survive long-term past their initial diagnosis – a dramatically improved outcome from past decades. Select individual CRI-funded scientists have been instrumental in improving outcomes for childhood cancer patients.

“CRI has funded a multi-institutional, national study of checkpoint inhibitor therapy in pediatric patients with relapsed or refractory hepatocellular carcinoma (HCC) or HCC-like tumors.  We are close to completing accrual on the first arm of the study, have not met any safety stopping rules, and have detected enough of a signal of efficacy to continue to enroll,” says CRI CLIP Investigator Allison O’Neill, MD (Dana-Farber Cancer Institute). “The trial will allow us to collect serial assessments of immune function which will give us a correlative understanding of why some patients respond to treatment. Support from CRI has been invaluable for this ongoing initiative.”

Fortunately, advances in immunotherapy have produced five FDA-approved treatments for childhood cancers. They are effective in treating lymphoblastic leukemias, lymphomas, neuroblastoma, and advanced melanoma. There are also multiple immunotherapy treatments undergoing clinical trials that could prove useful in treating various forms of childhood cancer.

“Many of these cancers are curable, and we present it that way to families when appropriate. But every family wants to know: why did my child get this? Is it from something I did, or from something they did, or from where I live,” says CRI Lloyd J. Old STAR Robbie Majzner, MD (Dana-Farber Cancer Institute). “I explained that in almost every circumstance, pediatric cancer is a genetic accident, no different than the genetic accident of where you’re born, or what country you’re born in. Through research, we’ve been able to start learning how to tackle these diseases.”

Thanks to the scientific contributions of Drs. O’Neill and Majzner, and several other current and former CRI-funded scientists, outcomes for childhood cancer patients are greatly improving. With their continued scientific rigor and your ongoing support, CRI hopes to eliminate childhood cancers as we strive to create a world immune to cancer.