Report makes key recommendations on childhood cancer

Date: 10/14/2016

SPRINGFIELD – Earlier this year President Barack Obama announced the establishment of a Cancer Moonshot initiative to accelerate progress toward ending cancer as we know it. September was National Childhood Cancer Awareness Month, and on Sept. 7 the National Cancer Moonshot Blue Ribbon Panel presented a report encompassing 10 key recommendations – two relating directly to children.

One of these recommendations is to intensify research on the major drivers of childhood cancers with a focus on fusion oncoproteins – proteins that are the result of genetic abnormalities where two genes, which were never meant to come together, combine to form a new gene. The new gene can then produce a protein that drives a cell to develop into cancer.

The other recommendation is to create a clinical trials network, with easier access especially for children, devoted exclusively to immunotherapy. In this approach, antibodies, which naturally fight infection, are developed to recognize and fight cancer cells.

“Both of these initiatives are very exciting and worthy of intense support,” said  Dr. Matthew Richardson, a pediatric oncologist at Baystate’s D’Amour Center for Cancer Care.  

Richardson noted that, in the past, chemotherapy has indiscriminately attacked any growing cell whether cancer or healthy, leading to many of the side effects that accompany chemotherapy – hair loss, mouth sores, and low blood counts with risks of infection or the need for blood transfusions.

“Targeting an oncoprotein made by a unique gene combination would allow doctors to develop medicines that attack only the cancer cells that have the oncoprotein.  Healthy cells would then, theoretically, be left alone,” said the pediatric oncologist.

He noted as a perfect example – imatinib mesylate – a medicine that targets an oncoprotein that is made when part of chromosome #9 combines with chromosome #22, which is seen in chronic myeloid leuekmia (CML). That medicine, developed in the late ’90s, is now the standard of care in children and adults with CML.

“Similarly, if one can develop antibodies that specifically target cancer cells, then one could use the body’s own immune system to destroy the cancer and leave healthy cells alone. Dinutuximab is an antibody that attacks neuroblastoma, a type of childhood tumor that historically has been difficult to cure. The antibody was developed and tested through the Children’s Oncology Group (COG) and found to increase significantly cure rates of some types of neuroblastoma,” said Dr. Richardson.

Dinutuximab is now FDA approved for children and is standard treatment. Baystate Children’s Hospital is a member of COG – dedicated to improving the outcome for all children with cancer through global research – and is anticipating using dinutuximab for some of its patients in the near future.

In the United States in 2016, an estimated 10,380 new cases of cancer will be diagnosed among children from birth to 14 years old, and about 1,250 children are expected to die from the disease. Although pediatric cancer death rates have declined by nearly 70 percent over the past four decades, cancer remains the leading cause of  non-accidental death among children. The major types of cancer in children ages 0 to 14 years are acute lymphocytic leukemia (ALL), brain and other central nervous system (CNS) tumors, and neuroblastoma, which are expected to account for more than half of new cases in 2016.

A cancer diagnosis is upsetting at any age, but especially for children and their families. The good news for these young cancer patients and their families, is that many types of cancer that were once fatal, now have cure rates exceeding 80 percent – surpassing that of adult cancers – thanks to research and the development of new therapies.

Baystate Children’s Hospital sees families from all over western Massachusetts for treatment of benign hematology blood disorders, such as sickle cell anemia and thalassemia, to more serious and intense childhood cancers ranging from leukemia to brain cancer.

Most treatment of childhood cancers today involves standardized protocols – receiving the same treatment as another child with the same cancer elsewhere – often involving a combination of surgery, radiation, and chemotherapy for which there are new and better drugs, and methods to help children deal with the side effects of treatment.