An NCI study shows that treatment with intensive chemotherapy, together with radiation therapy and autologous stem cell transplantation, lowers the risk of recurrence and improves survival in children with high-risk neuroblastoma (a cancer of the nerve tissue). Adding stem cell transplantation to neuroblastoma therapy makes it possible for patients to recover from the intense chemotherapy needed to effectively fight the disease. In this approach, doctors harvest healthy stem cells from the patient's own bone marrow before chemotherapy and then re-implant them intravenously after treatment. The infusion of stem cells helps rebuild the patient's blood-production and immune system, which are both impaired by chemotherapy.

As more and more children are cured of their cancer, researchers realize the need to better understand the long-term health effects of their cancer – and cancer treatment – as they age. In 1998, researchers from centers across the United States and Canada launch the Childhood Cancer Survivor Study, the first major study to monitor the long-term health of childhood cancer survivors. Over the coming years, their research sheds light on major long-term risks for these survivors, from increased risk of heart disease and stroke to secondary cancers later in life. These results enable physicians to monitor for these effects and provide more timely care.

Researchers identify two inherited gene mutations associated with an increased risk of developing childhood and adult cancers – one for retinoblastoma, a cancer of the eye, and another for Li-Fraumeni syndrome, a condition that predisposes children and adults to several different cancers. These discoveries make it possible to screen for the mutations in children with a family history of the diseases, creating the potential for earlier detection and more effective treatment.

The NCI establishes the Intergroup Rhabdomyosarcoma Study Group (IRSG) to investigate rhabdomyosarcoma and other forms of sarcoma – tumors of the soft tissues that support and connect the body – in patients age 21 and younger. By pooling resources, expertise and patients, the group accelerates the development and evaluation of new classification strategies, surgical approaches, and therapies.

Researchers at St. Jude Children's Research Hospital first show that adding central nervous system radiation and intrathecal therapy (chemotherapy that is injected into the fluid-filled space between the layers of tissue that cover the brain and spinal cord) to standard chemotherapy, provides prolonged remissions and the first long-term cures for acute lymphocytic leukemia (ALL). ALL is the most common form of childhood leukemia.

The Clinical Trials Cooperative Group Program is established by the U.S. National Cancer Institute. This nationwide cancer research network soon becomes instrumental in testing the safety and benefits of promising new cancer therapies. The Children's Cancer Group, a key part of the new program, is tasked with developing new treatments for childhood cancers. Over the next 55 years, research conducted through the program helps to dramatically increase childhood cancer cure rates, from less than 10 percent in the 1950s to nearly 80 percent today.