Childhood Cancer - NFCR

Childhood Cancer

Childhood Cancer

There are dozens of cancers and numerous subtypes that strike children of ages 0 -14 years. Childhood cancers are often different than adult cancers as their cancers are not linked strongly to risk factors related to lifestyle or the environment. In the US, cancer is the leading cause of death by disease in children.

Key Facts

  • An estimated 11,050 new cancer cases will be diagnosed in the U.S. in 2020. One in 389 children will be diagnosed with cancer by age 15.
  • About 1,190 children under the age of 15 are expected to die from cancer this year.
  • Leukemias make up 28% of all childhood cancers; Brain and other central nervous system tumors 26%; Neuroblastoma 6%; Wilms tumor (kidney cancer) 5%; Non-Hodgkin lymphoma 5%; Hodgkin lymphoma 3%; Rhabdomyosarcoma (soft tissue) 3%; Retinoblastoma (eye cancer) 2%; Osteosarcoma (bone) 2%; and Ewing Sarcoma (bone) 1%.
  • In the 1970’s, the 5-year survival for children with cancer was 58%. Due to treatment advances, 84% of children with cancer now survive 5 years. Still, survival rates can vary greatly depending on the cancer type among other factors.
  • There is high demand for development of treatments for childhood cancer rather than using treatments developed for adults. Even those who are cured may suffer long-term side effects as a result of the cancer treatments they received. Children who were treated for cancer are twice as likely to suffer chronic health conditions later in life versus children without a history of cancer.
Source: American Cancer Society’s Cancer Facts & Figures 2020 and National Cancer Institute
Gold Childhood Cancer Ribbon
11050
expected diagnoses in 2020
1190
expected deaths of children under 15

1
leading cause of death by disease for children

Childhood Cancer Research

In addition to specific projects listed below, genomics research is helping us attack childhood cancer – and all types of cancer. NFCR has distinguished itself from other organizations by emphasizing long-term, transformative research and working to move people toward cancer genomics.

Cesare Spadoni, Ph.D.
Cesare Spadoni, Ph.D.

Dr. Cesare Spadoni is developing treatments for rhabdomyosarcoma, the most common pediatric soft tissue sarcoma and it represents 3% of childhood cancers. Rhabdomyosarcoma can occur in the head and neck, genitourinary area, trunk, and extremities, and may cause pain and/or a mass or swelling.

The aggressive subtype, alveolar rhabdomyosarcoma, results from the abnormal fusion of two genes and is difficult to treat. These young patients have a poor prognosis. Dr. Spadoni’s team is using the small molecule drug, volasertib, a highly potent inhibitor of the enzyme PLK1 – resulting in reduced activity and stability of the abnormal fused proteins. Significantly, the anti-cancer effects of volasertib are more pronounced when combined with the chemotherapy, vincristine.

Volasertib has already been studied as single agent in Phase I clinical trials in children with leukemia and refractory solid tumors. With support from the NFCR AIM-HI Translational Research Initiative, volasertib has advanced in pre-clinical research toward clinical trials. A Phase I clinical trial will begin in 2020 to bring this combined treatment to patients with relapsed or refractory rhabdomyosarcoma, with and without the gene fusion abnormalities.

Brain and other cancers of the central nervous system account for 26% of childhood cancers. Medulloblastoma is the most common type of childhood brain cancer. Dr. Cesare Spadoni’s team is focused on developing a therapy with the ‘2Hit approach’ – a compound or combination of agents that attack two or more therapeutic targets in medulloblastoma cancer cells. The 2Hit approach aims to simultaneously increase effectiveness and reduce potential drug resistance. Ongoing research has identified 3 synergistic combinations that inhibit several targets. Next steps with bioinformatics will identify a lead compound against two targets in medulloblastoma models. The team of scientists are hopeful a new treatment for this childhood cancer is on the horizon in the next two years.

Laurence J.N. Cooper, M.D., Ph.D.
Laurence J.N. Cooper, M.D., Ph.D.

T cells are powerful white blood cells in our immune system that track down and kill cancer cells. However, cancer cells can still grow and spread by tricking T cells and escaping from their immune attack. Dr. Laurence Cooper, a pediatric oncologist and scientist who received NFCR support for 11 years, is a pioneer in developing adoptive immunotherapy—a novel approach that treats cancer patients’ white blood cells in the laboratory to stimulate their immune systems and enhance their cancer-fighting capacity. Certain leukemia and lymphoma cells express molecules called CD19 on their surface. Dr. Cooper advanced the approach of CAR T cells where T cells are collected from patients’ blood and engineered to express an antenna-like molecule called CAR (chimeric antigen receptor). Since CAR readily binds to the CD19 molecules, the CAR T cells, once infused back into patients, should be able to target and mount a full-blown immune attack on CD19+ cancer cells. Dr. Cooper led the first-in-humans trial to infuse CD19-specific T-cells into patients with malignant B-cell Lymphoma. His research has helped advance the new promising immunotherapies using CAR T cells for leukemia and lymphoma patients.

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