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
expected diagnoses in 2020
expected deaths of children under 15

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.

Related Content

Pediatric Cancer Facts and the Need for Improved Treatment

September is childhood (or pediatric) cancer awareness month. While all cancers are painful to both the patient, and their loved ones, perhaps no cancer has a more devastating impact on the patient, or his/her family unit, than pediatric cancer.   Cancer is the leading cause of death among children under the age of 14. Sadly, over 11,000 children in the United States are expected to be diagnosed with cancer and 1,190 children under the age of 15 are expected to die of cancer this year.   The most common forms of childhood cancers are blood and neurological cancers. Blood cancers account for over a third of all pediatric cancers, with Leukemias accounting for 28% of childhood cancer, Non-Hodgkin lymphoma 5% and Hodgkin lymphoma 3%. Brain and Neuroblastomas account for 34%, collectively, of pediatric cancers.  Other common forms of childhood cancers include Wilms tumor (kidney cancer) 5%, Rhabdomyosarcoma (soft tissue) 3%, Retinoblastoma (eye cancer) 2%, Osteosarcoma (bone) 2%, and Ewing Sarcoma (bone) 1%. Pediatric cancers present unique challenges, psychologically, logistically, and for treatment. According to Oncoheroes Biosciences, a pediatric cancer therapeutics development company funded by the NFCR-co-founded AIM-HI Accelerator Fund, childhood cancers tend to be more disruptive to the family unit and can affect the patient’s physical and emotional health, even decades after remission. The average family spends more than 40 hours per week caring for childhood cancer patients, parents are often forced to take leaves from work (creating financial strain), both the patient and his/her siblings have increased risk of psychiatric issues, and the patient is at a greater risk for neurocognitive development issues. Additionally, 95% of pediatric cancer patients will have significant health issues by the age of 45. Pediatric cancer cases are significantly different from adult cancers and present both unique obstacles and opportunities for treatment. Yet, for the most part, children are treated in many cases using similar therapies to adults – chemo, radiation, surgery, and certain forms of immunotherapies.   Pediatric cancers tend to have far fewer mutations, and as a result, targeted therapies – drugs that target specific proteins on the cancer caused by these mutations – tend to be less effective in children than adults. Many of the same chemotherapies used to treat adults are also used in children.  However, since chemotherapy tends to target more rapidly replicating cells, and since a child’s whole body is growing with cells replicating more quickly than in adults, chemo can cause long-term developmental effects not seen in adults. Similarly, children tend to be more sensitive to radiation than adults, and also, may be differentially affected by surgery (extractions) as their bodies are still growing.   While pediatric treatment approaches have been limited, improvements in chemos and newly approved immunotherapies have had a dramatic effect on childhood cancer survival rates.   In the 1970s, the 5-year survival rate for pediatric cancer was 58%. Now, the 5-year survival rate stands at 84%.   Dr. Cesare Spadoni and Dr. Laurence J.N. Cooper, two NFCR-funded scientists, have made significant advancements in alternative, new approaches to treating pediatric cancer. Dr. Spadoni is focused on developing the so-called “2Hit approach” drugs – compounds or combinations of compounds that attack more than one […]

Ricardo Garcia: The Power of Repurposing

When volasertib was discontinued after an unsatisfactory Phase III clinical trial as an anti-leukemia drug, it did not die a quick death, or die at all. Like Viagra and thalidomide before it, volasertib was “repurposed;” if the original vision for the compound fails, it is simply a matter of finding another vision. Indeed, in the compound found a new life via Ricardo Garcia and his company, Oncoheroes Biosciences, as a treatment for alveolar rhabdomyosarcoma, an aggressive cancer commonly occurring in teens or young adults. “Our strategy is to bring new drugs into the market as soon as possible,” Garcia continues. “For this reason and in parallel to our discovery lab, Oncoheroes is scouting for compounds from biopharma companies and academic research centers that hold real potential to cure several types of childhood cancer.” The drug’s path to clinical development, however circuitous, is thanks in large part to continued investment from the NFCR through AIM-HI Accelerator Fund. Volasertib, an inhibitor of Polo-like-kinase 1 (an enzyme well-known to oncologists to be involved in the progression in a number of diseases including cancer), is the first asset that Oncoheroes has in-licensed from original developer Boehringer Ingelheim; Oncoheroes now owns volasertib worldwide and is planning to initiate the clinical development plan for alveolar rhabdomyosarcoma.  In fact, Garcia and AIM-HI are a duo going back to 2018. “The NFCR’s/AIM-Hi support helped Oncoheroes not only advance our science and strategy plans, but also to convince others to invest in our project,” he says. “We were able to launch the 2HIT project, project focused on developing new treatments for medulloblastoma patients, by establishing a drug discovery lab in Barcelona thanks to initial support received from the NFCR.” It was through that lab that Oncoheroes’ initiated its first discovery project with the aim to develop a new biomarker-related treatment for medulloblastoma, the most common brain cancer in children, with greater therapeutic effect and lower toxicity.  Garcia, whose son was diagnosed with medulloblastoma when he was six years old, expects to have a preclinical candidate for medulloblastoma by early 2021. “Our strategy is to bring new drugs into the market as soon as possible,” Garcia explains. “For this reason and in parallel to our discovery lab, Oncoheroes is scouting for compounds from biopharma companies and academic research centers that hold real potential to cure several types of childhood cancer.”

Leukemia Drug Finds New Use

Approved by the FDA in 1993, the drug cladribine offered hope for the then-fatal blood cancer called hairy cell leukemia, or HCL, and was soon expanded as a therapy for multiple sclerosis. Developed by NFCR scientist Dr. Dennis Carson at the University of California, San Diego, cladribine acts like a purine nucleoside agent, which prevents cells from making DNA and RNA, and can selectively kill hairy cell leukemia cells. Heralded as a breakthrough cure, cladribine remains the first-line treatment for HCL and is also tapped as a treatment for B-cell chronic lymphocytic leukemia. Now researchers have expanded its use to another deadly form of leukemia, drug-resistant T-cell prolymphocytic leukemia (T-PLL). In a small study, T-PLL patients were desensitized to the antibody drug, alemtuzumab when it was combined with cladribine; the cancer essentially lost its drug resistance. In seven out of eight patients, the cladribine-alemtuzumab combo resulted in complete remissions, and a partial remission in the final patient. As of 2015, some patients remain in remission, although others have passes away. Alemtuzumab is marketed under the name Campath; cladribine under the name Leustatin. Considered very rare, T-PLL is an aggressive cancer of the out of control growth of mature T-cells, the assassins of the immune system. Chromosomal abnormalities are a hallmark in T-PLL patients; the most common chromosomal abnormalities are inversions or translocations involving chromosome 14 that result in mutations to the proto-oncogene TCL-1 (oncogenes are genes which in certain circumstances can transform a healthy cell to a cancerous one). Also frequently detected in patients with T-PLL are abnormalities in chromosome 8, primarily trisomy 8q, which is an extra copy of genetic material on the long arm (q) of chromosome 8. Deletions or mutations to the tumor suppressor gene ATM have also been observed in patients with T-PLL. Symptoms include bruising easily, rashes or skin lesions, swollen lymph nodes, quickly feeling full when eating, abdominal pain on the left side of the body due to a swollen spleen, and fullness. Cladribine was a trailblazer at the time of its introduction over 20 years ago. “[It was] a targeted agent directed against lymphocytes at a time when there was no such thing as targeted agents,” Carson recalls; targeted drugs and therapies are now among the hottest fields in medicine. Other treatments for T-PLL include combining alemtuzumab with the drug pentostatin, the FMC drug combination (fludarabine, mitoxantrone, and cyclophosphamide) followed by intravenous alemtuzumab, and hematopoietic stem cell transplantation. While it is not FDA-approved as a treatment for T-PLL, the drug nelarabine can also be used in some cases. Researchers admit that the initial trial of the cladribine-alemtuzumab combination dealt with a very small number of patients and that larger studies will have to be done per standard medical protocol to confirm safety and efficacy. That being said, the drug combination may move more quickly out of the trial phase and into practice due to the fact that both drugs are already approved by the FDA. References Fike, Bradley. (2015). Leukemia drug cladribine may get new use. Retrieved from:

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