Immunotherapy for Childhood Cancer

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  • Childhood Cancer
  • Treatment Options
  • CRI's Impact
  • Clinical Trials

How is Immunotherapy Changing the Outlook for Patients with Childhood Cancer?

Reviewed By: Alex Huang, M.D.

Although children are affected by several of the same types of cancer as adults, the ways in which they develop―and the ways in which they must be treated―can be drastically different between the two for several reasons.

The most common types of cancer in children under the age of 15 are acute lymphocytic leukemia, brain tumors, neuroblastoma, and non-Hodgkin lymphoma, while the most common types in adolescents between the ages of 15-19 are Hodgkin lymphoma, thyroid carcinoma, brain cancer, and testicular germ cell tumors.

The survival rates for these different types of cancers vary, but overall, the majority of children diagnosed with cancer are able to survive long-term, thanks to advances in treatments, including immune-based therapies, over the past several decades.

Unfortunately, some of these treatments can be damaging to these young patients and leave them with chronic conditions for the rest of their lives.

Urgent Need

Approximately 1 of every 285 children in the United States will be diagnosed with cancer before the age of 20, and in about 80% of these cases, the child’s cancer will have metastasized to other tissues by the time it is discovered. The average age of diagnosis is 6 years old.


Surgery, radiation, and chemotherapy are all common options for childhood cancers. In addition to stem cell transplants, which have been used for decades, the immunotherapeutic antibody dinutuximab⎯which targets the glycolipid GD2⎯is approved for childhood cancer patients with neuroblastoma. The anti-PD-1 checkpoint immunotherapy pembrolizumab is also approved for children with Hodgkin lymphoma, while a cell-based immunotherapy known as anti-CD19 CAR T cells have been approved for children and young adults with leukemia (a form known as acute lymohblastic leukemia, or ALL). Some of the above immunotherapies are used in combination with chemotherapy, which causes long-lasting damage in these growing children. Fortunately, efforts in both the lab and in the clinic are working to overcome this. Researchers are working to improve stem cell transplants so that they don’t require immune-depleting chemotherapy beforehand, and clinical trials evaluating novel immunotherapy approaches, such as CAR T cells, that might be able to help young patients while sparing them the debilitating side effects. These clinical trials have been, and continue to be, important for the advancement of improved immunotherapies for all patients, including children. For people already infected with HPV or facing a cervical or ano-genital cancer diagnosis, the FDA-approved vaccines are of no benefit. In addition to bevacizumab (Avastin®) a targeted antibody that is already approved for cervical cancer patients, there are several other types of immunotherapies that are being evaluated for cervical cancer, including some in combination.

CRI Contributions & Impact

  • Carl H. June, M.D. of the Perelman School of Medicine at the University of Pennsylvania, a member of CRI’s Scientific Advisory Council and Clinical Leadership Committee, has pioneered many advances in CAR T cell immunotherapy, which supplements patients’ immune system with genetically enhanced anti-cancer immune cells. He has also led clinical trials that have successfully treated young patients with CAR T cells. One such patient is Emily Whitehead, who, with the help of the CAR T cells Dr. June designed and treated her with, overcame her leukemia and has been cancer-free for over four years now.

  • Three CRI postdoctoral fellows⎯Kevin C. Barry, Ph.D., at the University of California, San Francisco; Sofia L. Novais de Oliveira, Ph.D., at the University of Wisconsin-Madison; and Kevin M. Sullivan, M.D., at the University of Washington School of Medicine; and assistant professor of medicine Amy K. Kim, M.D., at Johns Hopkins University―are exploring how immunotherapy could help against fibrolamellar hepatocellular carcinoma (FL-HCC), a rare form of liver cancer that primarily affects young people. Their work encompasses several strategies, including: developing FL-HCC animal models to characterize tumor-immune interactions, exploring if a mutated protein associated with FL-HCC could be targeted by immunotherapy, identifying immune checkpoints that could potentially serve as targets for immunotherapy as well as biomarkers for analyzing patients, and evaluating the effectiveness of immunotherapy strategies against FL-HCC patient samples in the lab. Funded with generous support from the Fibrolamellar Cancer Foundation.

  • Mark P. Rubinstein, Ph.D. a CRI CLIP Investigator at the Medical University of South Carolina, is working to improve the effectiveness of stem cell transplants, a common treatment for childhood cancers. The need to wipe out a patient’s existing immune system beforehand can be problematic, so Dr. Rubinstein is developing a strategy that eliminates the need to do so.

  • Bradley Wayne Blaser, M.D., Ph.D., a CRI Irvington Postdoctoral Fellow at the Dana-Farber Cancer Institute, is also tackling the challenges of stem cell transplantation. Dr. Blaser is identifying the factors that promote successful engraftment of transplanted cells in animal models, which he will then attempt to translate into improved approaches for human patients.

Featured Patient

It was empowering to know that it was my immune system that did the job.

Sharon Belvin
Melanoma  |  Diagnosed 2004
Read My Story
Featured Scientist
Geoffrey Lovely, Ph.D.
National Institute on Aging
Postdoctoral Fellow  |  2016
View Funding Profile

Childhood Cancer Statistics

4th Most frequently diagnosed cancer of women worldwide
91% 5-year survival rate for localized disease
4 Types of immunotherapy clinical trials
$12 Million Awarded by CRI to cervical cancer research

Clinical Trials

  • Adoptive Cell Therapy
  • Checkpoint Inhibitors/Immune Modulators
  • Cytokines/Monoclonal Antibodies
  • Oncolytic Virus Therapy
  • Cancer Vaccines

One promising and active immunotherapy for childhood cancer is adoptive T cell transfer. In this approach, T cells are removed from a patient, genetically modified or treated with chemicals to enhance their activity, and then re-introduced into the patient with the goal of improving the immune system’s anti-cancer response.

  • A phase I/II trial of KTE-C19 (anti-CD19 CAR T cells) for pediatric and adolescent subjects with relapsed/refractory B-precursor acute lymphoblastic leukemia (NCT02625480).

  • A phase I/II trial of genetically modified T cells directed against CD19 for pediatric and young adult patients with relapsed/refractory CD19+ leukemia (NCT02028455).

  • A phase I trial of anti-GPC3 (glypican-3) CAR T cells along with lymphodepleting chemotherapy for patients with pediatric solid tumors (NCT02932956).

  • A phase I trial of anti-HER2 CAR T cells for subjects, including pediatric patients, with advanced sarcoma (NCT00902044).

Another avenue of clinical research in childhood cancer is the use of immune checkpoint inhibitors. These treatments work by targeting molecules that serve as checks and balances in the regulation of immune responses. By blocking inhibitory molecules or, alternatively, activating stimulatory molecules, these treatments are designed to unleash or enhance pre-existing anti-cancer immune responses.

  • A phase I/II trial of nivolumab (anti-PD-1) and low dose cyclophosphamide in progressive/relapsed pediatric solid tumors (NCT02901145).

  • A phase I/II trial of MPDL3280A (anti-PD-L1) in pediatric and young adult participants with solid tumors (NCT02541604).

Cytokines are messenger molecules that help control the growth and activity of immune system cells. Monoclonal antibodies are molecules, generated in the lab, that target specific antigens on cells.

  • A phase I trial of ramucirumab, a human monoclonal antibody against the vascular endothelial growth factor-2 (VEGFR-2) receptor, in children with refractory solid tumors, including CNS tumors (NCT02564198).

  • A pilot phase II trial of hu14.18K322A (anti-GD-2)⎯given in combination with chemotherapy, radiation therapy, and cell transplants⎯for children with advanced stage neuroblastoma (NCT01857934).

  • A phase II trial of brentuximab vedotin (anti-CD30 antibody-drug conjugate) in combination with chemotherapy and radiation therapy for younger patients with advanced Hodgkin lymphoma (NCT01920932).

  • A phase I/II trial of TB-403 (anti-PIGF) for pediatric subjects with relapsed or refractory medulloblastoma (NCT02748135).

Oncolytic virus therapy uses a modified virus that can cause tumor cells to self-destruct and generate a greater immune response against the cancer.

  • A phase I trial of HSV207, a modified herpes virus designed to target tumor cells and increase their immunogenicitiy, alone or in combination with a single radiation dose,  in children with recurrent supratentorial brain tumors (NCT02457845).

Cancer vaccines are designed to elicit an immune response against tumor-specific or tumor-associated antigens, encouraging the immune system to attack cancer cells bearing these antigens. Clinical studies include:

  • A phase 1 trial of a dendritic cell (DC) vaccine administered with imiquimod, a TLR7 agonist that stimulates the innate immune system, for patients with malignant glioma and glioblastoma (NCT01808820).

  • A phase I trial of the H3.3K27M-specific peptide vaccine combined with poly-ICLC for newly diagnosed H3.3K27M-positive diffuse intrinsic pontine glioma (DIPG) and other newly diagnosed H3.3K27M-positive gliomas (NCT02960230).

  • A pilot study of vaccination with glioma antigen-peptides with Poly-ICLC for children with newly diagnosed malignant brain stem gliomas, non-brainstem high-grade gliomas, recurrent low-grade gliomas or recurrent high grade gliomas (NCT01130077).

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*Immunotherapy results may vary from patient to patient.