While immunotherapy has revolutionized cancer treatments, its efficacy remains relatively undefined in pediatric settings for brain tumors.

While immunotherapy has revolutionized cancer treatments, its efficacy remains relatively undefined in pediatric settings for brain tumors. Children’s National Hospital experts and other institutions argue in a review published in Nature Cancer that there is a need for closer collaborations between academia, industry partners, regulatory bodies and funders to progress the field.

Eugene Hwang, M.D., associate division chief of Oncology and neuro-oncologist at Children’s National, led the review that outlines immunotherapeutic hurdles and simultaneously proposes next steps for immunotherapy use in these patients. These considerations will aid pediatric oncologists make better recommendations and advances in this type of treatment.

“The promise of immunotherapy in helping to cure children with brain tumors is exciting,” Dr. Hwang said. “This type of approach has already revolutionized treatments for many different kinds of cancer, and a comprehensive review of this complicated arena, especially by leading voices in the field, can help set the stage for finally moving the needle for these patients.”

The review is especially helpful as children harbor unique elements of immunity and the brain presents distinct obstacles to immune attack that are not present in other cancers. For example, there are challenges in antigen identification, the blood-brain barrier and the tumor microenvironment. For many pediatric cancer doctors as well this novel, complex form of therapy is outside of their historical training.

To overcome these challenges, the authors encourage philanthropic organizations and patient advocacy groups to be part of the process that can help fill funding gaps in patient-focused pre-clinical and clinical research and educate patients and families.

“Multiple stakeholders around pediatric brain cancer immunotherapy must be mobilized in a concerted fashion,” Hawk et al. argue in the piece. “The need for close academic collaboration with industry partners and regulatory bodies is increasingly apparent given the unique pediatric phenotypes and complex outcomes in immunotherapeutic trials, and progress will be made at the interface of the interactions of all these key stakeholders.”

The group of internationally renowned pediatric brain tumor-focused immunotherapy experts comprehensively reviewed the advances in the major modalities of immunotherapy and the landscape of preclinical modeling for these patients to date.

Investigators at Children’s National, for example, are leading several national and international trials involving immunotherapy which have spurred international meetings with a focus in childhood brain tumor immunotherapies.

“The multiple T cell trials led by Children’s National are perfect examples of truly field-leading innovative immunotherapy, as are the other trials that are led by our own investigators,” Dr. Hwang added.