Pediatric phase II trials of poly-ICLC in the management of newly diagnosed and recurrent brain tumors

J Pediatr Hematol Oncol. 2014 Aug;36(6):451-7. doi: 10.1097/MPH.0000000000000047.


Brain tumors are the most common solid tumor diagnosed in childhood that account for significant morbidity and mortality. New therapies are urgently needed; hence, we conducted the first ever prospective open-label phase II trials of the biological response modifier, poly-ICLC, in children with brain tumors. Poly-ICLC is a synthetic double-stranded RNA that has direct antiviral, antineoplastic, and immune adjuvant effects. A total of 47 children representing a variety of brain tumor histopathologic subtypes were treated with poly-ICLC. On the basis of the results of the initial phase II trial, an expanded prospective phase II trial in low-grade glioma (LGG) has been initiated. MRI was used to acquire volume-based measures of tumor response. No dose-limiting toxicities have been observed. In the initial study 3 of 12 subjects with progressive high-grade gliomas (HGGs) responded, and 2 of 4 children with progressive LGG experienced stable disease for 18 to 24 months. In the follow-up LGG phase II study, 2 of 5 LGG patients were stable over 18 months, with 1 stable for 6 months. Overall 5 of 10 LGG patients have responded. On the basis of low toxicity and the promising LGG response, poly-ICLC may be effective for childhood LGG, and the results justify biomarker studies for personalization of poly-ICLC as a single agent or adjuvant.

Publication types

  • Clinical Trial, Phase II
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Antineoplastic Agents / therapeutic use*
  • Antineoplastic Combined Chemotherapy Protocols / administration & dosage
  • Antineoplastic Combined Chemotherapy Protocols / adverse effects
  • Brain Neoplasms / pathology
  • Brain Neoplasms / therapy*
  • Carboxymethylcellulose Sodium / administration & dosage
  • Carboxymethylcellulose Sodium / adverse effects
  • Carboxymethylcellulose Sodium / analogs & derivatives*
  • Child
  • Child, Preschool
  • Dose-Response Relationship, Drug
  • Female
  • Glioma / pathology
  • Glioma / therapy*
  • Humans
  • Infant
  • Magnetic Resonance Angiography
  • Male
  • Neoplasm Grading
  • Poly I-C / administration & dosage*
  • Poly I-C / adverse effects
  • Polylysine / administration & dosage
  • Polylysine / adverse effects
  • Polylysine / analogs & derivatives*
  • Prospective Studies
  • Treatment Outcome
  • Young Adult


  • Antineoplastic Agents
  • Polylysine
  • poly ICLC
  • Carboxymethylcellulose Sodium
  • Poly I-C