The US Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to burtomab for pediatric relapsed/refractory metastatic neuroblastoma with central nervous system or leptomeningeal metastasis.1

“Currently, there are no approved drugs to treat this type of advanced neuroblastoma,” Nai-Kong Cheung, MD, PhD, of Memorial Sloan Kettering Cancer Center in New York, and creator of burtomab, said in a press release.

Burtomab is a radio-iodinated (iodine-131) murine monoclonal antibody that targets the immunomodulatory glycoprotein, B7-H3. This glycoprotein is expressed on the cell membrane primarily by solid tumors, with limited expression by normal tissues.

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A phase 1 study presented at the 2017 American Society of Clinical Oncology Annual Meeting demonstrated substantially prolonged overall survival (OS) with burtomab compared with historic controls among patients with neuroblastoma with central nervous system metastasis.2

The study enrolled 105 patients, of whom, 80 received treatment with burtomab in addition to standard intraventricular compartmental chemotherapy with irinotecan, temozolomide, and carboplatin. The remaining 25 patients were not eligible for burtomab.

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Radiologic improvement after burtomab treatment occurred among 36% of patients with measurable disease. The median duration of response was 49 weeks (range, 2.6 to 586 weeks).

The median OS was 58 months; 45% of patients were alive at 36 months and 29% at over 60 months. The mean survival of patients who did not receive burtomab was 8.6 months.


  1. Burtomab receives breakthrough therapy designation for advanced form of pediatric cancer. BusinessWire website. Published June 7, 2017. Accessed June 8, 2017.
  2. Kramer K, Kushner BH, Modak S, et al. A curative approach to central nervous system metastases of neuroblastoma. J Clin Oncol. 2017;35(suppl; abstr 10545).