A Phase I Study of Mebendazole for the Treatment of Pediatric Gliomas


This is a study to determine the safety and efficacy of the drug, mebendazole, when used in combination with three additional chemotherapy agents, vincristine, carboplatin, and temozolomide, for the treatment of pediatric brain tumors. Mebendazole is a drug used to treat infections with intestinal parasites and has a long track record of safety in humans. Recently, it was discovered that mebendazole may be effective in treating cancer as well, in particular brain tumors. Studies using both cell cultures and mouse models demonstrated that mebendazole was effective in decreasing the growth of brain tumor cells. This study focuses on the treatment of a category of brain tumors called low-grade gliomas. Low-grade gliomas are tumors arising from the glial cells of the central nervous system and are characterized by slower, less aggressive growth than that of high-grade gliomas. The category of low-grade glioma includes many subtypes. One such subtype, pilomyxoid astrocytoma, is a grade II tumor which has an aggressive nature with an increased rate of recurrence and decreased survival rate compared to other low-grade gliomas. Low-grade gliomas are often able to be treated by observation alone if they receive a total surgical resection. However, tumors which are only partially resected and continue to grow or cause symptoms, or those which recur following total resection require additional treatment, such as chemotherapy. Due to their more aggressive nature, pilomyxoid astrocytomas, even when totally resected, will often be treated with chemotherapy. The current first-line treatment at our institution for these low-grade gliomas involves a three-drug chemotherapy regimen of vincristine, carboplatin, and temozolomide. However, based on our data from our own historical controls, over 50% of patients with pilomyxoid astrocytomas will continue to have disease progression while on this treatment. We believe that mebendazole in combination with vincristine, carboplatin, and temozolomide may provide an additional therapeutic benefit with increased progression-free and overall survival for low-grade glioma patients, particularly for those with pilomyxoid astrocytomas.

Study Start Date

April 2015

Estimated Completion Date

April 2020


  • Drug: Bevacizumab
  • Drug: Mebendazole
  • Drug: Carboplatin
  • Drug: Irinotecan
  • Drug: Vincristine
  • Drug: Temozolomide


  • Pediatrics: Pediatric Heme/Oncology
  • Oncology: Head and Neck,Peds Heme/Oncology,Pharmacology/Therapy

MeSH Terms

  • Carboplatin
  • Glioma
  • Mebendazole
  • Pediatrics
  • Temozolomide
  • Vincristine

Study ID

North Shore Long Island Jewish Health System -- CCMC1311



Trial ID


Study Type


Trial Phase

Phase 1/Phase 2

Enrollment Quota



North Shore Long Island Jewish Health System

Inclusion Criteria

    1. Age ? 21 years 2. Timing of therapy 1. Patients must be enrolled before treatment begins. Treatment must start within 14 days of study enrollment. 2. Patients with pilomyxoid astrocytomas and pleomorphic xanthoastrocytoma must be newly diagnosed and start therapy within 28 days of diagnostic biopsy. 3. Patients pilocytic astrocytomas and optic pathway gliomas who are observed and later noted to have:
  • progression on imaging studies, or
  • progression of symptoms, or
  • appearance of new symptoms are eligible, provided that the patients have previous diagnostic biopsy of pilocytic astrocytoma or optic pathway glioma, or MRI consistent with optic pathway glioma. These patients do not need another biopsy at the time of progression and therapy should start within 4 weeks after progression. These patients must have a baseline MRI of the primary lesion not more than 30 days prior to initiation of therapy. 3. Histological diagnosis: 1. Pilomyxoid Astrocytomas 2. Pleomorphic Xanthoastrocytomas, without anaplastic transformation 3. Pilocytic Astrocytomas 4. Optic Pathway Gliomas
  • Patients with optic pathway gliomas are eligible without biopsy, but must have MRI consistent with OPG. 4. Adequate hematologic, renal, liver function as demonstrated by laboratory values performed within 21 days, inclusive prior to administration to temozolomide: 1. Absolute neutrophil count ? 1,000/ul 2. Hemoglobin ?8.0 gm/dl 3. Platelet count ? 100,000/ul 4. Adequate Liver Function Defined As
  • Total bilirubin ? 1.5 x upper limit of normal (ULN) for age, and
  • Alanine Aminotransferase (ALT) < 2.5 x upper limit of normal (ULN) for age. 5. Adequate Renal Function Defined As
  • Creatinine clearance or radioisotope glomerular filtration rate ? 70ml/min/1.73m2
  • A normal serum creatinine based on age and gender 5. Life expectancy ? 3 months 6. Concurrent medications: It is recommended that patients are weaned off or are on a tapering dose of corticosteroids before starting therapy on study. 7. Patient or legal guardian must give written, informed consent or assent (when applicable) 8. Recent mothers must agree not to breast feed while receiving medications on study. All females of childbearing age must have a negative pregnancy test within two weeks of beginning study therapy.

Exclusion Criteria

    1. Patients who received prior chemotherapy or radiotherapy for this tumor are excluded. 2. Pregnant and Nursing Women are excluded as temozolomide is potentially mutagenic. 3. Men &Women who are sexually active and refuse birth control are excluded from participating in this study 4. Patients who are unable to take oral medications because of significant vomiting will be excluded. 5. Patients with Neurofibromatosis Type 1 6. Patients who have known allergy to mebendazole or benzimidazole class drugs. 7. Patients who have previously had a severe side effect, such as agranulocytosis and neutropenia, in conjunction with previous mebendazole or benzimidazole class drug for a parasitic infection . 8. Patients who are taking metronidazole and cannot be safely moved to a different antibiotic greater than 7 days prior to starting mebendazole therapy.




21 Years and younger

Accepts Healthy Volunteers


Study Locations and Contact Information (1)

Study Location Distance Name Phone Email
Cohen Childrens Medical Center of New York - New Hyde Park, New York 39.6 miles Mark P Atlas MD 718-470-3460 matlas@nshs.edu

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