A Phase I Study of Mebendazole for the Treatment of Pediatric Gliomas
Status:
Recruiting
Trial end date:
2023-04-01
Target enrollment:
Participant gender:
Summary
This is a study to determine the safety and efficacy of the drug, mebendazole, when used in
combination with standard chemotherapy drugs 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 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. Some
low-grade gliomas have a more aggressive biology and an increased likelihood of resistance or
recurrence.
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.
High grade gliomas are more aggressive tumors with poor prognoses. The standard therapy is
radiation therapy. A variety of adjuvant chemotherapeutic combinations have been used, but
with disappointing results. For high-grade gliomas this study will add mebendazole to the
established combination of bevacizumab and irinotecan to determine this combinations safety
and efficacy