South Plains Oncology Consortium – Neuroblastoma

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Currently there is no known effective treatment for recurrent/resistant neuroblastoma.

Fenretinide is an anticancer agent that may work differently than standard chemotherapy
medicines. It may cause the buildup of wax-like substances in neuroblastoma cancer cells,
called “ceramides” or other chemicals, called ‘reactive oxygen species’. In laboratory
studies it was found that if too much ceramide or reactive oxygen species build up in
neuroblastoma cells, they may die.

In addition, researchers are testing to see if a drug called ketoconazole, commonly used to
treat fungus infections, can increase fenretinide levels in the body by interfering with the
body’s ability to break down fenretinide.

This study is being done: 1) to allow patients with recurrent/refractory neuroblastoma
patients who would otherwise not be able to access fenretinide/LXS oral powder for treatment
to do so; 2) to further describe the side effects of fenretinide and ketoconazole when given
by mouth for seven days every three weeks; 3) to determine if a patient’s tumor gets smaller
after treatment with fenretinide oral powder plus ketoconazole or fenretinide oral powder
alone.

Rare Disease: