Pediatric Brain Tumors

Pediatric brain tumors are the leading cancer-related cause of death in children under the age of 14. Children with recurrent high grade glioma have very few treatment options and their prognosis remains poor. Temozolomide (TMZ) has failed to improve survival, partially due to a high expression of MGMT, known to cause TMZ-resistance, and frequent deficiencies in the DNA mismatch repair (MMR) pathway, a secondary TMZ-resistance mechanism.

VAL-083 for the Treatment of Pediatric Brain Tumors

Hope Against Pediatric Brain Tumors

Our research has demonstrated promising pre-clinical results for the treatment of pediatric brain tumors with our VAL-083. In prior clinical trials, VAL-083 demonstrated activity against pediatric brain cancers.

Major challenges in the treatment of pediatric brain tumors include the difficulty for therapeutics in penetrating the blood brain barrier and resistance mechanisms in the tumors, including MGMT, MMR and p53. VAL-083 readily crosses the blood brain barrier and maintains functionality regardless of the tumors’ MGMT or MMR status, and furthermore appears unaffected by the p53 status of the cancer cells.

VAL-083 has been shown to cause robust and irreversible DNA double strand breaks and S/G2 phase cell cycle arrest in the cancer cells, ultimately leading to cancer cell death. We’re investigating VAL-083 in pediatric high-grade gliomas.

The FDA Office of Orphan Products Development (OOPD) has granted us orphan drug designation for VAL-083 in the treatment of medulloblastoma.

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