MTX110 is a new formulation of panobinostat, a chemotherapy drug that has shown promise in laboratory models of medulloblastoma, the most common malignant brain tumor in children. Now, MTX110 is the focus of a novel trial that places the therapy directly into the fourth ventricle of the brain to treat patients with recurrent medulloblastoma.
According to the American Cancer Society, about 500 children are diagnosed with medulloblastoma every year. Patients undergo surgery and then receive radiation therapy and chemotherapy. Current treatments are often associated with considerable toxicity, and when tumors recur despite these treatments, survival rates are low.
“The current treatments for children and adults with medulloblastoma are inadequate,” said David Sandberg, MD, director and Dr. Marnie Rose Professor in Pediatric Neurosurgery at McGovern Medical School at The University of Texas Health Science Center at Houston (UTHealth). “Children have low survival rates despite salvage therapy, and novel approaches are needed. This is a new trial with a novel drug and we are very hopeful that we can help patients with this devastating disease.”
The pilot study, which has been approved by the Food and Drug Administration, will enroll five patients with recurrent medulloblastoma at Children’s Memorial Hermann Hospital. MTX110 is a new formulation of panobinostat from Midatech Pharma PLC.