MTX-110 for newly-diagnosed diffuse intrinsic pontine glioma

MTX-110 is in clinical development for newly-diagnosed diffuse intrinsic pontine glioma (DIPG). DIPG is a high grade childhood brain tumour. The cause is unknown. DIPG develops in a part of the brain stem known as the pons, which controls essential body functions including heartbeat, breathing, eyesight and balance. The symptoms of DIPG may include; abnormal alignment of the eyes, arm and leg muscle weakness, unstable balance and speech difficulties. Because the tumour grows amongst normal nerves cells of the brain stem, it is difficult to treat as they are not surgically resectable. Radiotherapy can temporarily slow the tumour's growth but is not curative and chemotherapy is often ineffective.
MTX-110 contains the anticancer drug panobinostat, which works by inhibiting an enzyme that increases the division and growth of cancer cells. Orally administered panobinostat cannot cross the blood brain barrier and its use in treating DIPG is currently limited. MTX-110 contains a special formulation of panobinostat that enables the direct delivery of high concentrations of panobinostat by infusion to tumour sites, which bypasses the blood-brain barrier. This is expected to improve both the safety and efficacy of the treatment while reducing the potential for toxicity and other side effects.