A new drug therapy has been confirmed promising against highly aggressive and difficult to treat incurable brain tumors found at the base of the brain in children.
According pre-clinical studies by US-Australian drug discovery company Novogen in conjunction with Australian paediatric cancer researchers, an experimental cancer drug-candidate code-named, TRXE-009, is shown to provide an important new therapy against incurable paediatric brain cancer called Diffuse Intrinsic Pontine Glioma (DIPG).
The prognosis of children with newly diagnosed DIPG is very poor with a median survival of less than 1 year, a statistic that has not improved in over 50 years.
Due to the possibility of damaging crucial neural structures, surgery is not an option to treat the deadly tumors, which invade diffusely throughout the brain stem, growing between normal nerve cells and eventually making them hard to target. Radiation provides only temporary relief while the role of chemotherapy remains unclear.
Investigating the ability of TRXE-009, a super-benzopyran compound, to kill freshly established patient-derived cell cultures collected from patients with DIPG, the latest study indicated that TRXE-009 kills DIPG cells at therapeutically relevant concentrations by inducing a specific type of cell death – caspase-dependent apoptosis.
According to the pre-clinical data, TRXE-009 has a high therapeutic index and is able to hit cancer cells at concentrations that have little effect on normal cells.
“These are preliminary studies but we are very excited about the striking activity we are seeing in these highly resistant tumorspheres. TRXE-009 is one of the most potent compounds we have studied to date in this setting,” says Lead Investigator David Ziegler.
The main challenge in treating brain tumors is to overwhelm the blood-brain barrier, a tight-structure filtering system that must keep toxins, chemicals and alien substances from reaching the brain tissue, also blocks chemotherapeutic drugs.
Novogen says TRXE-009 has been designed with this challenge in mind and has further enlisted the combined efforts of several international research groups to identify the optimal means of delivering it to adults and children so that it crosses the blood-brain barrier.