Exclusive: Interview With DelMar Pharmaceutical CEO Jeffrey Bacha This drug, Val-083, might just be the next big step forward. Late stage trials are about to start. It already had been approved in China for other diseases, and over 1,000 patients took it and have shown that toxicity is not a problem. It might especially be useful for gbm patients who have unmethylated MGMT.
The article may have missed a little on the statistics. I doubt if 200,000 GBMs are diagnosed a year. They say "less than 200,000" but it is more like 20,000.
High response rate in phase I/II paediatric brain cancer trial Luckily, the drug they used, dabrafenib, is already approved for other types of cancer so it can be used off label immediately. Unfortunately, in melanoma, it was found that most patients become resistant to it quickly. There is work going on now using another drug, Trametinib, combined with the dabrafenib, which may prevent that resistance. The FDA approved this combination for melanoma. Maybe it would work in brain tumors also!