Musella Foundation Awards $250,000 Brain Tumor Research Grant This is the 3rd of the 4 payments of $250K that we pledged toward this $1 million project. I still need help raising the final $250K! If anyone is interested in helping, contact me! (Al Musella, DPM musella@virtualtrials.com)
Complete radiological response following subtotal resection in three glioblastoma patients under treatment with Tumor Treating Fields. This is a relatively small trial but shows a huge effect supporting the use of Optune for Glioblastoma.
In the big EF-14 phase 3 trial for newly diagnosed glioblastoma, the trial was stopped at the first interim analysis because it was obvious the Optune arm was doing so much better than the control group that it was unethical to continue withholding Optune from newly diagnosed patients. The people running this study did not do that - they allowed it to go on for 5 years - knowing that they were withholding the best treatment. Doesn't sound fair to me.
Regorafenib in glioblastoma recurrence: how to deal with conflicting ‘real-life’ experiences? Very interesting question. The clinical trial for Regorafenib in recurrent gbm showed a small benefit over Lomustine. However, the control group of Lomustine did terrible compared to the large trial testing Lomustine. In the Regorafenib trial, the control group of Lomustine had 5.6 months of overall survival. In the Lomustine trial, the same type of patients had a median overall survival of 8.6–9.8 months. The Regorafenin group had 7.4 months. So although Regorafenib did better than the randomized control group it did not do as well as the Lomustine in the Lomustine trial.
However, reports from real world use of the drug came in that said almost all patients get severe side effects and they did not see the benefit seen in the trial. So what do you do?
Obviously more research needs to be done hopefully to find if there are biomarkers that would push you toward Lomustine, Regorafenib or the combination. Perhaps the best way would be for having all patients be followed in a registry so we can see how it works in the real world and correlate with biomarkers. As in our brain tumor virtual trial project or our new Excelsior registry https://clinicaltrials.gov/ct2/show/NCT03793088?term=musella&draw=2&rank=4
Cost-effectiveness analysis of the addition of bevacizumab to temozolomide therapy for the treatment of unresected glioblastoma. This is one of the biggest problems we have - the high cost of treatments. This analysis found a small benefit to adding bevacizumab (Avastin) to Temodar for people with inoperable Glioblastomas. However, because of the high cost of the drug, it was determined that is not cost effective. They set the acceptability bar at $26,500 to add 1 year of life. This treatment worked out to $171,638 for each year of life added. Not even close.
This is a societal problem. I do not blame the drug companies, as the cost to develop a drug under our system is so high that to recoup their investment, the prices have to be high. Without high prices, there would not be new drugs getting approved. We need to change the system so that any researcher with a good idea could afford to bring a drug through the system to get approval - which not only will drastically lower the cost of new drugs but gives us a wider range of treatments to use.
