A Q&A with Al Musella, DPM, President, Musella Foundation For Brain Tumor Research & Information, Inc., Hewlett, NY; email: firstname.lastname@example.org, phone: 888-295-4740
Q: You direct an established foundation that supports research and information about brain tumors. What would you do if you yourself were diagnosed with a glioblastoma multiforme (GBM)?
A: Now that GBMs are in the news again, I would like to discuss what I would do if it happened to me—a newly diagnosed GBM in an adult in otherwise good shape. There are several choices.
Standard of care: Surgery, radiation, Temozolomide. Chance of 5 year survival is about 5%.
Standard of care PLUS Optune. Bumps my chance of 5 year survival up to 24.9% (if used over 90% of the time) with no added toxicity.
Phase 3 Clinical trials: There are now about nine phase 3 trials for newly diagnosed GBM. Some have impressive phase 1 and phase 2 data. By the time a treatment gets to phase 3, it has shown enough promise in earlier trials that the sponsor is willing to risk a lot of money to test in a phase 3 trial. Most have two big downsides: 1) Most have a control group of patients who receive the old standard of care so that some of the participants do not get the experimental treatment. 2) Most do not allow you to use Optune, so you are trading a known benefit for a chance at an unknown benefit.
“When approved therapies don’t work, or stop working, for people with serious or life-threatening illnesses, it puts them in a difficult position. Some turn to clinical trials that are testing experimental treatments. But many can’t do that because they are too sick, don’t meet the requirements of the trial, or can’t afford to travel to the site of a trial. That doesn’t mean they are out of options.”
“In the last six months Colorado, Louisiana, Missouri, Michigan and, most recently, Arizona have passed ‘right to try’ laws that allow terminally ill patients to access treatments that have only passed FDA Phase I clinical trials. All patients need is permission from a drug company and a prescription from a doctor.
“Right to try laws are designed to ensure that terminally ill patients taking part in clinical trials are true volunteers and have no incentive to cheat the clinical trials system as has happened in the past.
“Recently, these laws have been critiqued as misguided, and the ethics of allowing patients to use experimental drugs are still up for debate. These laws do not guarantee access to experimental treatments and patients may have to pay for them out of pocket.
“Critics of these laws worry that alternative trial designs, or access to such experimental drugs outside the clinical trials system will significantly delay the development of effective therapies.”
Over the last few years, there have been significant advances in treatment options for patients with castration-resistant prostate cancer (CRPC). An article published in the Asian Journal of Andrology reviews new and experimental treatments that may improve the outlook for treating CRPC.