Options to Treat a Glioblastoma

A Q&A with Al Musella, DPM, President, Musella Foundation For Brain Tumor Research & Information, Inc., Hewlett, NY; email: musella@virtualtrials.com, 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.

  1. Standard of care: Surgery, radiation, Temozolomide. Chance of 5 year survival is about 5%.
  2. 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.
  3. 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.
  4. Continue reading…


Why Can’t Dying Patients Get the Drugs They Want?

Excerpt:

“At first glance, a bill passed by the House of Representatives this week seems like the kind of thing anyone could get behind.

“Known as the “Right to Try” legislation, it would allow terminally ill patients access to experimental drugs without the approval of the Food and Drug Administration.

“But the bill and a similar one passed last summer by the Senate do little to address the main barrier that patients face in getting unapproved treatments: permission from the drug companies themselves.”

Go to full article.

If you’re wondering whether this story applies to your own cancer case or a loved one’s, we invite you to use our ASK Cancer Commons service.


Should Dying Patients Have the Right to Access Experimental Treatments?

“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.”


New Laws Give Dying Patients ‘Right to Try’ Unproven Drugs

“Colorado, Missouri and Louisiana are poised to become the first states in the nation to give terminally ill patients the right to try experimental drugs without the blessing of the Food and Drug Administration, setting the stage for what could be a lengthy battle over who should decide whether a drug is too risky to try.

“Lawmakers in the three states have passed “Right to Try” laws with unanimous votes in recent weeks, after high-profile, social media campaigns in which families of dying patients have pushed for access to unapproved but potentially lifesaving drugs. Colorado’s governor is expected to sign that state’s law Saturday.”

Editor’s note: New cancer drugs can take a long time to reach the clinic, even after they have already proven safe and beneficial. Some patients are successful in gaining early access to drugs (see our Chief Scientist’s blog post on so-called “compassionate access”), but it is a difficult process. Some drug companies are trying to remedy the issue by starting “expanded access trials” that give drugs to patients unable to enroll in the clinical trials testing them. For example, Novartis made its promising drug LDK378 available under expanded access in trial number NCT01947608 in September, 2013.


New Laws Give Dying Patients ‘Right to Try’ Unproven Drugs

“Colorado, Missouri and Louisiana are poised to become the first states in the nation to give terminally ill patients the right to try experimental drugs without the blessing of the Food and Drug Administration, setting the stage for what could be a lengthy battle over who should decide whether a drug is too risky to try.

“Lawmakers in the three states have passed “Right to Try” laws with unanimous votes in recent weeks, after high-profile, social media campaigns in which families of dying patients have pushed for access to unapproved but potentially lifesaving drugs. Colorado’s governor is expected to sign that state’s law Saturday.”

Editor’s note: New cancer drugs can take a long time to reach the clinic, even after they have already proven safe and beneficial. Some patients are successful in gaining early access to drugs (see our Chief Scientist’s blog post on so-called “compassionate access”), but it is a difficult process. Some drug companies are trying to remedy the issue by starting “expanded access trials” that give drugs to patients unable to enroll in the clinical trials testing them. For example, Novartis made its promising drug LDK378 available under expanded access in trial number NCT01947608 in September, 2013.


New Laws Give Dying Patients ‘Right to Try’ Unproven Drugs

“Colorado, Missouri and Louisiana are poised to become the first states in the nation to give terminally ill patients the right to try experimental drugs without the blessing of the Food and Drug Administration, setting the stage for what could be a lengthy battle over who should decide whether a drug is too risky to try.

“Lawmakers in the three states have passed “Right to Try” laws with unanimous votes in recent weeks, after high-profile, social media campaigns in which families of dying patients have pushed for access to unapproved but potentially lifesaving drugs. Colorado’s governor is expected to sign that state’s law Saturday.”

Editor’s note: New cancer drugs can take a long time to reach the clinic, even after they have already proven safe and beneficial. Some patients are successful in gaining early access to drugs (see our Chief Scientist’s blog post on so-called “compassionate access”), but it is a difficult process. Some drug companies are trying to remedy the issue by starting “expanded access trials” that give drugs to patients unable to enroll in the clinical trials testing them. For example, Novartis made its promising drug LDK378 available under expanded access in trial number NCT01947608 in September, 2013.