News & Blog

Advice Column, April 2018: Exploring Novel Brain Tumor Treatments

National Brain Tumor Society recently announced the launch of a new patient and care-partner education, empowerment, and preparedness effort. The new initiative will provide the resources, information, tools, and guidance brain tumor patients and their care-partners need to play a more active role in their treatment planning and decision-making.

As part of the broader effort to inform and empower patients to partner more closely with their medical teams and play a proactive role in their treatment pathways, we’ve started a monthly series of ongoing “advice” blogs.

In our first two installments, we looked at Brain Tumor Treatment Options and Questions to Consider Asking Your Medical Team During Treatment. This month, we’ll examine how to explore experimental treatment options – sometimes also referred to as “novel” and/or “investigational” treatments.

As we mentioned in our first Advice Column on Brain Tumor Treatment Options, one potential route to consider during your brain tumor treatment experience is novel (or “experimental” or “investigational”) treatments. This refers to new treatments that have not yet been approved by the U.S. Food and Drug Administration (FDA).

Though there is inherent risk involved in trying unproven treatments that have yet to be fully deemed safe and effective by the FDA, exploring one of the various avenues to access novel therapies can open new opportunities and options beyond the few standard treatments currently approved and available to brain tumor patients.

If, in consultation with your medical team, you choose to explore novel treatment options – either at initial diagnosis or further in your treatment experience – there are a few options to consider:


First, you may be eligible for a clinical trial; you can also search for brain tumor clinical trials using the NBTS Clinical Trial Finder. Clinical trials not only provide early access to potentially beneficial treatments, but they also advance science and are an integral part of the drug development and approval process.

However, many patients do not qualify for clinical trials for a variety of reasons (other conditions, poor overall health, previous therapies, etc.). It’s important to discuss with your doctors what trials you might be eligible for, what risks may be involved, what participation in that trial will require, and what other options you have to forgo to participate. It’s best to have this conversation as early as possible in the decision-making process on treatments, because (as noted above) certain previous therapies may be an exclusion criterion for some trials you’d otherwise be eligible for.


In some cases, patients may wish to try an experimental treatment, but not qualify for any of the trials evaluating that therapy – either because they do not meet the inclusion/exclusion criteria or because the trial is already fully enrolled and/or not enrolling new patients. In these cases, the FDA has a program that allows patients the potential to gain access to investigational medical products outside of a clinical trial called, “Expanded Access.”

The FDA approves approximately 99% of Expanded Access requests that it receives. However, there is no guarantee that the patient’s doctor will be able to apply for expanded access from the FDA, as they first need confirmation from the manufacturer that they will provide the drug. It is up to the company what their policy for expanded access will be and they may deny requests.

Through the 21st Century Cures Act, pharmaceutical companies must make public and easily accessible the information regarding the company’s policy for evaluating and responding to requests for expanded access (sometimes called “Compassionate Use”). If you do not qualify for a clinical trial, this may be an option you want to discuss with your doctor, so that he may help you apply. Additionally, there are some resources that help patients navigate the Expanded Access process, like the Reagan-Udall Foundation Navigator.


Sometimes, a therapy is potentially effective for the treatment of a particular brain tumor (often based on its biological make-up) is already approved for another cancer type or disease; in these cases, your doctor may prescribe the therapy through a process called “off-label” use on a case-by-case basis. As long as the treatment has been approved by the FDA (again, even for another disease) and there is evidence for the use of the therapy for your brain tumor, your doctor will not need to get FDA approval to administer this treatment for individual patients they believe could benefit. For example, if you have your tumor sequenced (or molecularly/generically tested) and the results show that a mutation to your BRAF gene could be driving your tumor, your doctor may discuss the possibility of treating you with a “BRAF inhibitor,” even though these types of drugs haven’t been approved in brain tumors yet (they have been for other cancers though). See a blog we did on a news story about a situation like this, here.

Off-label use can also describe when your doctor gives you a different dose or dosing type (such as a liquid solution instead of a pill) of a medication than what is approved by the FDA. It also describes the use of therapies approved for adults in children.

Like all experimental treatment options, off-label therapies have no guarantee of success and could come with unexpected side-effects. It’s best to discuss that rationale for off-label use and what you might expect with your doctors.

Though all investigational medical products require the FDA’s Institutional Review Board (IRB) approval for use in humans, it is important to note that unapproved medical products may have serious, unexpected side-effects. You should discuss the possible side-effects of any treatment with your doctor and keep your medical team informed of any side effects you experience.

The full section on Exploring Novel Treatments can be found on our Brain Tumor Experience web resources here:

Brain Tumor Treatment

The above content is intended as broad suggestions for topics and issues to discuss with your medical team. National Brain Tumor Society is not a medical provider and therefore cannot endorse any individual treatments or make specific medical recommendations for patients. Your ultimate treatment decisions should always be made in full consultation with licensed medical providers, preferably at high-volume medical centers that have specialists with deep experience and knowledge in treating brain tumor patients.

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