While there are many drugs and other therapies now available to treat brain tumors, the continuance of research and clinical trials is necessary to keep pioneering newer – and better – treatments for this disease. Are any of these trials right for you?
Clinical Trials
greybarUnderstanding what a clinical trial is can help you decide if it might be an option for you.

Clinical trials are the cornerstone of cancer care – drugs and procedures currently used to treat cancer have to be proven to be safe and effective.

This is why volunteers for clinical trials are closely screened and monitored by their physicians, so treatments can be refined to be the most effective and safest possible. As a brain tumor patient, navigating the process of qualifying for clinical trials can seem mysterious and confusing. It is important to understand the elements that comprise clinical trials.

There are treatment trials – to evaluate whether a new drug, surgery or radiation therapy (or a combination) has a higher success rate than current treatment options. Next are quality-of-life trials – which focus on improving quality of life issues and treatment-related symptoms. And there are prevention, screening and diagnostic trials – which focus on ways to reduce the chances of getting cancer.

Randomized controlled clinical trial

A computer determines the treatment each study participant receives. This ensures that there is no bias in the selection of treatment and that every participant has the same chance of being selected for either the experimental treatment arm (the treatment under investigation) or the standard treatment arm (the usual treatment given for a disease, to which the experimental treatment is being compared). At the end of the trial, the treatment assignments are revealed and researchers can identify whether the experimental treatment was more effective than the standard treatment, and also can compare the side effects of each treatment.

Double-blind clinical trial

In a double-blind trial, neither you nor your oncologist will know whether you are receiving the experimental or standard treatment.  At the end of the trial, the study is “unblinded” and it is revealed which treatment was received by each participant.


A placebo is a substance that is given in place of the experimental treatment, but in fact has no active ingredients.   Neither patients nor the oncologist will know which treatment a participant is receiving. Of course if a patient truly needs active treatment, a placebo alone would not be given. It might be given in addition to a standard treatment, to see if the combination of the experimental treatment with the standard treatment is more effective than the standard treatment alone.


Phases of Clinical Trials

The four phases of clinical trials give researchers a systematic way to learn about and understand the treatments while keeping those patients participating as healthy as possible.

This is the first leg of a trial of treatment (drug or procedure) where the research is designed to establish the best dose, delivery and schedule for the new treatment. Safety and side effects of the drugs are very closely monitored. The number of patients in Phase I are typically small.
A greater number of patients are used with the treatment once it has reached this second phase. At this level, efficacy and safety of the drug is more closely monitored as to specific cancers. Side effects are evaluated as well.
Upon review of phases I and II, the primary attention given in Phase III is given to the risks versus the overall efficacy/benefit of the new treatment against the standard ones.

Trials in this phase continue to evaluate safety as well as considering the safety and efficacy of combining treatments – such as radiation and surgery or some other simultaneous procedures. If and when results of a Phase III trial proves the new treatment is deemed safe and beneficial – meaning it outweighs the risks – the FDA will then decide whether the treatment will be approved and made available to everyone.

Once the FDA approves a drug, an even larger number of patients can be included in this last phase – all of whom will be closely monitored for long-term side effects and therefore the relative safety of the new treatment.
Participation in Clinical Trials

Before you decide to participate, it is important to do your own research. Speak to other patients to learn about the pros and cons of volunteering for clinical trials.

brain tumor quoteNever trust your fears. They don’t know your strength.-Athena Singh
Choosing to participate is not only a proactive role on your part but could potentially benefit and save so many future patients suffering from brain tumors. It is suggested that if your physician does not recommend applying for a clinical trial that you seek a second opinion if this is a course of action you feel is right for you.

The potential risks are weighed against potential benefits. This is why a lot of patients are skeptical of signing up for these trials. But participation is always voluntary and the patient can always withdraw if they feel it best for them. Furthermore, the decision to volunteer or withdraw has absolutely no effect on the patient’s routine medical care.

Many patients recognize the potential benefits of a clinical trial but are frightened of the risks that may be involved. Patients sign an agreement stating that they know of the potential risks, but withdrawal from the trial is within their right at any point in time – whether it is because they are experiencing too many side effects or are just not happy with the treatment results.

Questions you may ask if you are considering participation in a clinical trial:

  • What is the purpose of the trial?
  • What kind of tests and treatments are involved in this trial?
  • What are the side effects/risks of this trial? The benefits?
  • How will I know if the treatment is effective and how will I get the trial results?
  • Will I have to be in the hospital or clinic often?
  • Is it permissible to speak with another patient who has been in the trial?
  • Am I assured privacy of my health information?
  • What happens if I decide to leave the trial?
  • Will insurance cover any of the costs of the trial?
Options to Consider

Provides free access to medical studies from hundreds of journals supported by unrestricted grants from several drug companies.

Brain Tumor Trials at Weill Cornell Medicine

Clinical Trials at Perlmutter Cancer Center

Clinical Trials Offered in the US

Clinical Trials run by MD Anderson

Clinical trials funded by Cure Brain Cancer Foundation.

Current Clinical Trials at Duke University

Current clinical trials at Duke University.

Current Trials at Johns Hopkins Medicine

Current clinical trials at Johns Hopkins Medicine.
Current Clinical Trials

Current Trials at Mayo clinic

Current clinical trials at Mayo Clinic.

Current Clinical Trials at the Cancer Research Institute

Current clinical trials at the Cancer Research Institute.

Lists more than 41,000 active industry and government-sponsored clinical trials; sponsored by company that recruits participants.

Mass General

Mass General Cancer Center provides access to a wide variety of clinical trials.

Medifocus Digest Alert on Glioblastoma

A free expertly selected listing of the latest medical journal articles published in Medline with direct links to the specific article abstracts focusing on cutting edge research, clinical trials, and the latest treatment advances.

The National Library of Medicine’s consumer site with a link to PubMed, its bibliographic database with citations and abstracts from nearly 4,500 journals world-wide.

Memorial Sloan-Kettering

Neuro-oncology Clinical Research and Trials

Research and Clinical Trials at Lenox Hill Hospital-Norwell Health

The Trial Matching & Referral Service

The Trial Matching & Referral Service at The University of Pennsylvania Cancer Center allows patients to complete a single questionnaire and determine their eligibility to participate in any ongoing clinical trials.

The trial listings site of the Center for Clinical Trials and Evidence-Based Healthcare at Brown University Medical Center.

Records and analyzes the outcomes of your treatments, enabling researchers to quickly identify which treatments or combinations of treatments look the most promising.
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