The Brain Tumor Center at Johns Hopkins | Clinical Trials for brain cancers, new drugs for brain cancers

Clinical Trials

The Role of Research in Treating Malignant Brain Tumors
Definition of Clinical Research

The Benefits of Participating in Clinical Research

When to Participate in a Clinical Research

Current Clinical Trials at The Brain Tumor Center at Johns Hopkins

The Role of Research in Treating Malignant Brain Tumors: There have been tremendous advances made in the last two decades in the battle against malignant brain tumors. Advances in neuroradiology have lead to more timely and accurate diagnoses, improved neurosurgical approaches have permitted more complete and safer tumor removal and new chemotherapies and radiation therapies have extended survival.

The Brain Tumor Team at Johns Hopkins is proud to offer state of the art care for patients with malignant brain tumors. We are achingly aware, however, that despite the advances in radiology, surgery, radiation and oncology, malignant brain tumors are aggressive and remain deadly tumors. This is painfully illustrated by the fact that the average survival for the most common primary malignant brain tumor, glioblastoma multiforme, remains only 1-2 years despite maximal therapy.

Clearly, we need to do much more. At Johns Hopkins Hospital we believe that well designed and carefully conducted research will lead us to the next major breakthrough in the treatment of patients with malignant brain tumors and eventually lead to cures. Research takes many forms. Many of the ideas advanced for treating brain tumors start in basic science laboratories on the cellular and molecular level. These ideas are then typically tested in animals and, if they show promise, are then advanced to studies in patients. Johns Hopkins benefits from a long and rich tradition of collaboration between basic scientists and clinical scientists, which allows for efficient advancement of new ideas from the laboratory to patients.

Definition of Clinical Research: Clinical research is any research done with human subjects (patients) or any material collected from human subjects (such as tissue samples, blood, radiograph images or test scores). This is also called “patient-oriented” research, as patients are the focus of the studies. It is a powerful tool for understanding the mechanism of diseases; determining how diseases affect people in various physical and emotional ways; developing more accurate diagnostic techniques; testing new treatments; and assessing the success of various diagnostic tests and treatments. There are two main types of clinical research:
Observational -- samples are collected and investigators look for patterns. These studies tend to require very little patient sacrifice. For example, a study may require blood to be drawn once a month
Interventional -- this requires an intervention of some type, such as a new treatment, test or surgery

Informed Consent: The key to all forms of clinical research is that the patient is the central focus of the research effort and must be willing and able to fully participate. For all clinical research, an essential component of the process is informed consent. This ensures that the patient-participant has a thorough understanding of the proposed research, the possible risks and benefits and freely gives their consent to participate.

Clinical Trials: Clinical trials are an important form of clinical research. They are designed to test new therapies or diagnostic techniques in patients. In general, clinical trials address whether a new treatment or technique is safe and more effective than existing therapies. Clinical trails are an enormously important weapon in our arsenal against malignant brain tumors.

There are four stages to clinical trials:

Phase I trials test a new treatment in a small number of patients to evaluate its safety. They are used to determine things like the safest dose of a drug, identify side effects or determine if a diagnostic test is safe. These trials are a required first step for any new therapy.

Phase II trials study if the intervention is effective. In other words, does the drug or test work as intended. Phase II trials include a larger group of patients than phase I trials. Data about safety is collected throughout a phase II trial to ensure there are no previously unrecognized side effects or toxicities.

Phase III trials require a much large number of participants and are designed to test whether the new therapy works as intended and how it compares to existing therapies. Information gathering about side effects and safety is continued throughout this phase. These are typically large studies that involve several medical institutions. Only the most promising therapies progress to a phase III trial due to the intense effort, time, expense and patient involvement they require. If a phase III trial is positive, the drug or therapy is submitted to the Food and Drug Administration for approval and the drug is marketed.

Phase IV trials are designed to monitor the effectiveness of a drug after it has been approved and marketed to ensure it continues to have the effects intended and to monitor for any previously unrecognized side effects.

The Benefits of Participating in Clinical Research: First and most importantly, the providers at Johns Hopkins are dedicated to providing the very best care possible to all patients -- regardless of their interest in participating in clinical research. There is absolutely no requirement to participate in research. We feel strongly that research is our best hope for advancing treatment, and eventually finding a cure for brain tumors. We are therefore committed to performing high quality, comprehensive clinical research.

You are likely to be offered research opportunities at some point during your care at Johns Hopkins. In fact, patients frequently come to us expressly to be involved in our research efforts. Many patients tell us they greatly enjoy their involvement in clinical trials. Often, patients feel they get even more attention and oversight of their care while they are in a clinical trial since there are frequent designated evaluation points over and above standard clinical assessments. Moreover, there are entire staffs appointed to work with patients in clinical trials in addition to the clinical staff coordinating the overall care for the patient. Finally, the diagnosis of a malignant brain tumor is often emotionally devastating and patients may feel that they are being proactive and contributing to a greater good when they participate in a clinical trial.

As mentioned, there are several clinical studies that are observational. In these studies the commitment on the part of the patient is generally minimal. Patients can frequently participate in many of these studies simultaneously. The benefit of participating in these studies is that you are able to add to our knowledge about a rare disease with relatively little inconvenience to you. While these types of studies are unlikely to offer direct benefit to your disease at the time of your involvement, the information gained may be used to develop new therapies to improve survival or quality of life for you or other patients like you in the future.

Involvement in clinical trials generally requires more patient commitment and has more associated risks. Hence, we tend to offer clinical trials to patients who have had recurrence of a tumor that we know to be aggressive and generally incurable with current therapies. It is worth mentioning that some patients wish to start their therapy with experimental agents since there are currently no widely curative treatments for tumors such as glioblastoma multiforme and we offer trials to support this interest.

When to Participate in a Clinical Research: We invite you to ask your physician or any of our research coordinators about our current clinical trials. It is advisable to learn about available clinical trials as soon as possible after a diagnosis is made. This provides you the most possible options to consider while working with your physicians to plan your treatment. Many trials have restrictions that may limit patient involvement after they have started one form of therapy or another. However, due to the number of clinical trials available at Johns Hopkins, we frequently have clinical research options for patients in all stages of their disease.

Current Clinical Trials at The Brain Tumor Center at Johns Hopkins: Johns Hopkins is the coordinating site and a participant in the New Approaches to Brain Tumor Therapy (NABTT) CNS Consortium. The NABTT Consortium is made up of five academic institutions. Founded in 1994, their goal is to improve the therapeutic outcome for adults with primary brain tumors. It is one of two nationwide consortiums that is funded by the National Cancer Institute to conduct Phase I and II clinical trials of promising new treatment strategies. Many of the clinical trials offered at Johns Hopkins are offered as part of the NABTT Consortium. NABTT trials are categorized based on the stage of disease:

Class A and B studies refer to newly diagnosed glioblastoma multiforme
Class C studies refer to recurrent high grade astrocytomas (with measurable disease)
Class D studies refer to locally recurrent high grade astrocytomas
Class E studies address all other brain tumors (non-astrocytomas)
Class F studies are observational, “non-therapeutic” protocols.

Please refer to the NABTT website for currently available trials as availability changes frequently. http://www.nabtt.org.

Additional trials are offered at The Johns Hopkins Hospital independently of NABTT. These trials also have frequently changing availability. Please contact one of the research coordinators below for up-to-date information.

Kelly Szajna, RN
Johns Hopkins Hospital
Hopkins NABTT Program Coordinator
550 Building, Suite 1001
550 North Broadway
Baltimore, Maryland
410.502.5343
kszajna1@jhmi.edu

Bing Coa, MD
Johns Hopkins Hospital
Hopkins Brain Cancer Program Research Coordinator
550 Building, Suite 1001
550 North Broadway
Baltimore, Maryland
410.955.8837
byao1@jhmi.edu

Deanna Glass-Macenka, RN, BSN
Surgical Research Nurse
Johns Hopkins Hospital
Department of Neurosurgery, Meyer 9-100
600 N. Wolfe Street
Baltimore, MD 21287
410.502.8273
dglassma@jhmi.edu

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