CLINICAL TRIALS

New medical treatments must be proven safe and effective before they can be offered to a large number of patients.

Clinical trials are procedures in medical research conducted to evaluate the safety and information about adverse drug reactions and adverse effects of other treatments. The procedures in a clinical trial must follow a pre-defined protocol.  Clinical trials can only take place after acceptable information has been gathered on the quality of the non-clinical safety, and Health Authority/Ethics Committee approval is granted in the country where the trial is taking place.

Clinical Trial Participants

Individuals participating in clinical trials gain access to new research treatments before they are widely available.  Participants in a clinical trial can be either healthy or suffering from an illness, but must qualify and meet a certain criteria. These criteria are based on such factors as the type and stage of a disease, previous treatment history, age, gender, and other medical conditions.

Interventional and Observational Studies

There are two types of studies conducted at a clinical trial, interventional and observational.  Interventional studies are those in which the participants are assigned by an investigator to a treatment or other intervention, and their outcomes are measured.  Observational studies are those in which participants are observed and their outcomes are measured by the investigators.

Clinical Trial Location and Size

Clinical trials can take place in a variety of locations, such as hospitals, universities, doctors' offices, or community clinics. The size of a clinical trial varies from a single center in one country to a multicenter trial in several countries.

Clinical Trial Funding

Clinical trials are funded by individual physicians, organizations, medical institutions, foundations, voluntary groups, and pharmaceutical companies.  In addition, clinical trials can be sponsored by federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD), and the Department of Veteran's Affairs (VA).

Clinical Trial Phases

There are four phases conducted in a clinical trial.  The trials at each phase have a different purpose and help scientists answer different questions.

In Phase I trials, researchers test an experimental drug or treatment in a small group of 20 to 80 participants for the first time to evaluate its safety, how the drug or treatment effects the body, determine a safe dosage range, and identify side effects.

In Phase II trials, the experimental study drug or treatment is given to a larger group of 100 to 300 participants to distinguish if it is effective, identify common short-term side effects, and to further evaluate its safety.

In Phase III trials, the experimental study drug or treatment is given to large groups of 1,000 to 3,000 participants to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the experimental drug or treatment to be used safely. The results from phase III are used to make recommendations to healthcare providers about the use of the drug or treatment in all patients.

In Phase IV trials, post marketing studies outline additional information including the drug's risks, benefits, and optimal use.

Clinical Trial Results

The results of clinical trials are often published in peer-reviewed, scientific journals. Peer review is a process by which experts assess the report before it is published to make sure the analysis and conclusions are sound. If the results are particularly important, they may be featured by the media, discussed at scientific meetings, and by patient advocacy groups before they are published. Once a new approach has been proven safe and effective in a clinical trial, it may become a widely accepted approach.

BURZYNSKI CLINICAL TRIALS DATA

Results of Brain Tumor Trials

NOTE: Most patients who come to Dr. Burzynski already have failed at least one type of therapy and many have failed more than one type. Many of these patients are considered to be in the final stages of cancer and have no remaining conventional treatment options available to them.

Over 100 clinical trials using different chemotherapy regimens have been conducted on recurrent brain tumors. The general consensus is that such tumors cannot be cured by chemotherapy, and the response rate is only modest. Complete responses are almost never seen, and the total response rate usually includes stable disease. Even though some of these studies report significant responses, the durations of improvement are short. Ultimately, almost all of these patients die from their brain tumors. Patients who are being treated with antineoplaston therapy often are the exception.

Antineoplastons have been used to treat neoplastic disease since the 1970s. Patients can tolerate administration of antineoplastons very well, for as long as approximately 10 years in one case.

The Burzynski Research Institute have different Phase II clinical trials with Antineoplastons A10 and AS2-1 in several forms of cancer. Five of these trials have reached the final point and proved antincancer activity in various types of malignant brain tumors.

Adverse Reactions

Almost all patients experience increased urine output and slight thirst. The treatment usually is free from adverse reactions or is associated with mild side effects. Moderate side effects (Grade 2 by NCI criteria) include fluid retention, hypernatremia, hypocalcemia, hypokalemia, hypomagnesemia, nausea and vomiting, elevation of SGPT, leukopenia, allergic skin rash, fever, chills, headache, tinnitus and decreased hearing, decreased and blurred vision.

The Burzynski Research Institute - Clinical Trial Phase III

The Burzynski Research Institute is sponsoring a Phase III clinical study in diffuse intrinsic brainstem gliomas.

Brainstem Gliomas constitute approximately 10% of all primary brain tumors and are diagnosed primarily in children. The diffuse intrinsic brainstem gliomas (DBSG) is the most common type of gliomas of the brainstem and occurs in approximately 85% of cases. DBSG are inoperable and the only palliative treatment recommended currently is standard radiation therapy, which offers 2 year survival prognosis to only 7% of the patients. Cytotoxic chemotherapy is not effective and the recently introduced targeted therapies did not provide better results. The prognosis is especially dismal for recurrent DBSG with estimated survival of less than 6 months, regardless of the treatment used (S.R. Burzynski: Recent Clinical Trials in Diffuse Intrinsic Brainstem Glioma. Cancer therapy Vol 5, 379-390, 2007).