What is a clinical trial?
A sizable percentage of mesothelioma patients use clinical trials. Clinical trials are medical studies that involve people. First, researchers work for many years to understand the effects of a new drug or treatment on cancer cells in the laboratory and in animals. They also try to figure out the side effects it may cause. Usually after many years of research, doctors are ready to test the treatment in actual mesothelioma patients. This is called a clinical trial and there are three phases.
The goal of a phase I clinical trial is to show that the new drug or treatment is safe to use. Therefore, better survival is not the purpose, only safety. Mesothelioma patients who join a phase I clinical trials are often the first people to get a new therapy or drug.
In Phase I clinical trials, doctors typically increase the dose of the drug being studied in order to find the optimum dose with the fewest side effects. The first patients in the trial get a small dose of the drug. If there are no or few side effects, the next group of patients get higher drug amounts and so on. Doctors keep testing until they find the highest dose with the least side effects.
Phase I clinical trials often last several months to a year and may have only 10 to 30 mesothelioma patients. Doctors offer treatment in Phase I clinical trials to people whose cancers will not respond to standard treatments. If the treatment has acceptable toxicity then it may move to Phase II.
Phase II clinical trials
Phase II clinical trials focus on whether the new drug or therapy actually works. Doctors may measure the mesothelioma tumor size or analyze blood test results to learn if the treatment is effective.
Phase II clinical trials may last about two years and may have 30 to 100 mesothelioma patients. Sometimes, phase II clinical trials will assign patients to several possible treatments (called “randomized trial”). This may include standard treatment compared with the treatment under study in combination with the standard treatment. In other cases, patients may be assigned to receive different doses of the treatment.
If the Phase II trial demonstrates efficacy and manageable toxicity it may move to Phase III.
Phase III clinical trials
Phase III clinical trials compare the experimental treatment to the standard treatment to help determine if the new therapy is better. The goal is to determine if the new therapy or drug is superior to the standard treatment in respect to survival time, tumor response (if the cancer shrinks) or less side effects. Phase III trials gather data from larger numbers of mesothelioma patients, perhaps 80 or more.
Phase III clinical trials are often randomized. Mesothelioma patients may be randomly sorted to receive the new treatment or the standard treatment.
Once the research suggests that the new treatment works well against a specific cancer, researchers can apply for FDA approval. If approval is granted, the treatment can be used in the larger population of mesothelioma patients.
Getting Experimental Drugs Outside Clinical Trial
Sometimes an experimental drug can be used outside of a clinical trial. The advantage of this is that the patient actually gets the drug (is not randomized) and other selection criteria may not be involved that could exclude the patient from the trial. This is referred to as compassionate use. But getting access to drugs that are not FDA approved for mesothelioma can be a difficult process. Another possibility is to contact the manufacturer of the drug directly. Sometimes they have a program in place where the drug may be available outside their clinical trial. Finally, doctors have the latitude to use drugs that are already FDA approved for one disease in another disease. For example, if the drug is already FDA approved for one cancer, your doctor can use it in mesothelioma. This is called off-label use. However, keep in mind that off-label use of drugs is not always covered by insurance.
Risks & Benefits of Clinical Trials
Clinical trials help doctors determine if newer therapies work. But a mesothelioma patients wants to be careful before they bet on a new therapy because they are literally betting their life. Therefore, keep in mind that there are risks and benefits to enrolling in clinical trials and using experimental drugs.