With rare cancers like pleural mesothelioma, it can often take years, sometimes more than a decade, for a new drug or therapy to receive approval from the U.S. Food and Drug Administration (FDA). The reason for this is that the FDA requires a certain number of clinical trial participants to take part in a study, while the rarity of the diseases means there are not many people who are eligible for the trials.
Researchers have yet to discover a cure or ideal way to manage pleural mesothelioma. Traditional treatment options have had limited successes, often extending survival by a few months and sometimes years. Clinical trials allow patients to gain access to cutting-edge treatments such as gene therapy and immunotherapy. Many oncologists believe that these treatments have a lot of promise for prolonging the life of mesothelioma patients. Without joining a clinical trial, patients today will have no access to these types of therapies.
The FDA requires that testing for all new medical treatments or drugs be carried out in three distinct phases of clinical trials before they can receive approval.
This phase focuses on determining correct dosage and relative safety of the drug or treatment in question. It typically involves a small number of participants. Researchers want to see how humans react to the drug and examine the side effects. The first participants start with a low dose to minimize the risk of adverse side effect. If the low dose is deemed safe, the dosage is gradually increased to find an optimal level of effectiveness.
During this longer phase, researchers focus on how the disease responds to the treatment. Trial subjects are given the highest dose that previously was determined to be safe. Patients are carefully monitored throughout this phase to determine the effectiveness. Their overall health is also monitored to determine any adverse side effects. This phase includes a larger sampling of patients.
During this phase, the drug or therapy is tested against an existing treatment for the disease. Researchers want to know if Drug Y works better than Drug Z in treating the disease. This phase normally includes the largest number of participants. Patients commonly are divided into two groups, with only one receiving the drug being tested. The other group – known as the “control group” – receives the standard treatment. These are often considered “blind” studies because patients do not know if they are getting the experimental treatment or the already approved treatment.
Throughout all phases of clinical trials, the disease is carefully monitored. Researchers are required to stop the trial if any of the participants suffers from severe side effects. The study also may be suspended if one of the groups suddenly shows dramatic improvement compared with the other group.
Not all pleural mesothelioma patients qualify for a clinical trial. Each trial has specific eligibility requirements. There are both inclusion and exclusion criteria that allow or disallow a person from participating.
Treatment history, physical health, stage of disease and exact cell type of pleural mesothelioma, along with age and even gender, can determine someone’s eligibility for a clinical trial. Some trials seek patients who have not been treated yet, while others want patients who have had specific treatments. Nearly all studies require participants to be in overall good physical health and some studies will exclude current or former smokers. Most studies exclude people with heart and cardiovascular disease, psychiatric disorders, bodily infections, liver or kidney disease, low blood cell counts and history of another recent cancer.
Participation is voluntary, and patients should consult closely with their doctor before joining a clinical trial. Participants are encouraged to fully understand the trial before beginning it. They are permitted to withdraw from the trial at any point, for almost any reason.
Questions to Ask About Clinical Trials
While some patients in a clinical trial for pleural mesothelioma can benefit greatly, others may not, and will leave without a change in their prognosis. Even if a patient doesn’t benefit personally, they usually leave a trial with the satisfaction that they are helping future patients and the search for better treatment.
Because pleural mesothelioma is such a rare cancer – an estimated 2,000 to 3,000 people in the U.S. are diagnosed annually – participation in trials is critical for researchers to make advancements in treatment.
It often is difficult to recruit enough patients to complete trials for rare cancers. The National Cancer Institute (NCI) estimates that only 5 percent of cancer patients participate in a clinical trial.
“For studies to produce meaningful results, you need a large number of people involved,” said Cary Gross, M.D., Yale University associate professor of medicine. “That’s why it’s so important for patients to participate. It’s critical for a rare cancer like mesothelioma.”
Part of today’s standard treatment for pleural mesothelioma, a combination of chemotherapy drugs Alimta (pemetrexed) and cisplatin, stems from a clinical trial that finished in 2002. Research then reported a 25-30 percent survival improvement for patients who received the two-drug chemotherapy combination, which led to approval.
Finding a trial that fits your particular need isn’t always the easiest task. There are more than 100 ongoing trials today for pleural mesothelioma, spread across various medical centers and in various phases. Some are actively recruiting new patients; others are in a follow-up stage.
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