Clinical trials often provide the best chance of survival for a patient with malignant mesothelioma, offering access to the latest, cutting-edge treatments at a leading specialty center.
They also create hope for the future and make progress possible, fueling the belief that a cure can become a reality. Joining a clinical trial might be the most important step any cancer patient can take — a leap of faith that goes well beyond one’s own best interests.
Participants have been defined as heroes, paving the way so others can benefit, along with helping themselves.
“Participation of patients with mesothelioma in clinical trials is essential if we are going to make progress and develop novel therapies,” said Raffit Hassan, M.D., a senior investigator at the National Cancer Institute.
If we keep using the same treatments, we'll never find a better one. Patients usually hope the newest one works better. If you do nothing, no progress will be made.
What Are Clinical Trials?
Clinical trials are research studies that involve people.
Each one has a well-defined and distinct purpose. They often involve the newest drugs, the latest therapies and the most up-to-date treatment procedures. Many of them provide access to potentially curative measures that are working their way through the decade-long and tedious approval process from the U.S. Food and Drug Administration (FDA).
With an aggressive cancer like mesothelioma, patients can’t afford to wait for approval, making these trials the best hope for survival. The majority of oncologists agree, for example, that future treatment with many cancers will involve immunotherapy drugs, which still are awaiting FDA approval.
Immunotherapy, the subject of many mesothelioma clinical trials, involves triggering a body’s own immune system to fight off the disease instead of the toxic treatments like radiation and chemotherapy.
These clinical trials help doctors measure the effectiveness, and get a better picture, of new therapies. They can be sponsored by the National Cancer Institute, by Specialized Programs of Research Excellence or by Community Clinical Oncology Programs. They often are conducted concurrently at multiple cancer centers across the country.
The Clinical Trial Process
There are three phases of clinical trials that every new drug or therapy must pass through before it reaches the FDA for possible approval. Each phase can take several years to finish.
- Phase I
A Phase I trial usually involves checking for safety. It includes only a small group of patients (20 or more) looking to see what side effects come with the drug. It helps to establish dosage levels, and analyzes how well the body can safely process the drug.
- Phase II
A Phase II trial takes a closer look at safety and dosage levels, but now is looking to see how effective the treatment or drug will be. It can include up to 300 patients. It may involve a placebo or a comparison to other treatment options.
- Phase III
A Phase III trial precisely measures the effectiveness of the treatment when compared to the traditional approach. It could involve thousands of patients. This trial is used to again analyze side effects and finalize dosage levels.
Each phase, and each trial, has a list of qualifications that must be met before a patient is accepted. Depending on what is being tested, some trials involve only patients who already have had surgery. Some want patients who have had no treatment. Others want patients who are at different stages of their treatment.
Because mesothelioma lacks a definitive cure and is likely to return after treatment, researchers are always striving to discover newer and better therapies.
- WT1 The Wilms’ tumor vaccine
The Wilms’ tumor (WT1) vaccine is being tested for its ability to stop mesothelioma recurrence after surgical resection in a Phase II trial. The early returns have been promising. To qualify, patients must have completed a multimodal therapy that included either a pleurectomy/decortication or an extrapleural pneumonectomy. MD Anderson in Houston is conducting the clinical trial. This is an immunotherapy trial that involves giving the patient six injections over a 12-week period.
- VS-6063 Stem cell inhibitor defactinib
Stem cell inhibitor defactinib (VS-6063) is being tested in a multicenter, multinational Phase II clinical trial that has researchers around the world excited about the possibilities. Although stem cells represent only a fraction of the cells in a tumor, they are the major reason for cancer recurrence. This is the first time a particular protein (FAK) within the stem cells has been targeted in a mesothelioma trial. It is designed to prevent recurrence after chemotherapy. Centers in Cleveland, Chicago, Baltimore, Dallas, Philadelphia and New York are conducting the research in this country.
- CRS-207 Phase II cancer vaccine
Cancer vaccine CRS-207 is a weakened form of listeria that has been genetically modified and proven to stimulate an immune response against tumor cells. This is a Phase I trial and a vaccine that will be used alongside standard chemotherapy (pemetrexed and cisplatin). It is designed to prompt an immune response against the antigen mesothelin, which is present at higher levels on certain tumor cells than on normal cells. This trial will evaluate both the safety and response of the vaccine. It is being tested in Tampa, San Francisco, Chicago, Philadelphia and Bethesda, Maryland.
- Tremelimumab Immunotherapy drug
Researchers are hoping for an estimated 600 patients with unresectable pleural or peritoneal mesothelioma for a Phase II clinical trial involving tremelimumab, another immunotherapy drug. It is a double-blind, parallel-group study and half the participants will receive a placebo. Both study groups also will receive standard chemotherapy treatment. Tremelimumab has been successful with other cancers, but has never been tried before with mesothelioma. There are more than 125 sites around the world participating in the study, including 28 centers spanning 18 different states in the U.S.
- Measles Virus Genetically altered to destroy tumor cells
A genetically altered measles virus is being tested in a Phase I clinical trial for its ability to destroy mesothelioma tumor cells, without harming the healthy cells. Previous studies have proved effectiveness against both prostate and ovarian cancers. The trial is a joint effort between the National Cancer Institute in Bethesda, Maryland, and the Mayo Clinic in Rochester, Minnesota. The investigation is looking to help determine proper dosage levels by measuring side effects.
Not everyone is eligible for clinical trials. Those who can join are strongly encouraged to participate. Mesothelioma specialists can help you determine your eligibility.
- Past Treatment History Previous therapies affect current options
Some trials test therapies that must be given before any other treatment, which means patients who had prior treatment would not qualify. Others seek previously treated patients to test how well a new chemotherapy drug works right after an earlier treatment like surgery or radiation therapy. Patients in either situation have options.
- Stage and Type of Mesothelioma The location and extent of disease
Because the standard treatment plan differs greatly depending on where the mesothelioma formed and how far it has spread, type and stage are important factors for recruitment. A trial may explore new treatments for a single type of mesothelioma, or only recruit patients diagnosed at a specific stage of disease.
- Age and Gender Trials for specific patient groups
The age and gender of participants is important to researchers because the disease affects these groups in different ways. Most studies will accept patients of either gender that are 18 years and older, but there may be special trials only for women or older patients.
- Overall Health Choosing patients healthy enough for testing
Your overall health (aside from the cancer) can have a major impact on the treatments you qualify to receive. Before the trial, researchers must determine your performance status, a measure of your general well-being and how well you can take care of yourself. Some trials are only safe for early stage patients who are relatively healthy, while others focus on bed-bound patients with advanced disease.
- Other Medical Conditions Pre-existing health problems can increase risk
Because the average mesothelioma patient is 60 years old, many people with the disease also suffer from a chronic medical condition like heart disease or hypertension. Unfortunately, the presence of these conditions may increase the risk of complications and death during the study. Most trials exclude patients with certain conditions to ensure their safety.