Straight Talk about Mesothelioma, a blog series created by Michael T. Milano, M.D., Ph.D., a radiation oncology specialist, as a resource for mesothelioma patients and their loved ones.


A clinical trial is a type of research that involves human participants to study any aspect of medicine. Clinical trials may help us understand how to diagnose a disease, or how to prevent or treat it. They can also look at whether or not a specific drug is effective or safe to use. Each study answers specific questions and tries to find better ways to prevent, diagnose, or treat a disease. In other words, clinical trials use people, data, and numbers to find ways to better manage patients.

In some clinical trials, all groups receive the same type of care, and the goal of the study is determining how effective that intervention is (with the intervention perhaps being a new drug, a dietary change or a specialized diagnostic test).

In other types of clinical trials, volunteer participants are placed in groups with the goal of comparing outcomes between those groups. One group receives an intervention while another group receives a different type of treatment or no treatment at all. There can be many groups comparing many different things. And with at least 2 groups, researchers can compare how 1 type of care works compared to another. With a brand new drug or therapy, some studies are designed such that all patients get that new drug or therapy, but at different dose levels to determine how well it is tolerated (called Phase I studies).

The length of a clinical study varies, from weeks or months, to several years. It all depends on what is being studied and how complicated it is. Each study has a team of researchers. These may include doctors, scientists, nurses, social workers, and other medical professionals.

When a new product is studied, such as a new medication for pleural mesothelioma, it is not known whether it is helpful or harmful until it is tested in clinical trials. As an example, researchers had to study whether having surgery or taking medicine was more effective for certain patients with pleural mesothelioma. There are many things that clinical trials in mesothelioma observe, from very broad to very specific.

The Importance of Clinical Trials in Medicine

In the medical field, it is extremely important to have data from clinical trials. Picture this: a clinical trial shows that drug A lowers blood pressure 25 percent in 2 weeks, while drug B lowers it 5 percent in 2 weeks. Which is more effective? The numbers say Drug A. Doctors can then look at safety and other results from clinical trials and make the best decision on how to treat a disease.

“…it is clear how both preclinical and clinical researches aimed at identifying new therapeutic targets and testing them in early clinical settings are badly needed.”

– J. Pinton, MD

The desired end result of a clinical trial is to improve a doctor’s knowledge on how to manage patients. Clinical trials help the medical community study different types of care for diseases and provide patients with the best possible treatment.

With treatment that is based on results from clinical trials, patients can be confident that they are receiving the best possible treatment. This is because they know that it has been tested for safety and efficacy before they receive it.

Safety & Efficacy: A Critical Driver of Clinical Trials

Clinical trials are used to show us what works and what doesn’t work. In cancer research, including the study of mesothelioma, they are the ultimate way to learn what works best for patients. The 2 most important questions that a clinical trial will answer are:

  1. Does the new treatment work? If it doesn’t work, doctors can move on to something else. If it does work, doctors can look at how well it works. They also consider other factors such as side effects, cost, and how often a patient has to take a certain treatment.
  2. Is the new treatment safe? Even if a treatment works very well, if it is not safe, it can be very dangerous and may not be used. Measuring safety is very important in clinical trials.

When a new drug is discovered or created, it must be purified and rigorously tested in labs. This includes testing on other mammals – like mice – before testing on humans. Very few drugs actually pass this point. But when they do, they are then tested in humans. Amazingly, a new drug for cancer has to be studied for around 6 years before it even makes it to testing on humans. Even more amazing is that it takes an average of 8 more years of human study before it is approved for use.

The reason it takes so long is that researchers need to be absolutely sure it is safe and effective. And in cancer, it can take months, if not years, to see if a specific treatment actually works. As a result, you can be sure that every drug has been through an extensive process with many studies and reviewers looking at its safety and how well it works.

Clinical Trials in Mesothelioma

In mesothelioma, as in other areas of medicine, clinical trials are very important. Mesothelioma is caused by asbestos exposure, which usually occurred many years ago. It often takes 20-50 years for it to appear. Researchers suggest that the incidence rate of mesothelioma will peak in the next few years. In other words, more and more people exposed to asbestos years ago will be diagnosed with mesothelioma in the coming years. Data from clinical trials will be very important to help doctors manage these patients.

At this time, there are many clinical trials happening in mesothelioma. Some of these include:

  • A study looking at a new drug – Keytruda® (pembrolizumab) – in patients with malignant mesothelioma.
  • A study looking at genes and how they may be related to developing mesothelioma.
  • A study looking at using a type of virus to actually treat mesothelioma.

What patients and their families need to understand is that these clinical trials – and many others – are designed specifically to zero in on and improve the care of patients with mesothelioma. Clinical trials are paving the way for better mesothelioma treatments, which can hopefully lead to longer and better lives for patients suffering from this disease.

Participating in a Clinical Trial

Understanding what a clinical trial is can help patients decide if they may want to participate in one and it can also help doctors better understand the disease. In many cases, researchers seek participants who have a specific illness (patient volunteers) while others look for those who are not sick (healthy volunteers).

Patient Volunteer
Patient volunteers have a known health problem. For example, a patient with pleural mesothelioma may volunteer to have his/her cancer studied in a clinical trial. He or she would participate in the trial to help researchers better understand, diagnose, treat, or cure mesothelioma.

Healthy Volunteer
Healthy volunteers play an important role in research, too. Healthy volunteers are often compared to those who are sick to see how a specific treatment works. They are often “matched” to a sick patient on characteristics such as age, gender, or family history. In many cases, they receive the same test, procedure, or drug that the sick patients receive. By doing this, researchers can tell whether the drug works specifically in sick people.

Unfortunately, less than 5 percent of adults with cancer will take part in a clinical trial. On the other hand, 60 percent of children under age 15 do. This is partially why children with cancer are living much longer than they used to. Lots of young volunteers in clinical trials have helped doctors better understand childhood cancer. Rates have not improved as much in adults.

One sad truth is that many people don’t realize clinical trials are an available option for them. Others want to take part but don’t meet the requirements. Some are uncomfortable with participating in a trial and others worry that they could be harmed by an unproven treatment. Even so, many patients with certain diseases are interested when asked. They want to help scientists better understand diseases by being involved in clinical trials.

Clinical trials in mesothelioma are difficult since there are 3 main types of mesothelioma – and the disease is incredibly rare. Much rarer than, say, diabetes or high blood pressure. As a result, it is sometimes difficult to recruit enough people with mesothelioma to participate in clinical trials. That makes it even more important that a patient with mesothelioma consider entering into a clinical trial to help improve ways that doctors treat the disease.

If you or someone you know is interested in participating in a clinical trial, talk to your doctor. He or she can help explain to you what is involved and why they are so important.