Reliable Cancer Therapies

When researchers think that they have found a promising treatment they do not immediately treat patients with it. If it is a brand new treatment, they will first test it in the laboratory, usually on tumor cells. If results in the laboratory are encouraging, they will test the treatment on animals. These studies are highly regulated to avoid unethical tests on animals. If results of the tests on animals are encouraging, researchers will set up, together with doctors, the first step of the experimentation of the new treatment on humans. This first step in humans is called a phase I clinical trial. Phase II and phase III clinical trials will follow if results are encouraging during the previous step. Thus, the risk for participants is higher in phase I than in phase II, and risks of a phase II trial will be higher than in phase III.

Sometimes, the treatment tested is not new, but the way it is given, the dosage or the combination with other treatments differ. It may also be a treatment already used for one type of cancer. Before using it for another type of cancer, however, a new study has to be done to see whether the treatment is safe and works for the new type of cancer.

Clinical trials are highly regulated by national or international authorities because the experiments are done in humans. The main goal of this regulation is to protect people participating in clinical trials, to ensure that patients are informed of the possible risks and benefits of participating, and also to make sure that a relevant conclusion can be drawn at the end of the clinical trial to avoid that a study has to be repeated. An institutional review board, sometimes called ethical committee, will check if everything is in order to ensure patients’ safety and confidentiality. They will check if the information provided to participants is complete and understandable. Depending on the national laws, such a board includes scientists, doctors, lay-persons and a clergy member.

Phase I clinical trial
If the results of tests of the new treatment in laboratory and on animals are promising, the new treatment will be given for the first time to humans during a phase I clinical trial. For cancer treatments, a phase I clinical trial usually presents the following characteristics:
- The new treatment is given to a small number of patients, usually around 20-30
- The patients invited to participate are usually patients running out of treatment options
- The goal is to find the best way of giving the treatment and the highest dose that can be administered without being harmful. For drugs, ascending doses are given and side-effects are carefully watched
- The risks are important and the benefits are not guaranteed. The goal is not to see if the treatment can work. However, some patients have been helped by receiving new treatments in phase I clinical trials.
Thanks to phase I clinical trials, researchers and doctors will know the best dose, the best way to give it and whether the treatment is safe.

Phase II clinical trial
If the treatment has been shown to be safe in the phase I clinical trial, a phase II clinical trial can be implemented. For cancer treatments, a phase II clinical trial usually presents the following characteristics:
- The new treatment is given to a limited number of cancer patients, usually between a few dozens to 100
- The treatment is usually given to all patients but in some clinical trials, participants are randomly assigned to receive the new treatment or not.
- The goal is to find evidence that the treatment works. This will be evaluated by how the cancer reacts to the treatment. When the tumor is not growing, or even shrinking or disappearing, it is assumed that the treatment works.
- The side effects are also carefully watched to make sure that benefits of the treatment are higher than risks.
Thanks to phase II clinical trials, researchers and doctors know if the treatment works for one or for several types of cancer.

Phase III clinical trial
With the information given by the phase II clinical trial, a phase III clinical trial can be implemented. The new treatment seems to work, but it has to be compared to the current standard treatment to see if it has any added value for patients. A phase III clinical trial presents the following characteristics:
- The study involves a large number of patients, usually hundreds or thousands
- Two treatment groups are usually defined and participants are randomly assigned to one of the 2 groups. In addition to the current standard of care, patients in one group do not receive the new treatment while patients in the other group do.
- When the new treatment is a drug, patients who do not receive the new treatment receive a placebo looking like the new treatment. Whatever the group they belong to, patients do not know if they receive the new treatment or a placebo. Doctors taking care of the participants do not know whether one patient receives the new treatment or a placebo. This is defined as double-blinded, meaning that patients and doctors are blinded regarding the treatment. When patients and their doctors know what treatment they receive, they often are more confident in the chance of success if they receive the new treatment than if they receive the placebo. This can compromise the results of the study.
- To evaluate the efficacy of the new treatment, doctors will look if the tumor is not growing or even shrinking or disappearing. But to conclude on the efficacy of the new treatment, the researchers will look if patients receiving the new treatment live longer than patients not receiving it. Side-effects will also be carefully watched to see if they do not counterbalance the potential benefits of the treatment.

Thanks to phase III clinical trials, researchers and doctors know if the treatment has an added value in comparison to the current standard of care. If it has an added value, the results of the study will be analyzed by authorities in charge of the approval of medicines, mainly the European Medicines Agency or EMA in the European Union and the Food and Drug Administration or FDA in the Unites States of America. The EMA and FDA will decide if the treatment should be available and for which type of cancer. In Europe, each country decides if the new treatment will be covered by health insurance.

What are the main possible benefits of participating in clinical trials?
- Participants have access to a promising treatment which is not available elsewhere
- The new treatment may have a higher efficacy than the standard treatment
- Participants will be watched carefully by health professionals according to a protocol
- The clinical trial is an experiment and its results will be helpful for all. It will either allow other patients to benefit from a new and useful treatment or avoid researchers to continue their research on a useless or harmful treatment. The results will also help researchers to understand cancer better.

What are the main possible risks of participating in clinical trials?
- The new treatment may not have any added value
- The new treatment may have some side-effects that can range from minor and reversible side-effects to life-threatening side-effects. Some side-effects can also appear weeks, months or even years later and include damages to important organs such as heart or kidney or the development of a second cancer
- In phase III clinical trials you usually do not know if you receive the new treatment or not.

Where do clinical trials take place?
Clinical trials take place in all kinds of hospitals and clinics, but mostly in academic hospitals. They are organized by researchers and doctors.

Where does the money come from?
The money to organize clinical trials comes from different sources, usually pharmaceutical companies, public funds from either university or national or international institutes or private funds from non-profit organizations.

What is the typical development of a clinical trial?
Usually, doctors propose their patients to participate in a clinical trial either right after the diagnosis or later during the treatment. Patients can also tell their doctors that they want to participate in clinical trials or can search for information on clinical trials that are recruiting patients.

Before participation, 2 major things have to be done:
1)  Each clinical trial has its own criteria and all criteria must be respected, otherwise the participation in the clinical trial is not possible. Your doctor will have a checklist and will verify if all points can be ticked for you. Sometimes, this requires additional biological or radiological examinations.
2)  You will have to give your informed consent before participating. Informed consent means that you agree to participate and have been informed of the goal of the clinical trial, its risks and your rights. Your doctor will explain this to you and will give you written information. Once you agreed to participate in the clinical trial, your doctor will ask you to sign an informed consent form. Throughout the study, you will be informed of all relevant information on the safety and efficacy of the treatment. One of your most important rights is to leave the clinical trial at any time without giving any reason and without any consequence on your treatment. You will be treated according to the established standards of care.

Once your doctor verified the checklist and you signed the informed consent form, you will enter the clinical trial.

The development of a clinical trial is based on a protocol. A protocol is a strict procedure that doctors and patients have to comply with in order to ensure the quality of the clinical trial. When you agreed to participate, you agreed to comply with this protocol. According to the protocol, you will have a list of appointments with your doctor and for radiological and biological exams. You will also have to report all effects you experienced with the treatment and sometimes you will have to fill in some questionnaires. You also may have to avoid certain type of drugs or food during the treatment. All the information will be collected into a special file and data will be saved into a secure database. All this information and your identity will be kept confidential.

The length of a clinical trial depends on its phase. The longest are phase III clinical trials where doctors will usually follow you for at least 5 years. The frequency of the appointments is often high the first year but lower afterwards, close to the frequency of the appointments for cancer patients not involved in the clinical trial.

Your doctor proposes it to you.
Sometimes, doctors will propose you to participate in a clinical trial. You have the right to accept or refuse without any consequence on the quality of your treatment.

You tell your doctor that you want some help to find a clinical trial for you.

If your doctor does not propose any clinical trial but you really want to participate, the best way is to ask your doctor or oncologist if there is any clinical trial for your type of cancer implemented near your home or in your country. Clinical trials are registered and your doctor might know how to look for clinical trials.
You can also ask your doctor on the possibilities of participating in a clinical trial in another country if none is available in your country. The possibilities of doing so vary from one country to another. Feel free to contact your health insurance, patients’ organizations or any other support to make it possible if this is important for you.

You search yourself for a clinical trial.
You can also search for a clinical trial yourself. To enter in a clinical trial, there are criteria to be fulfilled like age, gender, cancer type or previous treatments. RCT provides a tool to search for phase III clinical trials by type of cancer and by country. For Belgium, the Netherlands, Switzerland, Luxembourg, France and the UK, RCT provides contacts to get more information about the phase III clinical trials currently ongoing.

Click here to go to our trials listing