Gastrointestinal oncology

A therapy study conducted with cancer patients is used in cancer research to assess new treatment methods: Each study is designed to answer specific scientific questions. The aim is to find new and better ways to help people with cancer.

The search for better ways begins in the laboratory and with animal experiments. Only the most promising active substances and treatment methods are tested on patients. In this way, it is hoped that many sick people can be helped in the future.

Before a new treatment method is trialled on humans, it is carefully tested in the laboratory. This is where it is determined which new methods are most likely to be successful and how they can be used as safely and effectively as possible. But all this research work cannot predict whether the new form of treatment will also work in humans. Successes and risks can occur with any new cancer therapy. Even risks that are not known in advance. The aim of therapy studies is to find out whether a new form of treatment is safe and effective. As the trial progresses, more and more is learnt about the new treatment.

Treatments that are already frequently used are called standard therapies. They are often the basis for new, perhaps better cancer therapies. Many new forms of treatment have been developed in this way: what has already been successful in the past is to be further improved.

Only those patients who really want to take part in therapy studies do so. Before you make a decision; find out all you can about it!

Advances in medicine and science are the result of new ideas. They are developed through research. A new treatment must be shown to be safe and effective in therapy studies on a certain number of patients. Before this, it must not be applied to all people in general.

Through therapy studies, we learn which treatment is more effective than another. Comparison is the best way to test a new therapy. A large number of today's standard therapies were first recognised as effective in this way in therapy studies. Even today, such studies help us to find new and better treatments.

Patients take part in treatment trials for various reasons: they usually hope that they themselves will be helped. They hope to perhaps be cured of cancer, live longer or feel better. They often want to support research efforts that will later help others.

Based on laboratory tests and previous studies, a therapy study is being developed to show whether the new treatment is better than an old one. The hope is that it will be. A therapy study is only ever carried out if this justified hope exists. Researchers often use components of standard therapies and try to develop better therapies from them.

Studies have often shown that a new therapy is better than an old one, and sometimes that it is not as good or at least not better. While a therapy study is being planned and carried out, there are always important reasons to assume that the new therapy is just as good or even better than the old one. Nevertheless, it can happen that the new therapy is a disappointment.

Patients taking part in therapy trials are the first to receive a new treatment. It is impossible to predict exactly how the new treatment will work. Even tried and tested standard therapies do not work the same for everyone. You should only decide whether you want to take part in a standard therapy or a therapy trial once you know both sides: the possible risks and the possible benefits. Your doctor can explain this to you.

If a therapy study shows that a new treatment is better than an old one, then the patients who take part in this study will also be the first to benefit from the new therapy. All patients are carefully monitored and followed, even after the end of the trial. They become part of the network of therapy studies being conducted throughout Germany. Doctors and researchers jointly contribute their ideas and experience to this network. The knowledge of many specialists flows into the design and evaluation of therapy studies. The patients in the therapy trials enjoy the benefits of this experience. At tumour centres, they are looked after by a special research team. New programmes are integrating more and more hospitals and doctors into this research network.

Yes, like any cancer therapy, treatment in therapy trials can also have side effects and risks. These side effects vary from person to person. They depend on the specific form of treatment and the patient's condition.

Therapy studies explore uncharted territory. Therefore, the risks are not always predictable, although many efforts have been made to find out which risks could arise. For this reason, treatment trials can harbour unknown dangers and side effects as well as hopes and benefits.

Most of the side effects of treatment trials go away as soon as the treatment is finished. For example, some cancer drugs (cytostatics) cause hair loss or nausea. They often also damage the bone marrow, where blood cells are produced. It can happen that the number of blood cells becomes so low that, for example, the risk of infections increases. For this reason, the number of blood cells (blood values) is checked regularly during the trial. Fortunately, the bone marrow usually recovers quickly so that the blood counts soon return to normal.

Some side effects do not go away; they can be very serious, some even life-threatening. There may also be side effects that are only noticed years later: these include damage to the heart, lungs and kidneys or infertility or a second cancer. All of these side effects can also occur with standard therapy. Nevertheless, many people suffering from cancer today live longer than in the past. This is mainly due to better treatment methods.

Before you decide to take part in a therapy trial, you will be given a detailed explanation of which treatment method is to be tested. You will then receive an information sheet explaining the possible risks and benefits of the new treatment. If you agree to take part in the treatment trial, you will be asked to sign a form known as informed consent. Make sure you understand the risks you may face before you sign. If there is anything you do not understand, ask. Doctors or nurses will answer your questions. You can always refuse to take part in a therapy study. Even if you have signed the form, you have the right to leave the therapy study at any time. You can then continue to be treated with a standard therapy.

It is certainly not easy for you as a patient to decide on your own treatment: there are so many things that have to be taken into account. Cancer is a disease with its own side effects that are independent of cancer therapy. These unavoidable risks of the disease itself must always be carefully weighed against the potential risks and benefits of a new cancer therapy. As a rule, both standard therapy and treatment in a therapy trial have side effects and risks.

Any medical treatment can have side effects for some people. Cancer therapy can have severe side effects because it is a particularly intensive treatment: it was developed to damage cancer cells so that they die. However, healthy cells can also be damaged in the process. This damage to healthy cells is the cause of many side effects. The major goal of cancer research is therefore to find a cancer therapy that causes cancer cells to die but does not damage healthy cells.

Researchers are trying to make cancer therapy more effective and minimise the side effects. These are examples of the results of these endeavours:

• new cancer drugs with fewer side effects

• more effective drugs against nausea

• shorter periods of time during which cancer drugs have to be taken

• better protection of healthy tissue during radiotherapy

• new surgical methods that allow more limited interventions and less disfigurement of the body

• better information and psychological support during difficult times.

Every therapy study is planned to answer very specific questions. Every study needs people at a certain stage of the disease and in a very specific state of health. Only if these conditions are the same for all participants can the standard therapy and the new treatment method be compared with each other. If you fulfil the general conditions, you may also be suitable for the relevant study.

Before a decision is made about your treatment, the type and stage of your disease will be analysed in detail. The "cancer stage" describes how far the disease has spread. The type of treatment depends on this and your general state of health. You will most likely be made aware of the treatment study by your own doctor, or perhaps by others who are familiar with your disease. In any case, you need to understand what to expect in a treatment trial. You must want to take part voluntarily. Ask what the consequences of taking part will be for you.

If you are thinking about taking part in a therapy study, ask these important questions:

• What is the purpose of the therapy trial?

• What does the therapy study entail?

• What tests and treatments? What will be done and how?

• What is likely to happen to the disease in my case? (With standard therapy? With the new therapy? What other options do I have? What are the advantages and disadvantages?

• Is there a standard therapy in my case?

• What is your relationship to the therapy study?

• How will the therapy study affect my everyday life?

• What side effects should I expect?

• How long will the trial last?

• Do I have to stay in hospital? If so, how often and for how long?

• Will I have any costs?

• If I am harmed by the trial or leave it, what treatment am I entitled to? Is there any insurance cover?

• What kind of long-term follow-up care is part of the study?

Finding an answer to this question can be very difficult for you: You may feel overwhelmed by the cancer diagnosis and the decisions you now have to make. Or you may be confused and upset. Talk to people close to you about these difficulties. It is also important to discuss all treatment options with medical professionals now - including your own doctor. Take the time to ask all your questions. It may help to write them down beforehand. No question is stupid: find out as much as you can! Find out what your options are and what the risks and benefits are for you.

Every patient is different: you are an individual with individual needs, and your health is important.

When you make your decision about cancer treatment, remember: you are not alone. Many people are there to help you: Doctors, nurses, social workers, clergy, your family, good friends, other people affected by cancer. They can all help you to find out what is best for you. Then make your own decision.

No study may be conducted without the knowledge and consent of the patients. If a doctor or researcher wishes to conduct a study, they need the consent of all patients who are to take part. Consent is preceded by detailed information: this process is called "informed consent": if you are one of the patients, you will receive comprehensible information about the contents of the study from a doctor. You are entitled to a written information sheet.

You will then receive an informed consent form. Read it and think about it carefully. Ask any questions you still have. Then decide freely whether you want to take part or not. If you want to take part in the study, sign the form. It is also possible that you only give your verbal consent: in this case, a witness should be present to hear you give your consent. Of course, you can also refuse to participate. This will not put you at a disadvantage.

You may want to consider whether you would like your sick child to take part in a study. In the case of underage children, you would give your consent. However, if your child is familiar with his/her illness and can understand the significance of a therapy study, then you should involve him/her: Your child should also be told what will happen in the study and should also be asked for their consent.

Informed consent is an ongoing process. If you take part in a study, you will constantly receive new information about your treatment. You will be told anything that could change your willingness to stay in the study. Signing the consent form does not bind you: you can leave the study at any time.

Whether cancer patients take part in a treatment trial or not, they are faced with a new world of medical terminology and procedures. Some people think of treatment trials as "experiments" and being a "guinea pig". Naturally, the fear of the unknown is great. Knowing about treatment trials and what's involved can alleviate some of your fears. For example, patients in a clinical trial receive their treatment at the same place where standard treatment takes place: in tumour centres and hospitals.

Here, doctors, nurses, social workers and other people from many different specialities work together to help you. Your well-being and privacy are respected.

If you take part in a therapy study, your condition will be closely monitored. Observations and data about your illness will be carefully noted. You may undergo more tests than usual: this is to assess progress and collect data. Of course, these examinations can entail various risks and inconveniences, but also benefits. In any case, they mean more observation during the course of the disease and therefore protection. If it turns out during the course of the study that the treatment is not to your benefit, you can consider other treatment options in consultation with your doctor.

Whether cancer patients take part in a treatment trial or not, they are faced with a new world of medical terminology and procedures. Some people think of treatment trials as "experiments" and being a "guinea pig". Naturally, the fear of the unknown is great. Knowing about treatment trials and what's involved can alleviate some of your fears. For example, patients in a clinical trial receive their treatment at the same place where standard treatment takes place: in tumour centres and hospitals.

Here, doctors, nurses, social workers and other people from many different specialities work together to help you. Your well-being and privacy are respected.

If you take part in a therapy study, your condition will be closely monitored. Observations and data about your illness will be carefully noted. You may undergo more tests than usual: this is to assess progress and collect data. Of course, these examinations can entail various risks and inconveniences, but also benefits. In any case, they mean more observation during the course of the disease and therefore protection. If it turns out during the course of the study that the treatment is not to your benefit, you can consider other treatment options in consultation with your doctor.

Yes, just as you can refuse to participate, you can also leave the therapy study at any time. Your individual rights do not change because you are a patient in a therapy study. You can decide whether you want to participate or not. And you can revoke your decision at any time, even after the study has started.

If you have any questions about any part of the study, ask your doctor. If you are not satisfied with the answers, you can consider leaving the study. If you decide to leave the study, you will not suffer any disadvantages. Don't be afraid: you are free to talk to your doctors and nurses about other possible therapies at any time.

The ethical and legal rules that govern medical practice naturally also apply to therapy studies. In addition, there are certain precautionary measures to protect patients. These measures include the regular monitoring of treatment plans and the progress of individual trials by external researchers.

Each therapy study is carefully monitored with regard to ethical issues, patient protection and scientific success: your well-being is closely monitored. If you feel worse during the trial, you can be withdrawn. In this way, the probability of something happening to you as a result of a trial is minimised. But you are also protected against this low probability: Insurance cover is guaranteed for all participants in a therapy study; your doctor can tell you the exact insurance conditions.

During the course of the therapy study, the results are made available to scientific congresses, medical journals and various government agencies. Data protection is always guaranteed.

You can use these questions to find out whether a study is being conducted correctly:

• What is the aim of the study?

• Who has reviewed and approved the study?

• Who is sponsoring the study?

• How are patient data and patient safety checked?

• Who will receive the information obtained?

Before you agree to participate, ask questions until you are satisfied with the answers. It is for your own protection.

There are many different types of therapy studies. They range from studies on the prevention, detection and diagnosis of cancer to studies on the psychological effects of the disease and studies on improving quality of life. This also includes pain control.

Most cancer therapy studies focus on new treatment methods, for example new surgical and radiotherapy procedures and new drugs. Alone or in combination with other therapies, these treatments can cure many cancer patients and prolong the lives of many others. Biological therapy is a relatively new area of cancer treatment: it involves the use of substances produced by the body's own cells, as well as substances that affect the body's defence system and protect it against disease

Therapeutic trials are conducted in different stages (phases), each of which is designed to gather specific information. Patients may be eligible for trials in certain phases, depending on their overall health and the stage of their cancer.

Phase I

In a phase I treatment trial, a new treatment method is given to a small number of patients. Researchers need to find out how the treatment works best and to what extent it is safe. They therefore carefully monitor all possible side effects. Efficacy and side effects have been intensively tested in the laboratory and in animal experiments. Nevertheless, it is not possible to predict with certainty whether this treatment will also have the hoped-for favourable effect in humans. For this reason, phase I trials can also harbour risks. They are only offered to patients for whom no other treatment is currently available. These new treatment methods can be effective and some patients have already been helped.

Phase II

Phase II therapy studies determine the effects of the new treatment method on different types of cancer. Each new phase of a treatment trial builds on the information from the earlier phase. If a treatment in phase II has shown a positive effect against the cancer, the therapy study enters phase III. Here, the new treatment method is compared with the standard therapy to see which form of therapy is more effective, and researchers often use standard therapies as a basis for developing new, possibly better methods.

Phase III

In Phase III, the new method is compared directly with the old one.

Phase IV

In phase IV studies, the new treatment method finally becomes part of the standard therapy for patients. For example, a new drug that has proven effective in phase I to III therapy trials can be used in combination with other effective treatments, such as other drugs, surgery and radiotherapy.

Those doctors who carry out a therapy study follow a carefully developed treatment plan. It sets out what is being done and why. Treatment trials are planned in such a way that patients are protected and important questions can be answered.

Some treatment trials test a particular treatment method in a specific group of patients. Others compare two or more methods in separate groups of patients who are comparable in certain respects, such as stage of disease, to ensure that the patient groups are similar and that the results can be compared.

One of the groups can receive the standard treatment (the most widely accepted treatment method), so the new treatment method can be directly compared with it. The group that receives the standard treatment is called the "control group": For example, the control group may receive the standard surgical treatment for a particular type of cancer, while another group of patients with the same type of cancer is treated by surgery plus radiotherapy to see if this gives better results.

Sometimes there is no standard therapy for certain cancer patients. In drug trials looking at such cases, one group of patients may receive a new drug while the control group receives no drug at all. However, no one is included in a no-treatment control group if there is a known treatment that could benefit patients. The control group is observed and treated just as carefully as the "treatment group".

One way of eliminating the influence of doctors and patients on the study is randomisation. If the patient agrees to be randomised, he or she is assigned to one group or the other. The researchers do not know which treatment method is the best: According to the latest state of knowledge, any treatment available for selection can have a positive effect for the patient.

If the treatment within the therapy study does not help patients, they can be removed from the study. Of course, each patient can also decide to leave the study: they will always receive help.

The results of the study are regularly reviewed and information is shared. This is important because if it turns out that a method is too dangerous or ineffective, it will be cancelled. Even if it turns out that one method is better than the others, the therapy study is terminated and all patients are treated with the better method. No patient will ever receive a treatment that is worse than any other known treatment.

Throughout the clinical trial, the patient's family doctor is kept informed of the patient's progress. Patients are encouraged to maintain contact with the referring doctor.

The knowledge gained through therapy studies is important for progress in cancer research. Such studies have significantly increased survival rates for childhood cancers, Hodgkin's disease, breast, uterine, testicular, bladder and other cancers.

Today, important scientific discoveries in laboratories are part of a revolution in biology and medicine. This knowledge is leading to promising new treatment approaches. Therapy studies are the link between this basic research and patients. The aim is to utilise the research results in such a way that they directly help people.

Adjuvant therapy

Adjuvant means "supportive", i.e. in addition to the actual treatment: one or more drugs (chemotherapy, hormones) are used in combination with surgical interventions or radiotherapy as part of the cancer treatment.

Antigen

A substance that stimulates the immune system to produce antibodies and enters the body from outside. Antigens include, for example, foreign proteins, bacteria, viruses and pollen. Antibodies Substances that the body's immune system produces as a defence reaction to foreign bodies (antigens) that have entered the body and that are specifically directed against these foreign bodies.

Biological therapy

The use of substances that also occur in the human body or substances that influence the patient's immune system.

Biopsy

Removal of a tissue sample which is analysed for its composition. This can be used to detect benign or malignant changes. Blood test (blood count) Determination of the number of red and white blood cells and platelets in a blood sample.

Chemotherapy

Treatment with drugs (cytostatics) that primarily kill cancer cells.

Double-blind study

In such studies, neither the doctor nor the patient knows which of two possible test drugs a particular patient will receive. In blind studies, the participants do not know which therapy they are being treated with. This is to avoid influencing the therapy assessment. However, if necessary, the treatment method can be identified using a special code.

Histology

The study of the tissues of the body. In a histological examination, the tissue removed is examined for its individual components; it serves to confirm the diagnosis if a tumour is suspected.

Hormones

Substances produced by the body's own glands. They enter other organs via the bloodstream and regulate processes such as growth, sexual behaviour and metabolism.

Immune system

A complex defence system of our body against foreign substances and organisms; organs, cells, white blood cells and specialised substances (e.g. hormones) are part of this system.

Immunotherapy

A form of biological therapy. Experimental method of fighting cancer using substances that stimulate the body's own immune system.

Informed consent

The process by which a patient learns about the aims and conditions of a treatment trial before potentially agreeing to participate. This process includes an information sheet that states what participants need to know about the potential risks or benefits of a treatment trial. Even after signing a participation form, you can leave the treatment trial at any time and receive a different treatment. Informed consent is required for all therapy trials.

Interferon

A protein produced by certain white blood cells. Various types of interferon have shown anti-tumour activity in experimental animals. Interferon is one of several groups of active substances in biological therapy.

Bone marrow

The spongy core of large bones. Blood cells are produced in the bone marrow.

Combination therapy

Combination of several procedures in the treatment of cancer, e.g. combination of chemotherapy, radiotherapy and surgical therapy. Combined chemotherapy The simultaneous treatment with two or more anti-cancer drugs.

Control group

In a treatment trial, the control group is the group that receives the standard treatment (a treatment that has been shown to be effective in previous trials and is normally used). The results of new treatments can be compared with the results of the control group. In cases where no recognised treatment exists, the control group receives no treatment. Studies with a control group without treatment are only permitted if there is no known therapy that could be of benefit to the patient.

Cancer

A generic term for over l00 diseases characterised by the uncontrolled growth of cells. Cancers often lead to tumour colonisation that invades normal tissue. As the disease progresses, metastases can form.

Metastasis

A "secondary tumour" that develops elsewhere in the body, away from the original site. Tumour cells usually spread via the bloodstream and lymphatic system.

Monoclonal antibodies

Genetically engineered, highly specific antibodies that can be used both for the diagnosis and treatment of tumour diseases.

Multimodal therapy

The combined use of one or more treatment methods, e.g. surgery and chemotherapy.

Side effect

A generally unfavourable effect of a drug or treatment method. Nausea, for example, is a side effect of some cancer drugs.

Oncology

The branch of medicine that deals with the research and treatment of tumour diseases.

Placebo

A placebo is a dummy drug. It is administered because of the possible psychological effect that taking medication can have. It serves as a control when evaluating the effectiveness of another drug. It is usually a capsule, tablet or injection that contains an ineffective and harmless substance but looks exactly like the drug being tested.

Protocol

Test plan for the application of an experimental procedure or an experimental treatment (treatment plan).

Randomisation

see randomisation

Regression

Regression means regression; this refers to the shrinking or disappearance of a centre of cancer. Remission The subsiding, the decline of a disease. A distinction is made between complete remission, in which no more tumour is detectable, and partial remission, in which not all but many signs of the tumour have improved.

Recurrence

Recurrence of a tumour after previous treatment. A distinction is made between local recurrence (at the site of the initial tumour) and metastasis (at a distance from the initial tumour). Risk/benefit ratio The relationship between the risks and benefits of a treatment. The risks must always be in an acceptable proportion to the expected benefits.

X-ray contrast medium

Aids that are introduced into the body in order to better visualise body areas or organs in X-ray images.

Standard therapy

The recognised and applied treatment method. Its effectiveness has been demonstrated in previous therapy studies.

Radiotherapy

Application of high-energy radiation (X-rays, Cobalt 60, radium, neutrons) for the treatment of tumour diseases.

Study arm

If different therapies are compared in a therapy study, the different groups are called "study arms". Patients are allocated to study arms according to a predetermined key (see random allocation).

Therapy study

A systematic investigation of the effects of substances or methods according to a predetermined plan within a group of patients with a certain disease or a certain stage of the disease is called a therapy study. In cancer research, a therapy study is usually the evaluation of treatment methods (operations, drug treatment or radiotherapy). In addition, methods for prevention, early detection and diagnosis can also be the subject of studies.

Randomisation

If there is evidence that a new therapy is just as good or even better than a standard therapy, the two must be compared in detail. To ensure that they are comparable, attempts are made to eliminate irrelevant aspects. For this reason, patients with the same clinical picture are randomly assigned to different study arms (see there) of a study (randomisation). This means that they are randomised to receive either the new or the standard therapy.

(Taken from the brochure "Therapiestudien - dafür sind sie gut" ("Therapy studies - what they are good for") by the Leading Commission on Cancer Therapy Studies of the German Cancer Society and German Cancer Aid; medical supervision: Prof. Dr Dieter Hoelzer, Prof. Dr Ulrich Tröhler; text: Simone Schiffner-Backhaus; Frankfurt, March 1994