adjuvant therapy: One or more anticancer drugs used in combination with surgery or radiation therapy as part of the treatment of cancer. Adjuvant therapy is given before or after the primary treatment to increase the chances of a cure. Adjuvant usually means "in addition to" initial treatment.

adverse effect: See side effects.

Adverse Event Expedited Reporting System: A Web-based program that enables researchers using NCI-sponsored investigational agents to expedite the reporting of serious and/or unexpected adverse events directly to NCI and FDA.

agent: In a cancer clinical trial, an agent is a substance that researchers believe might be capable of producing an effect that fights cancer.

assent: Children and adolescents are not deemed capable of giving true informed consent, so they are asked for their assent, or agreement, to participation in a clinical trial.

audit: In clinical trials, the onsite monitoring of trial procedures, documents, and data.

Belmont Report: A 1979 report by the National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research that delineated the ethical principles upon which today's regulations regarding research participants in the United States are based: respect for persons, beneficence, and justice.

bias: Human choices, beliefs, or any other factors besides those being studied that affect a clinical trial's results. Clinical trials use many methods to avoid bias because biased results may not be correct.

biological therapy: Treatment to stimulate or restore the ability of the immune system to fight infection and disease. Also used to lessen side effects that may be caused by some cancer treatments. Also known as immunotherapy, biotherapy, or biological response modifier (BRM) therapy.

cancer: A term for diseases in which abnormal cells divide without control. Cancer cells can invade nearby tissues and can spread through the bloodstream and lymphatic system to other parts of the body.

cancer vaccine: A form of biological therapy, which may encourage a person's immune system to recognize cancer cells. These vaccines may help the body reject tumors and prevent cancer from recurring.

chemoprevention: The use of drugs, vitamins, or other agents to try to reduce the risk of, or delay the development or recurrence of, cancer.

chemotherapy: Treatment with anticancer drugs.

clinical trial: A research study that tests how well new medical treatments or other interventions work in people. Each study is designed to test new methods of screening, prevention, diagnosis, or treatment of a disease.

combination chemotherapy: Treatment using more than one anticancer drug.

combination therapy: The use of two or more modes of treatment - surgery, radiotherapy, chemotherapy, immunotherapy - in combination or alternately to achieve optimum results against cancer.

Common Toxicity Criteria: A Web-based, interactive application that uses standardized language to identify and grade adverse events in cancer clinical trials.

NCI-designated Cancer Centers: There are 3 kinds of NCI-designated cancer centers:
1. Comprehensive cancer centers, which conduct basic, clinical, and preventive research programs, as well as community outreach and education programs
2. Clinical cancer centers, which conduct primarily clinical research programs but may have programs in other research areas as well
3. Cancer centers (formerly called Basic Science Cancer Centers), which conduct basic or preventive research programs and do not have clinical programs confidence intervals: These reflect a range of values surrounding the true score that would be obtained if everyone with a particular cancer were treated with the treatment under study. The wider the interval, the more variable the result and the less likely it is to be close to the true score. Confidence intervals are typically thought of as the approximate bounds or limits surrounding the true score. Researchers frequently use either a 95 or a 99 percent confidence interval.

control group: In a clinical trial, the group that receives the accepted standard treatment being studied. In cases where no standard treatment yet exists for a particular condition, the control group would receive no treatment. No patient is placed in a control group without treatment if there is any beneficial treatment known for that patient. This group is compared to the group that receives the investigational treatment. See also investigational group.

cooperative groups: Networks of institutions that jointly carry out large clinical trials following the same protocols.

data and safety monitoring board (DSMB): An independent committee whose membership includes, at minimum, a statistician and a clinical expert in the area being studied. Members may also include bioethicists or other clinicians knowledgeable about the trial's subject matter. The National Institutes of Health requires DSMB review of all phase 3 clinical trials. A DSMB might also review phase 1 or 2 trials that are blinded, take place at multiple locations, or employ particularly high-risk interventions or vulnerable populations.

diagnostic trial: A research study that evaluates methods of detecting disease.

disease-free survival: The amount of time a participant survives without cancer occurring or recurring, usually measured in months.

double-blinded: A clinical trial in which neither the medical staff nor the person knows which of several possible therapies the person is receiving.

eligibility criteria: Participant eligibility criteria for clinical trials can range from general (age, sex, type of cancer) to specific (prior treatment, tumor characteristics, blood cell counts, organ function). Eligibility criteria may also vary with trial phase. In phase 1 and 2 trials, the criteria often focus on making sure that people who might be harmed because of abnormal organ function or other factors are not put at risk. Phase 2 and 3 trials often add criteria regarding disease type and stage, and number of prior treatments.

endpoint: What researchers measure to evaluate the results of a new treatment being tested in a clinical trial. Research teams establish the endpoints of a trial before it begins. Examples of endpoints include toxicity, tumor response, survival time, and quality of life.

Food and Drug Administration (FDA): A consumer protection agency of the U.S. Department of Health and Human Services, FDA is required by law to review all test results for new drugs to ensure that they are safe and effective for specific uses.

gene: The functional and physical unit of heredity passed from parent to offspring. Genes are pieces of DNA, and most genes contain the information for making a specific protein.

gene therapy: Treatment that alters a gene. In studies of gene therapy for cancer, researchers are trying to improve the body's natural ability to fight the disease or to make the cancer cells more sensitive to other kinds of therapy.

genetic: Inherited; having to do with information that is passed from parents to offspring through genes in sperm and egg cells.

genetic epidemiologic research: Research that involves looking at tissue or blood samples from large populations of people in order to determine how one's genetic make-up can influence detection, diagnosis, prognosis, and ultimately, treatment.

genetics trials: Clinical trials that examine whether gene transfer therapy can be used to treat cancer, or whether genetic epidemiology research improves the understanding of cancer at the cellular level. Actual genetic intervention (such as gene transfer) trials are few in number, however trials are underway where actual cellular manipulation at the gene level occurs.

imaging: Tests that produce pictures of areas inside the body.

immunotherapy: See biological therapy.

informed consent: The process of providing all relevant information about the trial's purpose, risks, benefits, alternatives, and procedures to a potential participant, who then, consistent with his or her own interests and circumstances, makes an informed decision about whether to participate.

institutional review board (IRB): A board designed to oversee the research process in order to protect participant safety. Made up of researchers, ethicists, and laypeople from the community, the board must review the trial protocols and the informed consent forms participants sign.

intervention: The study agent or method that is being tested in a clincial trial or clinical study. The intervention is usually given to the investigational group while the control group receives standard treatment.

investigational group: In a clinical trial, the group that receives the new agent being tested. See also control group.

investigational new drug (IND): A drug that the Food and Drug Administration (FDA) allows to be used in clinical trials but that the FDA has not approved for commercial marketing.

metastasis: The spread of cancer from one part of the body to another. In cancer, metastasis is the migration of cancer cells from the original tumor site through the blood and lymph vessels to produce cancers in other tissues. Tumors formed from cells that have spread are called "secondary tumors" and contain cells that are like those in the original (primary) tumor. The plural is metastases.

metastatic cancer: Cancer that has spread from the place in which it started to other parts of the body.

monoclonal antibodies: Laboratory-produced substances that can locate and bind to cancer cells wherever they are in the body. Many monoclonal antibodies are used in cancer detection or therapy; each one recognizes a different protein on certain cancer cells. Monoclonal antibodies can be used alone, or they can be used to deliver drugs, toxins, or radioactive material directly to a tumor.

multimodality therapy: Therapy that combines more than one method of treatment.

National Cancer Institute (NCI): Part of the National Institutes of Health of the United States Department of Health and Human Services, is the Federal Government's principal agency for cancer research. NCI conducts, coordinates, and funds cancer research, training, health information dissemination, and other programs with respect to the cause, diagnosis, prevention, and treatment of cancer. Access the NCI Web site at www.cancer.gov.

New Drug Application (NDA): The application filed with FDA by the trial sponsor once a trial has generated adequate data to support a certain indication for a drug.

Office for Human Research Protections (OHRP): Safeguards participants in federally funded research and provides unity and leadership for 17 Federal departments and agencies that carry out research involving human participants. OHRP enforces an important regulation called the Common Rule, which sets standards for the informed consent process; formation and function of IRBs; involvement of prisoners, children, and other vulnerable groups in research; and many other protective measures.

oncologist: A doctor who specializes in treating cancer. Some oncologists specialize in a particular type of cancer treatment. For example, a radiation oncologist specializes in treating cancer with radiation.

p-value: A statistics term. A measure of probability that a difference between groups during an experiment happened by chance. For example, a p-value of .01 (p = .01) means there is a 1 in 100 chance the result occurred by chance. The smaller the p-value, the more likely it is that the difference between groups was caused by a difference between the tested treatments.

peer review: Scientific review by a panel of experts. The primary responsibility of these experts is to assess the scientific and technical merit of research proposals.

pharmacokinetics: The activity of drugs in the body over a period of time, including the processes by which drugs are absorbed, distributed in the body, localized in the tissues, and excreted.

phase 1 trial: Small groups of people with cancer are treated with a certain dose of a new agent that has already been extensively studied in the laboratory. During the trial, the dose is usually increased group by group in order to find the highest dose that does not cause harmful side effects. This process determines a safe and appropriate dose to use in a phase 2 trial.

phase 2 trial: Phase 2 trials continue to test the safety of the new agent and begin to evaluate how well it works against a specific type of cancer. In these trials, the new agent is given to groups of people with one type of cancer or related cancers, using the dosage found to be safe in phase 1 trials.

phase 3 trial: Phase 3 studies are designed to answer research questions across the disease continuum. Phase 3 trials usually have hundreds to thousands of participants, in order to find out if there are true differences in the effectiveness of the treatment being tested.

phase 4 trial: Phase 4 trials are used to evaluate the long-term safety and effectiveness of a treatment. Less common than phase 1, 2, and 3 trials, phase 4 trials take place after the new treatment has been approved for standard use.

Physician Data Query (PDQ): PDQ is an online database developed and maintained by the National Cancer Institute. Designed to make the most current, credible, and accurate cancer information available to health professionals and the public, PDQ contains peer-reviewed summaries on cancer treatment, screening, prevention, genetics, and supportive care; a registry of cancer clinical trials from around the world; and directories of physicians, professionals who provide genetics services, and organizations that provide cancer care.

placebo: An inactive substance that looks the same as, and is administered in the same way as, a drug in a clinical trial. A placebo may be compared with a new drug when no one knows if any drug or treatment will be effective.

preclinical testing: A process in which scientists test promising new cancer treatments in the laboratory and in animal models. This is done to find out whether agents have an anticancer effect and are safely tolerated in animals. Once an agent proves promising in the lab, the sponsor applies for Food and Drug Administration approval to test it in clinical trials involving people.

prevention trials: Trials involving healthy people who are at high risk for developing cancer. These trials try to answer specific questions about and evaluate the effectiveness of ways to reduce the risk of cancer.

principal investigator: The person responsible for overseeing all aspects of a clinical trial, specifically, for developing the concept and writing the protocol; submitting the protocol for institutional review board approval; recruiting participants; obtaining informed consent; and collecting, analyzing, interpreting, and presenting data.

protocol: A written, detailed action plan for a clinical trial. The protocol provides the background, specifies the objectives, and describes the design and organization of the trial. Every center participating in the trial uses the same protocol, ensuring consistency of procedures and enhancing communication among everyone working on the trial. This uniformity ensures that participant information from all centers can be combined and compared.

quality of life: The overall enjoyment of life. Many clinical trials measure aspects of an individual's sense of well-being and ability to perform various tasks to assess the effects of cancer and its treatment on the overall quality of life.

radiation therapy: The use of high-energy radiation from x-rays, gamma rays, neutrons, and other sources to kill cancer cells and shrink tumors. Radiation may come from a machine outside the body (external-beam radiation therapy), or it may come from radioactive material placed in the body in the area near cancer cells (internal radiation therapy, implant radiation, or brachytherapy). Systemic radiation therapy uses a radioactive substance, such as a radiolabeled monoclonal antibody, that circulates throughout the body. Also called radiotherapy.

randomization: A method used to prevent bias in research. A computer or a table of random numbers generates treatment assignments, and participants have an equal chance to be assigned to one of two or more groups (e.g., the control group or the investigational group).

randomized clinical trial: A study in which the participants are assigned by chance to separate groups that compare different treatments; neither the researchers nor the participants can choose which group. Using chance to assign people to groups means that the groups will be similar and that the treatments they receive can be compared objectively. At the time of the trial, it is not known which treatment is best. It is the patient's choice to be in a randomized trial.

recurrence: The return of cancer, at the same site as the original (primary) tumor or in another location, after the tumor had disappeared.

regimen: A treatment plan that specifies the dosage, the schedule, and the duration of treatment.

regression: A decrease in the size of a tumor, or in the extent of cancer in the body.

relative risk: In cancer treatment trials, the likelihood that cancer will recur within a specific timeframe in one intervention group versus another.

remission: A decrease in or disappearance of signs and symptoms of cancer. In partial remission, some, but not all, signs and symptoms of cancer have disappeared. In complete remission, all signs and symptoms of cancer have disappeared, although there still may be cancer in the body.

risk/benefit ratio: The relation between the risks and benefits of a given treatment or procedure. An institutional review board, usually located where the clinical trial is to take place, determines whether the risks in the trial are reasonable with respect to the potential benefits. It is up to individual potential participants to decide whether it is reasonable for them in particular to participate.

sample size: In clinical trials, the number of people participating in a trial.

screening trials: Clinical trials that assess the effectiveness of new means of detecting cancer early in healthy people, especially the earliest stages of cancer. For many types of cancer, early detection results in improved outcomes. In addition, these trials examine whether early treatment, as a result of early detection, actually improves overall survival or disease-free survival.

side effects: Problems that occur when treatment affects healthy cells. Common side effects of cancer treatment are fatigue, nausea, vomiting, decreased blood cell counts, hair loss, and mouth sores.

single-blinded: Describes clinical trials set up in such a way that participants do not know which therapy or intervention they are receiving.

stage: The extent of a cancer, especially whether the disease has spread from the original site to other parts of the body. Numbers with or without letters are used to define cancer stages (e.g., stage IIb).

standard treatment: A currently accepted and widely used treatment for a certain type of cancer, based on the results of past research.

statistical power: The chance of getting a statistically significant result when there is one. Ideally, in clinical trials statistical power should be .80 or .90--in other words, there is an 80 to 90 percent chance that the true difference in effectiveness between the treatments is the smallest size considered medically important to detect.

statistically significant: Describes a mathematical measure of difference between groups. The difference is said to be statistically significant if it is greater than what might be expected to happen by chance alone.

stratification: A separation process used in randomized trials when factors that can influence the intervention's success are known. For example, participants whose cancer has spread from the original tumor site can be separated, or stratified, from those whose cancer has not spread. Assignment of interventions within the two groups is then randomized. Stratification enables researchers to look in separate subgroups to see whether differences exist.

toxicity: Harmful side effects from an agent being tested.

treatment group: See investigational group and control group.

treatment trials: Treatment trials are designed to test the safety and effectiveness of new drugs, biological agents, techniques, or other interventions in people who have been diagnosed with cancer. These trials evaluate the novel treatment against standard treatment, if there is one.

tumor: An abnormal mass of tissue that results from excessive cell division. Tumors perform no useful body function. They may be benign (not cancerous) or malignant (cancerous).

vaccine: A substance or group of substances meant to cause the immune system to respond to a cancer or to microorganisms, such as bacteria or viruses.

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