Overview
Questions and Answers About Hydrazine Sulfate
Changes To This Summary (07/17/2007)
General CAM Information
Evaluation of CAM Approaches
Questions to Ask Your Health Care Provider About CAM
To Learn More About CAM

Overview

• Hydrazine sulfate is a chemical compound that has been studied as a treatment for cancer and certain side effects caused by cancer (see Question 1 ¹).



• Hydrazine sulfate may block the tumor from taking in glucose, which is a type of sugar that tumor cells need to grow (see Question 3 ²).



• In randomized clinical trials (a type of research study), hydrazine sulfate did not make tumors shrink or go away. In some randomized trials, however, hydrazine sulfate was reported to be helpful in treating anorexia and cachexia caused by cancer. (See Question 6 ³.)



• Hydrazine sulfate is sold as a dietary supplement in the United States. The US Food and Drug Administration has not approved the use of hydrazine sulfate as a cancer treatment, except in clinical trials (see Question 8 ⁴).

Questions and Answers About Hydrazine Sulfate

1. What is hydrazine sulfate?

   Hydrazine sulfate is a compound that has been studied as a treatment for cancer and for cancer-related anorexia (loss of appetite) and cachexia (loss of muscle mass and body weight).

2. What is the history of the discovery and use of hydrazine sulfate as a complementary or alternative treatment for cancer?

   It has been known since the early 1900s that hydrazine compounds are toxic to animals and to humans. More than 400 hydrazine-related compounds have been tested for their ability to kill cancer cells. One of these compounds, procarbazine, has been used to treat Hodgkin disease, melanoma, and lung cancer since the 1960s.

   In view of procarbazine’s anticancer activity, hydrazine sulfate (a compound similar to procarbazine) was studied for its effectiveness in fighting cancer beginning in the 1970s. Studies of hydrazine sulfate as a treatment for cancer-related cachexia also began during this time.

   Hydrazine compounds have also been used to make rocket fuel, as herbicides (chemicals that kill plants), and as chemical agents in boiler and cooling-tower water systems. Many scientists consider hydrazine sulfate and other similar substances to be cancer-causing agents and are concerned about the safety of using these compounds.

3. What is the theory behind the claim that hydrazine sulfate is useful in treating cancer?

   Two theories have been suggested to explain how hydrazine sulfate acts against cancer and cachexia:

   • Hydrazine sulfate may prevent the body from making sugar that cancer cells need to grow. It has been suggested that cachexia occurs because the cancer is using too much of the body's sugar, preventing healthy cells from getting what they need to live. This causes tissues to die and muscle to waste away, and the patient loses weight.
   
   
   
   • Hydrazine sulfate may block tumor necrosis factor-alpha (TNF-alpha). This is a substance made by the body’s white blood cells to fight infection and tissue damage. High levels of TNF-alpha have been found in cancer patients. These high levels of TNF-alpha may cause loss of appetite, tiredness, and the breakdown of muscle tissue. As muscle breaks down, it makes sugar that the cancer cells use to grow. Blocking the TNF-alpha might stop tumor growth and prevent cachexia.
   
   
   
4. How is hydrazine sulfate administered?

   Hydrazine sulfate is taken by mouth in pills or capsules. There is no standard dose or length of treatment time.

5. Have any preclinical (laboratory or animal) studies been conducted using hydrazine sulfate?

   Research in a laboratory or using animals is done to find out if a drug, procedure, or treatment is likely to be safe and useful in humans. These preclinical studies are done before any testing in humans is begun. The following has been learned from preclinical studies of hydrazine sulfate:

   • In most studies with rats, mice, and hamsters, hydrazine sulfate caused an increase in lung, liver, and breast cancers.
   
   
   
   • When used alone against certain types of cancer (including melanoma, leukemia, bladder, breast, and prostate), hydrazine sulfate slowed tumor growth in some animal studies and showed no effect in others. In cases where tumor growth was slowed the most, the animals lost large amounts of weight. This finding does not support the proposed use of hydrazine sulfate to treat cachexia caused by cancer.
   
   
   
   • When hydrazine sulfate was combined with an anticancer drug, it seemed to improve the anticancer effects in rats and mice. When hydrazine sulfate was combined with an anticancer drug that affects the way cells use sugar, however, it helped in some studies and did not help in others.
   
   
   
   • Preclinical studies by the National Cancer Institute (NCI) found that hydrazine sulfate showed no anticancer activity except in one type of cancer in rats. The NCI decided not to continue studying the compound as a treatment for cancer. Studies of hydrazine sulfate as a treatment for cancer-related anorexia and cachexia continued.
   
   
   

   For more information on the preclinical studies, see the PDQ health professional version of Hydrazine Sulfate ⁵.

6. Have any clinical trials (research studies with people) of hydrazine sulfate been conducted?

   Clinical trials are a type of research study that tests how well new drugs or other treatments work in people. There have been many studies of hydrazine sulfate in patients with advanced cancer. Researchers looked at the following:

   • Tumor response and/or survival among patients with various types of cancer.
   • Changes in body weight.
   • Quality of life.
   • Changes in nutritional status and metabolism (chemical changes that take place in cells and make energy needed for life).

   Clinical trials of hydrazine sulfate have reported the following:

   • In the mid 1970s, clinical trials by a drug company found that a small number of patients who were treated with hydrazine sulfate for advanced cancer reported having a better appetite, losing less weight, feeling stronger, or having less pain. In some patients, the tumor got smaller or did not grow, or there was improvement in a cancer-related symptom. These clinical trials do not prove that hydrazine sulfate is effective for advanced cancer, however, because of weaknesses in study design. There was no control group (a group of patients who did not receive hydrazine sulfate) and half of the patients in the trial could not be counted in the results for reasons that include missing information, short treatment times, and receiving other treatment along with the hydrazine sulfate.
   
   
   
   • From the 1970s to the mid 1990s, Russian studies with hydrazine sulfate had mixed results. Little information was reported about the patients and their treatment and about the study design and methods. All of the patients in these studies also received standard treatment with surgery, chemotherapy, and/or radiation therapy. Therefore, it is not known if results were caused by hydrazine sulfate or one of the standard treatments, or both.
   
   
   
   • Clinical studies funded by the NCI in the 1970s found that hydrazine sulfate did not cause tumors to shrink or go away. Some patients reported small improvements in appetite, pain, and weight, but they did not last. These studies did not include control groups.
   
   
   
   • Four randomized controlled trials were done in the 1990s. A randomized trial is a study in which the patients are assigned by chance to separate groups to compare different treatments; neither the researchers nor the patients can choose which group. These trials compared hydrazine sulfate with a placebo (an inactive substance that looks like the treatment being tested). The results showed that hydrazine sulfate was not effective in treating cancer. In some cases, it was found to be harmful.
     • In three of the trials, lung cancer patients received either hydrazine sulfate or a placebo, along with anticancer drugs. The patients who received hydrazine sulfate did not live longer or have their tumors shrink any more than the placebo group. In one of the studies, patients who took hydrazine sulfate showed better nutritional status than patients in the placebo group, although the increase in body weight was small. In another of the studies, patients who received hydrazine sulfate had a worse quality of life than patients who received the same anticancer drugs plus the placebo. [1]
     • A fourth trial found that patients with colorectal cancer who received only hydrazine sulfate lived for a shorter time than patients who received only a placebo.
   
   
   
   • Four other randomized controlled trials studied the effects of hydrazine sulfate on nutritional status and metabolism. The studies showed some benefit from hydrazine sulfate.
     • In 2 of the studies[2], patients receiving hydrazine sulfate showed improvement in metabolism, appetite, and in either gaining weight or not losing weight.
     • In the other 2 clinical trials[3], lung and colon cancer patients receiving hydrazine sulfate had less cancer-related muscle wasting.
   
   
   
7. Have any side effects or risks been reported from hydrazine sulfate?

   In general, the reported side effects of hydrazine sulfate treatment have been mild to moderate. Most side effects are reported to end when treatment with hydrazine sulfate is stopped. Some animal studies, however, suggest that hydrazine sulfate may be highly toxic (harmful) when combined with either alcohol or barbiturates (drugs with sedative and hypnotic effects).

   Most of the side effects caused by hydrazine sulfate have involved the nervous system and gastrointestinal tract. These side effects include the following:

   • Nausea and/or vomiting.
   • Dizziness.
   • Abnormal feelings in the arms and legs (such as burning or prickling).
   • Nerve inflammation.
   • Impaired fine motor function (such as trouble writing).
   • Dry skin and/or itching.
   • Being unable to sleep.
   • Abnormally low blood sugar.

   One case of fatal liver and kidney failure and one case of severe injury to the brain have been linked to the use of hydrazine sulfate.

8. Is hydrazine sulfate approved by the U.S. Food and Drug Administration (FDA) for use as a cancer treatment in the United States?

   The FDA has not approved hydrazine sulfate for use as a cancer treatment in the United States.

   The FDA has approved the study of hydrazine sulfate in clinical trials. Information about ongoing clinical trials can be found on the NCI Web site ⁶.

   Hydrazine sulfate is sold in the United States as a dietary supplement, which is regulated as a food, not a drug. More information about dietary supplements can be found in the National Center for Complementary and Alternative Medicine online fact sheet, What’s in the Bottle? An Introduction to Dietary Supplements ⁷.

References

1. Loprinzi CL, Goldberg RM, Su JQ, et al.: Placebo-controlled trial of hydrazine sulfate in patients with newly diagnosed non-small-cell lung cancer. J Clin Oncol 12 (6): 1126-9, 1994.  [PUBMED Abstract]
   
   
2. Chlebowski RT, Bulcavage L, Grosvenor M, et al.: Hydrazine sulfate in cancer patients with weight loss. A placebo-controlled clinical experience. Cancer 59 (3): 406-10, 1987.  [PUBMED Abstract]
   
   
3. Tayek JA, Heber D, Chlebowski RT: Effect of hydrazine sulphate on whole-body protein breakdown measured by 14C-lysine metabolism in lung cancer patients. Lancet 2 (8553): 241-4, 1987.  [PUBMED Abstract]
   
   

Changes To This Summary (07/17/2007)

The PDQ cancer information summaries are reviewed regularly and updated as new information becomes available. This section describes the latest changes made to this summary as of the date above.

Editorial changes were made to this summary.

General CAM Information

Complementary and alternative medicine (CAM)—also referred to as integrative medicine—includes a broad range of healing philosophies, approaches, and therapies. A therapy is generally called complementary when it is used in addition to conventional treatments; it is often called alternative when it is used instead of conventional treatment. (Conventional treatments are those that are widely accepted and practiced by the mainstream medical community.) Depending on how they are used, some therapies can be considered either complementary or alternative. Complementary and alternative therapies are used in an effort to prevent illness, reduce stress, prevent or reduce side effects and symptoms, or control or cure disease.

Unlike conventional treatments for cancer, complementary and alternative therapies are often not covered by insurance companies. Patients should check with their insurance provider to find out about coverage for complementary and alternative therapies.

Cancer patients considering complementary and alternative therapies should discuss this decision with their doctor, nurse, or pharmacist as they would any therapeutic approach, because some complementary and alternative therapies may interfere with their standard treatment or may be harmful when used with conventional treatment.

Evaluation of CAM Approaches

It is important that the same rigorous scientific evaluation used to assess conventional approaches be used to evaluate CAM therapies. The National Cancer Institute (NCI) and the National Center for Complementary and Alternative Medicine (NCCAM) are sponsoring a number of clinical trials (research studies) at medical centers to evaluate CAM therapies for cancer.

Conventional approaches to cancer treatment have generally been studied for safety and effectiveness through a rigorous scientific process that includes clinical trials with large numbers of patients. Less is known about the safety and effectiveness of complementary and alternative methods. Few CAM therapies have undergone rigorous evaluation. A small number of CAM therapies originally considered to be purely alternative approaches are finding a place in cancer treatment—not as cures, but as complementary therapies that may help patients feel better and recover faster. One example is acupuncture. According to a panel of experts at a National Institutes of Health (NIH) Consensus Conference in November 1997, acupuncture has been found to be effective in the management of chemotherapy-associated nausea and vomiting and in controlling pain associated with surgery. In contrast, some approaches, such as the use of laetrile, have been studied and found ineffective or potentially harmful.

The NCI Best Case Series Program ⁸, which was started in 1991, is one way CAM approaches that are being used in practice are being investigated. The program is overseen by the NCI’s Office of Cancer Complementary and Alternative Medicine (OCCAM). Health care professionals who offer alternative cancer therapies submit their patients’ medical records and related materials to OCCAM. OCCAM conducts a critical review of the materials and develops follow-up research strategies for approaches deemed to warrant NCI-initiated research.

Questions to Ask Your Health Care Provider About CAM

When considering complementary and alternative therapies, patients should ask their health care provider the following questions:

• What side effects can be expected?
• What are the risks associated with this therapy?
• Do the known benefits outweigh the risks?
• What benefits can be expected from this therapy?
• Will the therapy interfere with conventional treatment?
• Is this therapy part of a clinical trial?
• If so, who is sponsoring the trial?
• Will the therapy be covered by health insurance?

To Learn More About CAM

National Center for Complementary and Alternative Medicine (NCCAM)

The National Center for Complementary and Alternative Medicine (NCCAM) at the National Institutes of Health (NIH) facilitates research and evaluation of complementary and alternative practices, and provides information about a variety of approaches to health professionals and the public.

NCCAM Clearinghouse

Post Office Box 7923 Gaithersburg, MD 20898–7923

Telephone: 1–888–644–6226 (toll free) 301–519–3153 (for International callers)

TTY (for deaf and hard of hearing callers): 1–866–464–3615

Fax: 1–866–464–3616

E-mail: info@nccam.nih.gov

Web site: http://nccam.nih.gov

CAM on PubMed

NCCAM and the NIH National Library of Medicine (NLM) jointly developed CAM on PubMed, a free and easy-to-use search tool for finding CAM-related journal citations. As a subset of the NLM's PubMed bibliographic database, CAM on PubMed features more than 230,000 references and abstracts for CAM-related articles from scientific journals. This database also provides links to the Web sites of over 1,800 journals, allowing users to view full-text articles. (A subscription or other fee may be required to access full-text articles.) CAM on PubMed is available through the NCCAM Web site ⁹. It can also be accessed through NLM PubMed ¹⁰ bibliographic database by selecting the "Limits" tab and choosing "Complementary Medicine" as a subset.

Office of Cancer Complementary and Alternative Medicine

The NCI Office of Cancer Complementary and Alternative Medicine (OCCAM) coordinates the activities of the NCI in the area of complementary and alternative medicine (CAM). OCCAM supports CAM cancer research and provides information about cancer-related CAM to health providers and the general public via the NCI Web site ¹¹.

National Cancer Institute (NCI) Cancer Information Service

U.S. residents may call the NCI Cancer Information Service toll free at 1-800-4-CANCER (1-800-422-6237) Monday through Friday from 9:00 am to 4:30 pm. Deaf and hearing impaired callers with TTY equipment may call 1-800-332-8615. A trained Cancer Information Specialist is available to answer your questions.

Food and Drug Administration

The Food and Drug Administration (FDA) regulates drugs and medical devices to ensure that they are safe and effective.

Food and Drug Administration

5600 Fishers Lane

Rockville, MD 20857

Telephone: 1–888–463–6332 (toll free)

Web site: http://www.fda.gov/

Federal Trade Commission

The Federal Trade Commission (FTC) enforces consumer protection laws. Publications available from the FTC include:

• Who Cares: Sources of Information About Health Care Products and Services
• Fraudulent Health Claims: Don’t Be Fooled

Consumer Response Center

Federal Trade Commission

CRC-240

Washington, DC 20580

Telephone: 1-877-FTC-HELP (1-877-382-4357) (toll free)

TTY (for deaf and hearing impaired callers): 202-326-2502

Web site: http://www.ftc.gov/

Glossary Terms

Not normal. An abnormal lesion or growth may be cancerous, premalignant (likely to become cancer), or benign.

Cancer that has spread to other places in the body and usually cannot be cured or controlled with treatment.

A chemical substance found in beer, wine, and liquor. Alcohol is also found in some medicines, mouthwashes, essential oils (scented liquid taken from plants), and household products.

A laboratory experiment using animals to study the development and progression of diseases. Animal studies also test how safe and effective new treatments are before they are tested in people.

An abnormal loss of the appetite for food. Anorexia can be caused by cancer, AIDS, a mental disorder (i.e., anorexia nervosa), or other diseases.

A desire to satisfy a physical or mental need, such as for food, sex, or adventure.

A drug used to treat insomnia, seizures, and convulsions, and to relieve anxiety and tension before surgery. It belongs to the family of drugs called central nervous system (CNS) depressants.

Cancer that forms in tissues of the bladder (the organ that stores urine). Most bladder cancers are transitional cell carcinomas (cancer that begins in cells that normally make up the inner lining of the bladder). Other types include squamous cell carcinoma (cancer that begins in thin, flat cells) and adenocarcinoma (cancer that begins in cells that make and release mucus and other fluids). The cells that form squamous cell carcinoma and adenocarcinoma develop in the inner lining of the bladder as a result of chronic irritation and inflammation.

A tissue with red blood cells, white blood cells, platelets, and other substances suspended in fluid called plasma. Blood takes oxygen and nutrients to the tissues, and carries away wastes.

Glandular organ located on the chest. The breast is made up of connective tissue, fat, and breast tissue that contains the glands that can make milk. Also called mammary gland.

Loss of body weight and muscle mass, and weakness that may occur in patients with cancer, AIDS, or other chronic diseases.

A term for diseases in which abnormal cells divide without control. Cancer cells can invade nearby tissues and can spread to other parts of the body through the blood and lymph systems. There are several main types of cancer. Carcinoma is cancer that begins in the skin or in tissues that line or cover internal organs. Sarcoma is cancer that begins in bone, cartilage, fat, muscle, blood vessels, or other connective or supportive tissue. Leukemia is cancer that starts in blood-forming tissue such as the bone marrow, and causes large numbers of abnormal blood cells to be produced and enter the blood. Lymphoma and multiple myeloma are cancers that begin in the cells of the immune system. Central nervous system cancers are cancers that begin in the tissues of the brain and spinal cord.

In medicine, a sac of tissue and blood vessels that surrounds an organ, joint, or tumor. A capsule is also a form for medicine that is taken by mouth. It usually has a shell made of gelatin with the medicine inside.

The individual unit that makes up the tissues of the body. All living things are made up of one or more cells.

Treatment with drugs that kill cancer cells.

A type of research study that tests how well new medical approaches work in people. These studies test new methods of screening, prevention, diagnosis, or treatment of a disease. Also called a clinical study.

Cancer that forms in the tissues of the colon (the longest part of the large intestine). Most colon cancers are adenocarcinomas (cancers that begin in cells that make and release mucus and other fluids).

Cancer that develops in the colon (the longest part of the large intestine) and/or the rectum (the last several inches of the large intestine before the anus).

In science, a substance that is made up of more than one ingredient.

In a clinical trial, the group that does not receive the new treatment being studied. This group is compared to the group that receives the new treatment, to see if the new treatment works.

A clinical study that includes a comparison (control) group. The comparison group receives a placebo, another treatment, or no treatment at all.

A product that is added to the diet. A dietary supplement is taken by mouth, and usually contains one or more dietary ingredient (such as vitamin, mineral, herb, amino acid, and enzyme). Also called nutritional supplement.

The amount of medicine taken, or radiation given, at one time.

Any substance, other than food, that is used to prevent, diagnose, treat or relieve symptoms of a disease or abnormal condition. Also refers to a substance that alters mood or body function, or that can be habit-forming or addictive, especially a narcotic.

Food and Drug Administration. An agency in the U.S. federal government whose mission is to protect public health by making sure that food, cosmetics, and nutritional supplements are safe to use and truthfully labeled. The FDA also makes sure that drugs, medical devices, and equipment are safe and effective, and that blood for transfusions and transplant tissue are safe. Also called Food and Drug Administration.

FDA. An agency in the U.S. federal government whose mission is to protect public health by making sure that food, cosmetics, and nutritional supplements are safe to use and truthfully labeled. The Food and Drug Administration also makes sure that drugs, medical devices, and equipment are safe and effective, and that blood for transfusions and transplant tissue are safe. Also called FDA.

The stomach and intestines. The gastrointestinal tract is part of the digestive system, which also includes the salivary glands, mouth, esophagus, liver, pancreas, gallbladder, and rectum.

A type of sugar; the chief source of energy for living organisms.

A chemical that kills plants.

A cancer of the immune system that is marked by the presence of a type of cell called the Reed-Sternberg cell. The two major types of Hodgkin disease are classical Hodgkin lymphoma and nodular lymphocyte-predominant Hodgkin lymphoma. Symptoms include the painless enlargement of lymph nodes, spleen, or other immune tissue. Other symptoms include fever, weight loss, fatigue, or night sweats. Also called Hodgkin lymphoma.

A substance that has been studied as a treatment for cancer and as a treatment for cachexia (body wasting) associated with advanced cancer.

A trance-like state in which a person becomes more aware and focused and is more open to suggestion.

A loss of part or all of a physical or mental ability, such as the ability to see, walk, or learn.

Invasion and multiplication of germs in the body. Infections can occur in any part of the body and can spread throughout the body. The germs may be bacteria, viruses, yeast, or fungi. They can cause a fever and other problems, depending on where the infection occurs. When the body’s natural defense system is strong, it can often fight the germs and prevent infection. Some cancer treatments can weaken the natural defense system.

Redness, swelling, pain, and/or a feeling of heat in an area of the body. This is a protective reaction to injury, disease, or irritation of the tissues.

A condition in which the kidneys stop working and are not able to remove waste and extra water from the blood or keep body chemicals in balance. Acute or severe kidney failure happens suddenly (for example, after an injury) and may be treated and cured. Chronic kidney failure develops over many years, may be caused by conditions like high blood pressure or diabetes, and cannot be cured. Chronic kidney failure may lead to total and long-lasting kidney failure, called end-stage renal disease (ESRD). A person in ESRD needs dialysis (the process of cleaning the blood by passing it through a membrane or filter) or a kidney transplant. Also called renal failure.

Cancer that starts in blood-forming tissue such as the bone marrow and causes large numbers of blood cells to be produced and enter the bloodstream.

A large organ located in the upper abdomen. The liver cleanses the blood and aids in digestion by secreting bile.

One of a pair of organs in the chest that supplies the body with oxygen, and removes carbon dioxide from the body.

Cancer that forms in tissues of the lung, usually in the cells lining air passages. The two main types are small cell lung cancer and non-small cell lung cancer. These types are diagnosed based on how the cells look under a microscope.

A form of cancer that begins in melanocytes (cells that make the pigment melanin). It may begin in a mole (skin melanoma), but can also begin in other pigmented tissues, such as in the eye or in the intestines.

The total of all chemical changes that take place in a cell or an organism. These changes make energy and the materials needed for growth, reproduction, and maintaining health. They also help get rid of toxic substances.

In medicine, having to do with the movement of body parts.

A weakening, shrinking, and loss of muscle caused by disease or lack of use. Muscle wasting decreases strength and the ability to move.

The National Cancer Institute, 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. The National Cancer Institute 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 National Cancer Institute Web site at http://www.cancer.gov. Also called NCI.

NCCAM. A federal agency that uses science to explore complementary and alternative medicine (CAM) practices, trains CAM researchers, and provides authoritative information about CAM to professionals and the public. NCCAM awards grants for research projects, training, and career development in CAM; sponsors conferences, educational programs, and exhibits; studies ways to use proven CAM practices along with conventional medical practice; and supports adding CAM to medical, dental, and nursing school programs. NCCAM is part of the National Institutes of Health. Also called NCCAM.

A feeling of sickness or discomfort in the stomach that may come with an urge to vomit. Nausea is a side effect of some types of cancer therapy.

A bundle of fibers that receives and sends messages between the body and the brain. The messages are sent by chemical and electrical changes in the cells that make up the nerves.

The organized network of nerve tissue in the body. It includes the central nervous system (the brain and spinal cord), the peripheral nervous system (nerves that extend from the spinal cord to the rest of the body), and other nerve tissue.

The state of a person’s health in terms of the nutrients in his or her diet.

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, complementary and alternative medicine, 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. Most of this information, and more specific information about PDQ, can be found on the NCI's Web site at http://www.cancer.gov/cancertopics/pdq. Also called Physician Data Query.

An inactive substance or treatment that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.

Research using animals to find out if a drug, procedure, or treatment is likely to be useful. Preclinical studies take place before any testing in humans is done.

A drug that is used to treat cancer. It belongs to the family of drugs called alkylating agents.

Cancer that forms in tissues of the prostate (a gland in the male reproductive system found below the bladder and in front of the rectum). Prostate cancer usually occurs in older men.

The overall enjoyment of life. Many clinical trials assess the effects of cancer and its treatment on the quality of life. These studies measure aspects of an individual’s sense of well-being and ability to carry out various activities.

The use of high-energy radiation from x-rays, gamma rays, neutrons, protons, 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 near cancer cells (internal radiation therapy). Systemic radiation therapy uses a radioactive substance, such as a radiolabeled monoclonal antibody, that travels in the blood to tissues throughout the body. Also called radiotherapy and irradiation.

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.

In medicine, an improvement related to treatment.

A person who has studied science, especially one who is active in a particular field of investigation.

A drug or substance used to calm a person down, relieve anxiety, or help a person sleep.

A problem that occurs when treatment affects healthy tissues or organs. Some common side effects of cancer treatment are fatigue, pain, nausea, vomiting, decreased blood cell counts, hair loss, and mouth sores.

In medicine, treatment that experts agree is appropriate, accepted, and widely used. Health care providers are obligated to provide patients with standard therapy. Also called standard of care or best practice.

A procedure to remove or repair a part of the body or to find out whether disease is present. An operation.

The percentage of people in a study or treatment group who are alive for a certain period of time after they were diagnosed with or treated for a disease, such as cancer. The survival rate is often stated as a five-year survival rate, which is the percentage of people in a study or treatment group who are alive five years after diagnosis or treatment. Also called overall survival rate.

An indication that a person has a condition or disease. Some examples of symptoms are headache, fever, fatigue, nausea, vomiting, and pain.

A group or layer of cells that work together to perform a specific function.

Having to do with poison or something harmful to the body. Toxic substances usually cause unwanted side effects.

An abnormal mass of tissue that results when cells divide more than they should or do not die when they should. Tumors may be benign (not cancerous), or malignant (cancerous). Also called neoplasm.

A protein made by white blood cells in response to an antigen (substance that causes the immune system to make a specific immune response) or infection. Tumor necrosis factor can also be made in the laboratory. It may boost a person’s immune response, and also may cause necrosis (cell death) of some types of tumor cells. Tumor necrosis factor is being studied in the treatment of some types of cancer. It is a type of cytokine. Also called TNF.

To eject some or all of the contents of the stomach through the mouth.

Refers to a blood cell that does not contain hemoglobin. White blood cells include lymphocytes, neutrophils, eosinophils, macrophages, and mast cells. These cells are made by bone marrow and help the body fight infections and other diseases. Also called WBC.