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National Cancer Institute Fact Sheet
  • Reviewed: 06/06/2006

Drug Discovery at the National Cancer Institute: Fact Sheet

Key Points

  • The National Cancer Institute (NCI) has played an active role in the development of drugs for cancer treatment for over 50 years. This is reflected in the fact that approximately one half of the chemotherapeutic drugs currently used by oncologists for cancer treatment were discovered and/or developed at NCI.
  • NCI's Developmental Therapeutics Program (DTP) has over 400,000 drugs in its repository that have gone through some kind of screening process. About 80,000 compounds have been screened since 1990, using the current screening system.
  • Compounds can enter at any stage of the development process—with either very little or extensive prior testing.
  • NCI supports about 1,500 clinical trials through a variety of programs. Some are conducted by NCI researchers at the National Institutes of Health in Bethesda, Md., while others take place at cancer centers, hospitals, and community practices around the country.

The National Cancer Institute (NCI) has played an active role in the development of drugs for cancer treatment for 50 years. This is reflected in the fact that approximately one half of the chemotherapeutic drugs currently used by oncologists for cancer treatment were discovered and/or developed at NCI. The Developmental Therapeutics Program (DTP) promotes all aspects of drug discovery and development before testing in humans (preclinical development), and is a part of the Division of Cancer Treatment and Diagnosis (DCTD). NCI also funds an extensive clinical (human) trials network to ensure that promising agents are tested in humans. NCI's Cancer Therapy Evaluation Program (CTEP), also a part of DCTD, administers clinical drug development. This fact sheet focuses primarily on preclinical development within DTP. (http://dtp.nci.nih.gov/)

  1. What role does the DTP play in drug development at NCI?

    DTP participates in all stages of pre-clinical drug discovery and development. At the initial stage, DTP actively solicits scientists for candidate drugs. A drug is first screened in a variety of human tumor cell lines growing in tissue culture dishes to test for its ability to prevent the growth of specific kinds of tumor cells. If the drug shows promise, extensive testing in animals will determine whether it is effective and safe enough for testing in humans.

  2. How long has NCI had a drug development program?

    NCI has had a screening program to test experimental cancer drugs since 1937, but it was not until 1955 that a formal organization (called the Cancer Chemotherapy National Service Center) was formed to integrate laboratory resources with clinical facilities. In the early decades, compounds were screened in leukemic mice, and in l975, a program to screen mice bearing transplanted human tumors was introduced. Over time, the system has evolved into one that combines both in vitro (cancer cells grown in a dish in the laboratory) with in vivo (animal) testing. The current system, described in detail in question 5, has been operational since 1990.

  3. How many drugs has NCI tested?

    DTP has over 400,000 drugs in its repository that have gone through some kind of screening process at NCI. About 80,000 compounds have been screened since 1990, using the current screening system.

  4. Where do these drugs come from? How can they be characterized?

    NCI actively solicits drugs from government laboratories, research institutes, academic institutions, and companies throughout the world. DTP scientists systematically scan the latest literature for novel compounds and request samples of promising drugs.

    Another source of novel compounds comes from the Natural Products Branch, a part of DTP that collaborates with agencies throughout the world to collect thousands of plant and marine organisms for the tumor screen. To expedite drug discovery, NCI is providing sample sets of more than 140,000 synthetic chemicals, 80,000 natural products extracted from plants and marine organisms, and other biological materials to investigators who might have discovered potential cancer-associated molecular targets.

    Although many drugs are voluntarily submitted, a substantial fraction screened are solicited by NCI. Of all the drugs screened by NCI, about 40 percent come from industry. The remainder comes primarily from academic collaborators.

  5. What are the specific stages of testing in DTP once a drug has been chosen to be evaluated?
    • Preliminary in vitro screening: New drugs are first evaluated in a pre-screen consisting of three human tumor cell lines. Cells are exposed to each drug at a single concentration for 48 hours. If growth of one or more cell lines is inhibited, the drug is automatically evaluated against the full panel of 60 human tumors.

    • In vitro screen in human tumors: Each drug is exposed to 60 human tumor cell lines, including lung, colon, melanoma, prostate, ovarian, breast and kidney cancers at five different doses for 48 hours. If the drug is unique in some way—kills preferentially one or more of the tumor cell lines, has a unique mechanism of action, or can kill tumors at a very small concentration—testing will proceed to the next stage. Approximately 2,500 compounds are tested on a yearly basis. About 2 percent of those screened will be recommended for the next stage of testing in mice.

    • In vivo testing using hollow-fiber technique: Small hollow fibers (tubes one millimeter in diameter and two centimeters long made of a plastic, polyvinylidene fluoride), containing cells from human tumors are inserted underneath the skin and in the body cavity of the mouse. Each candidate drug is administered at two dosages and is tested against 12 target tumor cells in different hollow fibers. A total of about 150–200 compounds/year is screened by this method. Compounds that retard the growth of the cells are recommended for the next level of testing. The average length of this test is about four days.

    • In vivo testing using xenografts: Human tumors are injected directly below the skin of mice. Candidate drugs which have shown evidence of activity in the hollow fibers may be selected for testing in xenografts. The drugs are administered to the mice at various dosages, and those compounds that kill or slow down the growth of specific tumors with minimal toxicity to the animal will proceed to the next stage of testing. The average length of this test is about 30 days.

    • Pharmacology, formulation and toxicology studies: Drugs that reach this stage of development are under serious consideration for testing in humans so that substantial resources may eventually be committed to their development. There are two levels of development at this stage.

    • At the first level of development, scientists determine the basic pharmacology of the compound in animals to see where the drug is metabolized. The best chemical formulation for administering the drug, how much of the drug to give, how often and whether the drug should be taken orally or by injection is also established.

    • If a drug progresses to the next level, NCI commits resources, approximately $250,000 to $500,000, to further development. A large-scale production plan for the compound may be developed if needed. In addition, toxicology evaluations are done in two species of animals using the same material under consideration for human trials. If the drug has no serious problems, scientists recommend the initial dose, route and schedule for patients in early trials (phase I clinical trials).

  6. What is the usual procedure for a company or academic laboratory to enter into the process of NCI's drug development program?

    Compounds can enter at any stage of the development process—with either very little or extensive prior testing. A laboratory may only want its compound screened in tumor lines. Alternatively, a compound may have been already tested outside the NCI in tumor cell lines, requiring only animal evaluation. Some compounds have been tested in animals outside the NCI and are brought before the DDG for approval to test in humans.

  7. What happens after the final testing in animals?

    Once a drug is approved by the DDG, the next step is to file an Investigational New Drug Application (IND) with the Food and Drug Administration (FDA) to allow testing in people. The IND describes the chemical structure of the compound, how it is thought to work in the body, any toxic effects found in the animal studies, how the compound is produced and where and how the human trials will be conducted. The IND may be submitted by NCI, a company or an academic lab.

    CTEP is responsible for submitting INDs for NCI as well as drugs in which NCI is involved in a CRADA (Cooperative Research and Development Agreement) or CTA (Clinical Trials Agreement) with a company or supplier. NCI currently holds nearly 129 INDs; many of these represent cooperative drug development efforts with industry.

  8. What happens after the drug is approved by the FDA for testing in humans?

    The sponsor of the IND prepares a plan, or protocol, outlining the institutions and number of people that will take part in the study, the eligibility requirements, the medical tests and interventions they will receive, and how often. The protocol must be approved by NCI (or the organization sponsoring the study) and the Institutional Review Board (IRB) at each hospital or study site. This board, which includes consumers, clergy, and health professionals, reviews the protocol to ensure that the research will not expose patients to extreme or unethical risks.

    Once the centers and institutions have been selected and protocols approved, phase I and phase II trials are conducted with a limited number of people to determine the safety, dosage, effectiveness, and the side effects of a drug. If these preliminary trials indicate that the drug is well-tolerated and shows some efficacy, phase III trials are initiated. In phase III trials, hundreds of people around the country are assigned at random to receive either the new treatment, the standard treatment, or if no standard treatment is effective, a placebo.

  9. What groups carry out NCI-supported clinical trials?

    NCI supports about 1,500 clinical trials through a variety of programs. Some are conducted by NCI researchers at the National Institutes of Health in Bethesda, Md., while others take place at cancer centers, hospitals, and community practices around the country.

    The Clinical Trials Cooperative Groups (http://www.cancer.gov/cancertopics/
    factsheet/NCI/clinical-trials-cooperative-group    ) are composed of academic institutions and cancer treatment centers throughout the United States, Canada, and Europe, and are made up of more than 1,700 institutions that are responsible for placing more than 22,000 new patients into cancer treatment trials each year.

    The NCI Cancer Centers Program (http://cancercenters.cancer.gov) comprises 61 NCI-designated cancer centers throughout the country. Many cancer centers conduct NCI-approved protocols and have web sites that provide information about ongoing trials at their facilities.

    The Community Clinical Oncology Program (CCOP) (http://dcp.cancer.gov/programs-resources/programs/ccop) provides support to community physicians and local hospitals which are not a part of the Cooperative Groups so they can participate in NCI-supported clinical trials. By affiliating with either an NCI-supported clinical cooperative group or cancer center, the 390 participating hospitals and 2,320 participating physicians in CCOP, can enter patients into NCI-sponsored trials.

  10. How many cancer patients participate in NCI-sponsored clinical trials?

    Each year about 27,000 new patients enter into NCI-sponsored clinical trials. About 20,000 participate in Cooperative Groups, 5,000 in the CCOPs, and 2,500 in trials conducted at NIH in Bethesda, Md. This represents about 3 percent of adult cancer patients and about 60 percent of children who are diagnosed with cancer each year. Children under age 20 account for about 1 percent of all cancer cases. (http://www.cancer.gov/clinicaltrials/)

  11. What happens after Phase III trials?

    At the conclusion of the phase III trials, the results are reported at meetings and in peer-reviewed journals. If the treatment is proven safe and effective in phase III trials, the drug company sponsor of the drug files a New Drug Application (NDA) with FDA. The average time for FDA approval is six months to a year. Once approved by FDA, the drug becomes available for physicians to prescribe for patients.

  12. About how long does the entire drug approval process take?

    There is a growing recognition that a major factor contributing to the bottleneck in drug development is the time-consuming and less than optimal processes for preclinical and clinical testing of drugs. The Pharmaceutical Research and Manufacturers of America (PhRMA) estimates that on average the industry must spend $0.8–$1.7 billion and 12–15 years of research and development to bring a product to market. This leaves only 2–5 years of patent life and market exclusivity before generic competition results in lower prices and profits. With such a limited time to recoup its investment, industry is forced to charge increasingly higher prices.

  13. Does NCI have mechanisms for ensuring privacy of the test results when working with private industry?

    Yes, in order to encourage collaborations with private industry, procedures were developed for confidential handling of proprietary chemicals, called the Discreet Screening Agreement. All screening data are returned to the supplier.

  14. What is unique about the screening program at NCI?

    The NCI focuses on scientific merit and uniqueness. In particular, NCI scientists are looking for drugs with novel mechanisms of action.

  15. What are some success stories? What drugs developed by DTP at NCI are now in commercial use?

    Half of the FDA-approved anticancer drugs were sponsored by NCI. For example, cisplatin for treating testicular, ovarian, and lung cancer, and paclitaxel (Taxol) and fludarabine phosphate for treating several cancers and lymphoma, respectively are examples where NCI involvement in early stage of development resulted in products which eventually were licensed to commercial organizations and reached the market.

    Tamoxifen, Herceptin, Gleevec, Erbitux are examples of successful FDA approved drugs. For a complete list of drugs go to http://www.cancer.gov/cancertopics/druginfo/alphalist.

    Other examples include:


  16. What are some promising drugs under consideration now?

    NCI is supporting the development of drugs that target many biochemical pathways in the cell—genes involved in apoptosis, cell cycle control and cell signaling, angiogenesis, tumor invasion and metastasis, DNA synthesis and immune functions. Some of the promising agents and their biochemical pathways that are in early clinical development at NCI include:

    Antibodies to VEGF integrinsBMS-247550: cytotoxicity in paclitaxel-resistant tumor cell lines
    OSI-774 (OSI Pharmaceuticals) and Iressa (Astra Zeneca): inhibitors of growth factor signalingMS-275 and Vorinostat: histone deacetylase inhibitor
    CAI: anti-metastasis and anti-angiogenesis17-DMAG: heat shock protein inhibitor
    Rebeccamycin: topoisomerase inhibitoranakinra and enzastaurin: anti-angiogenesis
    Pyrrolobenzodiazepine: alters DNA functionSSI(dsFv)-PE38: immunotoxin
    O6 Benzylguanine: alters DNA repairPS-341: proteasome inhibitor
    Flavopiridol (Aventis), UCN-01 (Kyowa Hakko Kogyo), depsipeptide: cell cycleBay 43-9006: kinease inhibitor
    Rapamycin analogs (Wyeth-Ayerst): cell cycle functionMDX-010: protein antibody
    Halichondrin analogs (Eisai Co.): inhibits tubulin formationGW572016: cell cycle function
    KRN5500: mechanism uncertainAnti-Tac(Fv)-PE38 (LMB-2): cytotoxicity to cancer cells that overproduce CD-25 protein
    FR901228: binds to multi-drug resistance gene product 

  17. What are other sources of information about drug development at NCI?

    For DTP: http://dtp.nci.nih.gov/
    For CTEP: http://ctep.cancer.gov/
    For cancer clinical trials: http://www.cancer.gov/clinicaltrials/

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Glossary Terms

alkylating agent (AL-kuh-LAY-ting AY-jent)
A type of drug that is used in the treatment of cancer. It interferes with the cell's DNA and inhibits cancer cell growth.
anakinra (A-nuh-KIN-ruh)
A substance that is used to treat rheumatoid arthritis, and is being studied in the treatment of cancer. Anakinra blocks the action of interleukin 1 (IL-1). It is a type of interleukin receptor antagonist. Also called Kinaret.
analog (A-nuh-log)
In chemistry, a substance that is similar, but not identical, to another.
angiogenesis (AN-jee-oh-JEH-neh-sis)
Blood vessel formation. Tumor angiogenesis is the growth of new blood vessels that tumors need to grow. This is caused by the release of chemicals by the tumor.
antibody (AN-tee-BAH-dee)
A protein made by plasma cells (a type of white blood cell) in response to an antigen (a substance that causes the body to make a specific immune response). Each antibody can bind to only one specific antigen. The purpose of this binding is to help destroy the antigen. Some antibodies destroy antigens directly. Others make it easier for white blood cells to destroy the antigen.
apoptosis (A-pop-TOH-sis)
A type of cell death in which a series of molecular steps in a cell leads to its death. This is the body’s normal way of getting rid of unneeded or abnormal cells. The process of apoptosis may be blocked in cancer cells. Also called programmed cell death.
BAY 43-9006
A drug used to treat advanced kidney cancer and a type of liver cancer that cannot be removed by surgery. It is also being studied in the treatment of other types of cancer. BAY 43-9006 stops cells from dividing and may prevent the growth of new blood vessels that tumors need to grow. It is a type of kinase inhibitor and a type of antiangiogenesis agent. Also called Nexavar, sorafenib, and sorafenib tosylate.
BMS-247550
A drug used to treat metastatic or locally advanced breast cancer that has not improved after treatment with certain other anticancer drugs. It is also being studied in the treatment of other types of cancer. BMS-247550 stops the growth of tumor cells by blocking cell division. It is a type of epothilone analog. Also called ixabepilone and Ixempra.
breast (brest)
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.
cancer (KAN-ser)
A term for diseases in which abnormal cells divide without control and can invade nearby tissues. Cancer cells can also spread to other parts of the body through the blood and lymph systems. There are several main types of cancer. Carcinoma is a cancer that begins in the skin or in tissues that line or cover internal organs. Sarcoma is a cancer that begins in bone, cartilage, fat, muscle, blood vessels, or other connective or supportive tissue. Leukemia is a 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. Also called malignancy.
carboplatin (KAR-boh-pla-tin)
A drug that is used to treat advanced ovarian cancer that has never been treated or symptoms of ovarian cancer that has come back after treatment with other anticancer drugs. It is also used with other drugs to treat advanced, metastatic, or recurrent non-small cell lung cancer and is being studied in the treatment of other types of cancer. Carboplatin is a form of the anticancer drug cisplatin and causes fewer side effects in patients. It attaches to DNA in cells and may kill cancer cells. It is a type of platinum compound. Also called Paraplatin.
centimeter (SEN-tih-MEE-ter)
A measure of length in the metric system. There are 100 centimeters in a meter and 2½ centimeters in an inch.
cisplatin (sis-PLA-tin)
A drug used to treat many types of cancer. Cisplatin contains the metal platinum. It kills cancer cells by damaging their DNA and stopping them from dividing. Cisplatin is a type of alkylating agent.
clinical trial (KLIH-nih-kul TRY-ul)
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 clinical study.
colon (KOH-lun)
The longest part of the large intestine, which is a tube-like organ connected to the small intestine at one end and the anus at the other. The colon removes water and some nutrients and electrolytes from partially digested food. The remaining material, solid waste called stool, moves through the colon to the rectum and leaves the body through the anus.
compound (KOM-pownd)
In science, a substance that is made up of more than one ingredient.
cultured cell line (KUL-cherd sel line)
Cells of a single type (human, animal, or plant) that have been adapted to grow continuously in the laboratory and are used in research.
depsipeptide (DEP-see-PEP-tide)
A substance that is made naturally by some bacteria, fungi, and other organisms, and can also be made in the laboratory. Depsipeptides are being studied in the treatment of cancer.
diameter (dy-A-meh-ter)
The length of a straight line that extends from one edge of a tumor or other object, through its center and to the opposite edge. It is usually used to measure the size of round or spherical shapes.
DNA
The molecules inside cells that carry genetic information and pass it from one generation to the next. Also called deoxyribonucleic acid.
dose (dose)
The amount of medicine taken, or radiation given, at one time.
drug (drug)
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.
drug resistance (... reh-ZIH-stunts)
The failure of cancer cells, viruses, or bacteria to respond to a drug used to kill or weaken them. The cells, viruses, or bacteria may be resistant to the drug at the beginning of treatment, or may become resistant after being exposed to the drug.
efficacy (EH-fih-kuh-see)
Effectiveness. In medicine, the ability of an intervention (for example, a drug or surgery) to produce the desired beneficial effect.
enzastaurin (en-zuh-STAW-rin)
A substance being studied in the treatment of certain types of cancer, including non-Hodgkin lymphoma, breast, colon, lung, ovarian, and prostate. Enzastaurin blocks certain cell signaling pathways, and may prevent the growth of new blood vessels that tumors need to grow. It is a type of serine threonine kinase inhibitor and a type of antiangiogenesis agent. Also called enzastaurin hydrochloride and LY317615.
experimental (ek-SPAYR-ih-men-tul)
In clinical trials, refers to a drug (including a new drug, dose, combination, or route of administration) or procedure that has undergone basic laboratory testing and received approval from the U.S. Food and Drug Administration (FDA) to be tested in human subjects. A drug or procedure may be approved by the FDA for use in one disease or condition, but be considered experimental in other diseases or conditions. Also called investigational.
flavopiridol (FLAH-voh-PIH-rih-dol)
A substance being studied in the treatment of several types of cancer. It stops cells from dividing and may kill cancer cells. It is a type of cyclin-dependent kinase (CDK) inhibitor. Also called alvocidib and HMR 1275.
fludarabine (floo-DAR-uh-been)
The active ingredient in a drug used to treat B-cell chronic lymphocytic leukemia (CLL) that has not responded to treatment with other anticancer drugs or that has gotten worse. Fludarabine blocks cells from making DNA and may kill cancer cells. It is a type of purine antagonist and a type of ribonucleotide reductase inhibitor.
FR901228
A substance being studied in the treatment of cancer. FR901228 is a type of depsipeptide and histone deacetylase inhibitor. Also called romidepsin.
gene (jeen)
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.
generic (jeh-NAYR-ik)
Official nonbrand names by which medicines are known. Generic names usually refer to the chemical name of the drug.
Gleevec (GLEE-vek)
A drug used to treat different types of leukemia and other cancers of the blood, gastrointestinal stromal tumors, skin tumors called dermatofibrosarcoma protuberans, and a rare condition called systemic mastocytosis. It is also being studied in the treatment of other types of cancer. Gleevec blocks the protein made by the bcr/abl oncogene. It is a type of tyrosine kinase inhibitor. Also called imatinib mesylate and STI571.
growth factor (grothe FAK-ter)
A substance made by the body that functions to regulate cell division and cell survival. Some growth factors are also produced in the laboratory and used in biological therapy.
GW572016
A drug used with another anticancer drug to treat breast cancer that is HER2 positive and has advanced or metastasized (spread to other parts of the body) after treatment with other drugs. GW572016 is also being studied in the treatment of other types of cancer. It is a type of ErbB-2 and EGFR dual tyrosine kinase inhibitor. Also called lapatinib, lapatinib ditosylate, and Tykerb.
Herceptin (her-SEP-tin)
A drug used to treat breast cancer that is HER2-positive (expresses the human epidermal growth factor receptor 2). It is also used with other drugs to treat HER2-positive stomach cancer that has not already been treated and has spread to other parts of the body. It is being studied in the treatment of other types of cancer. Herceptin binds to HER2 on the surface of HER2-positive cancer cells, and may kill them. It is a type of monoclonal antibody. Also called trastuzumab.
histone deacetylase (HIS-tone dee-uh-SEH-tih-lays)
An enzyme that removes a small molecule called an acetyl group from histones (proteins found in chromosomes). This changes the way the histones bind to DNA and may affect its activity. Histone deacetylase inhibitors are being studied in the treatment of cancer. Also called HDAC.
hydroxyurea (hy-DROK-see-yoo-REE-uh)
An anticancer drug that belongs to the family of drugs called antimetabolites.
immunotoxin (IH-myoo-noh-TOK-sin)
An antibody linked to a toxic substance. Some immunotoxins can bind to cancer cells and kill them.
in vitro (in VEE-troh)
In the laboratory (outside the body). The opposite of in vivo (in the body).
in vivo (in VEE-voh)
In the body. The opposite of in vitro (outside the body or in the laboratory).
injection (in-JEK-shun)
Use of a syringe and needle to push fluids or drugs into the body; often called a "shot."
Institutional Review Board (IN-stih-TOO-shuh-nul reh-VYOO bord)
A group of scientists, doctors, clergy, and consumers that reviews and approves the action plan for every clinical trial. There is an at every health care facility that does clinical research. s are designed to protect the people who take part in a clinical trial. Institutional Review Boards check to see that the trial is well designed, legal, ethical, does not involve unneccesary risks, and includes safeguards for patients. Also called IRB.
IRB
A group of scientists, doctors, clergy, and consumers that reviews and approves the action plan for every clinical trial. There is an at every health care facility that does clinical research. s are designed to protect the people who take part in a clinical trial. IRBs check to see that the trial is well designed, legal, ethical, does not involve unneccesary risks, and includes safeguards for patients. Also called Institutional Review Board.
kidney (KID-nee)
One of a pair of organs in the abdomen. Kidneys remove waste from the blood (as urine), produce erythropoietin (a substance that stimulates red blood cell production), and play a role in blood pressure regulation.
KRN5500
An anticancer drug that is a type of antitumor antibiotic. It is an anthracycline.
lung (lung)
One of a pair of organs in the chest that supplies the body with oxygen, and removes carbon dioxide from the body.
lymphoma (lim-FOH-muh)
Cancer that begins in cells of the immune system. There are two basic categories of lymphomas. One kind is Hodgkin lymphoma, which is marked by the presence of a type of cell called the Reed-Sternberg cell. The other category is non-Hodgkin lymphomas, which includes a large, diverse group of cancers of immune system cells. Non-Hodgkin lymphomas can be further divided into cancers that have an indolent (slow-growing) course and those that have an aggressive (fast-growing) course. These subtypes behave and respond to treatment differently. Both Hodgkin and non-Hodgkin lymphomas can occur in children and adults, and prognosis and treatment depend on the stage and the type of cancer.
MDX-010
A drug used to treat melanoma that has spread to other parts of the body or that cannot be removed by surgery. It is also being studied in the treatment of other types of cancer. MDX-010 binds to a substance called CTLA-4, which is found on the surface of T cells (a type of white blood cell). MDX-010 may block CTLA-4 and help the immune system kill cancer cells. It is a type of monoclonal antibody. Also called ipilimumab and Yervoy.
melanoma (MEH-luh-NOH-muh)
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.
metastasis (meh-TAS-tuh-sis)
The spread of cancer from one part of the body to another. A tumor formed by cells that have spread is called a “metastatic tumor” or a “metastasis.” The metastatic tumor contains cells that are like those in the original (primary) tumor. The plural form of metastasis is metastases (meh-TAS-tuh-SEEZ).
millimeter (MIH-luh-MEE-ter)
A measure of length in the metric system. A millimeter is one thousandth of a meter. There are 25 millimeters in an inch.
mitoxantrone (MY-toh-ZAN-trone)
A drug used to treat advanced prostate cancer that does not respond to hormones, adult acute nonlymphocytic leukemia, and advanced or chronic multiple sclerosis. It is also being studied in the treatment of other cancers. It belongs to the family of drugs called antitumor antibiotics. Also called Novantrone.
MS-275
A substance that is being studied in the treatment of cancers of the blood. It belongs to the family of drugs called histone deacetylase inhibitors.
oncology (on-KAH-loh-jee)
The study of cancer.
oral (OR-ul)
By or having to do with the mouth.
ovarian (oh-VAYR-ee-un)
Having to do with the ovaries, the female reproductive glands in which the ova (eggs) are formed. The ovaries are located in the pelvis, one on each side of the uterus.
paclitaxel (PA-klih-TAK-sil)
A drug used to treat breast cancer, ovarian cancer, and AIDS-related Kaposi sarcoma. It is also used together with another drug to treat non-small cell lung cancer. Paclitaxel is also being studied in the treatment of other types of cancer. It blocks cell growth by stopping cell division and may kill cancer cells. It is a type of antimitotic agent. Also called Taxol.
pentostatin (PEN-toh-STA-tin)
The active ingredient in a drug that is used to treat hairy cell leukemia and is being studied in the treatment of other types of cancer. Pentostatin blocks a protein needed for cell growth and may kill cancer cells. It is made by a bacterium. It is a type of adenosine deaminase inhibitor. Also called Nipent.
phase I trial (fayz … TRY-ul)
The first step in testing a new treatment in humans. These studies test the best way to give a new treatment (for example, by mouth, intravenous infusion, or injection) and the best dose. The dose is usually increased a little at a time in order to find the highest dose that does not cause harmful side effects. Because little is known about the possible risks and benefits of the treatments being tested, phase I trials usually include only a small number of patients who have not been helped by other treatments.
phase II trial (fayz … TRY-ul)
A study to test whether a new treatment has an anticancer effect (for example, whether it shrinks a tumor or improves blood test results) and whether it works against a certain type of cancer.
phase III trial (fayz … TRY-ul)
A study to compare the results of people taking a new treatment with the results of people taking the standard treatment (for example, which group has better survival rates or fewer side effects). In most cases, studies move into phase III only after a treatment seems to work in phases I and II. Phase III trials may include hundreds of people.
placebo (pluh-SEE-boh)
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.
prostate (PROS-tayt)
A gland in the male reproductive system. The prostate surrounds the part of the urethra (the tube that empties the bladder) just below the bladder, and produces a fluid that forms part of the semen.
proteasome inhibitor (PROH-tee-uh-some in-HIH-bih-ter)
A drug that blocks the action of proteasomes. A proteasome is a large protein complex that helps destroy other cellular proteins when they are no longer needed. Proteasome inhibitors are being studied in the treatment of cancer.
protein (PROH-teen)
A molecule made up of amino acids that are needed for the body to function properly. Proteins are the basis of body structures such as skin and hair and of substances such as enzymes, cytokines, and antibodies.
protocol (PROH-tuh-KOL)
A detailed plan of a scientific or medical experiment, treatment, or procedure. In clinical trials, it states what the study will do, how it will be done, and why it is being done. It explains how many people will be in the study, who is eligible to take part in it, what study drugs or other interventions will be given, what tests will be done and how often, and what information will be collected.
PS-341
A drug used to treat multiple myeloma. It is also used to treat mantle cell lymphoma in patients who have already received at least one other type of treatment and is being studied in the treatment of other types of cancer. PS-341 blocks several molecular pathways in a cell and may cause cancer cells to die. It is a type of proteasome inhibitor and a type of dipeptidyl boronic acid. Also called bortezomib and velcade.
rapamycin (RA-puh-MY-sin)
A drug used to keep the body from rejecting organ and bone marrow transplants. Rapamycin blocks certain white blood cells that can reject foreign tissues and organs. It also blocks a protein that is involved in cell division. It is a type of antibiotic, a type of immunosuppressant, and a type of serine/threonine kinase inhibitor. Rapamycin is now called sirolimus.
rebeccamycin (reh-BEH-kuh-MY-sin)
A substance that is being studied in the treatment of cancer. It belongs to the family of drugs called antineoplastic antibiotics.
schedule (SKEH-jool)
In a clinical setting, the step-by-step plan for how patients are to be treated; for example, the drug or type of radiation therapy that is to be given, the method by which it is to be given, the amount of time between courses, and the total length of treatment.
side effect (side eh-FEKT)
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.
standard therapy (... THAYR-uh-pee)
Treatment that experts agree is appropriate, accepted, and widely used. Also called best practice, standard medical care, and standard of care.
tamoxifen (tuh-MOK-sih-FEN)
A drug used to treat certain types of breast cancer in women and men. It is also used to prevent breast cancer in women who have had ductal carcinoma in situ (abnormal cells in the ducts of the breast) and in women who are at a high risk of developing breast cancer. Tamoxifen is also being studied in the treatment of other types of cancer. It blocks the effects of the hormone estrogen in the breast. Tamoxifen is a type of antiestrogen. Also called tamoxifen citrate.
testicle (TES-tih-kul)
One of two egg-shaped glands inside the scrotum that produce sperm and male hormones. Also called testis.
topoisomerase inhibitor (TOH-poh-i-SAH-meh-rays in-HIH-bih-ter)
A substance that blocks topoisomerases (enzymes that break and rejoin DNA strands and are needed for cells to divide and grow). Blocking these enzymes may kill cancer cells. Certain topoisomerase inhibitors are being studied in the treatment of cancer.
topotecan (toh-poh-TEE-kan)
A drug used to treat certain types of ovarian cancer, lung cancer, and cervical cancer. Topotecan is a type of topoisomerase inhibitor. Also called Hycamtin and topotecan hydrochloride.
toxicity (tok-SIH-sih-tee)
The extent to which something is poisonous or harmful.
tumor (TOO-mer)
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 cancer), or malignant (cancer). Also called neoplasm.
UCN-01
An anticancer drug that belongs to the family of drugs called staurosporine analogs.
VEGF
A substance made by cells that stimulates new blood vessel formation. Also called vascular endothelial growth factor.

Table of Links

1http://www.cancer.gov/cancertopics/factsheet/NCI/clinical-trials-cooperative-gr
oup
2http://www.cancer.gov/cancertopics/factsheet/Information/clinical-trials
3http://cancercenters.cancer.gov
4http://www.cancer.gov/clinicaltrials
5http://dcp.cancer.gov/programs-resources/programs/ccop
6http://dtp.nci.nih.gov