Treatment in a clinical trial

Different types of treatment are available for children with visual pathway glioma. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment.

Because cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment

Four kinds of standard treatment are used:

• Watchful waiting (closely monitoring a patient's condition without giving any treatment until symptoms appear or change).



• Surgery (taking out the cancer in an operation).



• Radiation therapy (using high- dose x-rays to kill cancer cells).



• Chemotherapy (using drugs to kill cancer cells).

Experienced doctors working together can often give the best treatment for children with visual pathway glioma. Your child’s treatment will often be coordinated by a pediatric oncologist, a doctor who specializes in cancer in children. The pediatric oncologist may refer you to other doctors, such as a pediatric neurosurgeon (a specialist in childhood brain surgery), a pediatric neurologist, a psychologist, a radiation oncologist, and other doctors who specialize in the type of treatment your child requires.

Surgery is one treatment for visual pathway glioma. Depending on where the cancer is and the type of cancer, your child’s doctor may remove as much of the tumor as possible. If the tumor cannot be totally removed, radiation therapy and chemotherapy may also be given. If the cancer is in a place where it cannot be removed, surgery may be limited to a biopsy of the cancer.

Radiation therapy uses high-energy x-rays to kill cancer cells and shrink tumors. Radiation therapy for childhood brain tumors usually comes from a machine outside the body (external radiation therapy). Because radiation therapy can affect growth and brain development, clinical trials are testing ways to decrease or delay radiation therapy, especially for younger children. These include internal radiation therapy, in which radiation is put into the brain through thin plastic tubes, and hyperfractionated radiation therapy, in which radiation therapy is given in several small doses per day instead of all at once. Conformal radiation therapy uses a computer to create a 3-D picture of the tumor and the radiation beams are shaped to fit the tumor; this helps to keep the radiation away from healthy tissue as much as possible.

Chemotherapy uses drugs to kill cancer cells. Chemotherapy may be taken by pill, or it may be put into the body by a needle in a vein or muscle. Chemotherapy is called a systemic treatment because the drug enters the bloodstream, travels through the body, and can kill cancer cells throughout the body. Chemotherapy is being studied to delay the use of radiation therapy in some patients. Clinical trials are studying different chemotherapy drugs for visual pathway gliomas.

Some brain tumors and cancer treatments cause side effects that continue or appear years after cancer treatment has ended. These are called late effects. Late effects of cancer treatment may include physical problems; changes in mood, feelings, thinking, learning or memory; and having second cancers (new types of cancer). Some late effects may be treated or controlled. It is important to talk with your child's doctors about the possible late effects caused by some treatments. Refer to the PDQ summary on Late Effects of Treatment for Childhood Cancer ¹ for more information.

For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment.

Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment.

Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward.

Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment.

Clinical trials are taking place in many parts of the country. In the following lists of treatments for the different stages, a link to search results for current clinical trials is included for each section. These have been retrieved from NCI's clinical trials database. For some types or stages of cancer, there may not be any trials listed. Check with your child's doctor for clinical trials that are not listed here but may be right for your child.

Glossary Terms

3-dimensional. A graphic display of depth, width, and height. Also called 3-dimensional.

A procedure that uses a computer to create a 3-dimensional picture of the tumor. This allows doctors to give the highest possible dose of radiation to the tumor, while sparing the normal tissue as much as possible. Also called 3-dimensional radiation therapy.

The removal of cells or tissues for examination by a pathologist. The pathologist may study the tissue under a microscope or perform other tests on the cells or tissue. There are many different types of biopsy procedures. The most common types include: (1) incisional biopsy, in which only a sample of tissue is removed; (2) excisional biopsy, in which an entire lump or suspicious area is removed; and (3) needle biopsy, in which a sample of tissue or fluid is removed with a needle. When a wide needle is used, the procedure is called a core biopsy. When a thin needle is used, the procedure is called a fine-needle aspiration biopsy.

The growth of abnormal cells in the tissues of the brain. Brain tumors can be benign (non-cancerous) or malignant (cancerous).

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.

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.

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.

A type of radiation therapy that uses a machine to aim high-energy rays at the cancer from outside of the body. Also called external-beam radiation therapy.

Radiation therapy that gives smaller doses (fractions) of radiation more often than standard radiation therapy so that the full treatment course can be given with fewer side effects. In hyperfractionation, individual doses are given more often than the standard dose of once a day. Also called hyperfractionated radiation therapy and superfractionated radiation therapy.

A type of radiation therapy in which radioactive material sealed in needles, seeds, wires, or catheters is placed directly into or near a tumor. Also called brachytherapy, radiation brachytherapy, and implant radiation therapy.

Side effects of cancer treatment that appear months or years after treatment has ended. Late effects include physical and mental problems and second cancers.

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. It 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 http://www.cancer.gov. Also called National Cancer Institute.

A doctor who specializes in the diagnosis and treatment of disorders of the nervous system.

A doctor who specializes in surgery on the brain, spine, and other parts of the nervous system.

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.

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.

Having to do with children.

A specialist who can talk with patients and their families about emotional and personal matters, and can help them make decisions.

Energy released in the form of particle or electromagnetic waves. Common sources of radiation include radon gas, cosmic rays from outer space, medical x-rays, and energy given off by a radioisotope (unstable form of a chemical element that releases radiation as it breaks down and becomes more stable).

A doctor who specializes in using radiation to treat cancer.

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.

To come back or to return.

Refers to a new primary cancer in a person with a history of cancer.

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.

The extent of a cancer in the body. Staging is usually based on the size of the tumor, whether lymph nodes contain cancer, and whether the cancer has spread from the original site to other parts of the body.

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.

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

Treatment using substances that travel through the bloodstream, reaching and affecting cells all over the body.

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 rare, slow-growing tumor that usually forms in the optic nerve, optic chiasm, or optic tract. These are parts of the nervous system that carry messages from the eye to the brain. Also called optic pathway glioma.

Closely monitoring a patient's condition but withholding treatment until symptoms appear or change. Also called observation.

A type of high-energy radiation. In low doses, x-rays are used to diagnose diseases by making pictures of the inside of the body. In high doses, x-rays are used to treat cancer.