What is head and neck cancer?

The basics.

In head and neck cancer, certain cells in the mouth, nose, sinuses, salivary glands, throat, or the lymph nodes in the neck grow uncontrollably, forming a solid mass called a tumor. Head and neck cancer occurs in three basic types: early-stage, metastatic, and recurrent:

• In early-stage head and neck cancer, the tumor is confined to the place it started. It may not have spread at all, or it may have spread to the lymph nodes
• In metastatic head and neck cancer, the disease has spread from its original location to other parts of the body
• A cancer is called recurrent if it returns in a person who seemed to be disease-free after their treatment

Where are some of the places in the head and neck that cancer can develop?

Most tissues in the body can become cancerous, including the tissues that make up our heads and necks. When cancer occurs in the neck, it may start in the throat (the pharynx), on the voice box (larynx), or on the piece of cartilage (epiglottis) that acts as the “lid” of the windpipe to prevent food from going into it.

Head and neck cancer can start in the nose or the hollow areas behind the nose (the paranasal sinuses). And, in the mouth, cancer can affect the tongue or gums, as well as the salivary glands.

The lymphatic system is also sometimes a cancer site. Consisting of lymph nodes and lymphatic vessels, the lymphatic system stores and moves immune system cells.

How common are head and neck cancers?

Cancers of the head and neck make up about 3% to 5% of all cancers in the United States. They’re more common in people over the age of 50 and those who use tobacco.

The American Academy of Otolaryngology-Head and Neck Surgery (AAO-HNS) estimated that in 2009, about 55,000 men and women will develop head and neck cancer in the United States.

Of the new diagnoses of head and neck cancer made each year, the American Cancer Society estimates that about 24,000 are of specifically throat cancers. About half of those are cancers of the pharynx, the other half being cancers of the larynx.

Overview

How Does a Blood Stem Cell Transplant Work?

Blood stem cell transplantation may help cure cancers such as leukemia, Hodgkin's disease and other lymphomas. Blood stem cell transplants allow physicians to replace a patient's diseased or damaged marrow with healthy blood-forming cells. First, patients receive high doses of chemotherapy and/or radiation to destroy the cancer cells; however, along with the cancer, normal cells in the marrow are also destroyed. So chemotherapy and radiation are followed by an infusion into the bloodstream of healthy donor blood stem cells. The transplanted cells migrate into the spaces inside the bones to create new marrow, which contains cells -- known as hematopoietic (blood) stem cells -- that have the ability to grow and make healthy new red blood cells, white blood cells, and platelets in a process known as engraftment. (Hematopoietic stem cells differ from embryonic stem cells, which are primitive cells derived from embryos.)

The body's ability to produce enough white blood cells to regenerate the immune system and prevent infections is vital for a successful transplantation. In the 1970s, Memorial Sloan-Kettering Cancer Center investigator Malcolm A.S. Moore and his Center colleagues isolated granulocyte colony stimulating factor (G-CSF), a naturally occurring protein that encourages bone marrow to produce more white blood cells. G-CSF is also used prior to stem cell collection to mobilize, or stimulate, stem cells to move from the marrow into the circulating blood, boosting the number available for transplantation.

To reduce the toxicity and increase the anticancer activity of high-dose radiation, Memorial Sloan-Kettering Cancer Center scientists created a technique in 1978 called hyperfractionated total body irradiation, which allows patients to receive lower doses of radiation two or three times a day over several days prior to the transplantation, instead of receiving the entire high dose at one time.

Patients can receive a blood stem cell infusion in one of two ways: using their own stem cells or those from a donor. Autologous transplantation uses the patient's own marrow, which is removed from the patient's body and reinfused into the bloodstream. Allogeneic transplantation uses cells obtained from a donor -- either a family member or an unrelated individual -- whose tissue type closely matches that of the recipient.

When donor blood stem cells are transplanted into a patient to create new marrow and blood cells, there is a possibility of graft failure, in which donor cells may be rejected by the recipient. To prevent this complication, powerful immunosuppressive drugs, such as antithymocyte globulin, are given to the patient to ensure successful engraftment.

A major complication of allogeneic transplantation is graft-versus-host-disease (GvHD), in which some of the donor's immune cells, or T cells, attack cells in the recipient's body in areas such as the skin, liver, and gastrointestinal tract. There are two types of GvHD -- acute GvHD, which occurs within the first months after transplantation, and chronic GvHD, which happens later and has some similarities with an autoimmune disease.

When GvHD was first characterized, Memorial Sloan-Kettering Cancer Center researchers recognized that it was caused by T cells in the transplant. Reducing GvHD has been a major focus of the BMT Service. In 1981, the Memorial Sloan-Kettering Cancer Center transplantation program developed and introduced a new approach called T cell depletion. T cells, which develop in the thymus, are selectively removed from donor blood stem cells prior to their infusion, significantly reducing the chance that these donor T cells will attack the patient's body, thereby preventing GvHD, even when donor and recipient are only half HLA-matched. Following the transplant, new T cells develop from the donor's blood stem cells, providing a new immune system for the patient.

Diseases Treated with Bone Marrow Transplants (by Type of Transplant)

Autologous Transplants

Samples of the patient's own bone marrow or peripheral blood stem cells can be removed, frozen, and stored to be returned after chemotherapy or radiation therapy. Diseases treated in this manner include:

• Brain Tumors
• Ewing's Sarcoma
• Neuroblastoma
• Rhabdomyosarcoma
• Hodgkin's Disease
• Non-Hodgkin's Lymphoma

Allogeneic Transplants

In allogeneic transplants, the patient's bone marrow is replaced with marrow from a donor who is either a family member or a matched, unrelated donor. Our doctors provide allogeneic transplants for a wide range of blood diseases, including:

Hematologic Malignancies (Cancers of the Blood)

• Acute Lymphoblastic Leukemia
• Acute Myelogenous Leukemia
• Chronic Myelogenous Leukemia
• Juvenile Myelomonocytic Leukemia
• Myelodysplastic Syndromes
• Non-Hodgkin's Lymphoma

Genetic Diseases

• Hemoglobinopathies: sickle cell disease, thalassemia
• Severe combined immune deficiency
• Fanconi's Anemia
• Wiskott-Aldrich Syndrome

Other Blood Disorders

• Amegakaryocytic Thrombocytopenia
• Aplastic Anemia
• Diamond Blackfan Anemia
• Hemophagocytic Lymphohistiocytosis
• Kostmann Syndrome

Types of Transplants

The types of transplants include bone marrow, peripheral blood, and cord blood transplants using HLA-matched sibling donors, unrelated donors, and family mismatched donors.

• Bone Marrow
• Peripheral blood
• Cord blood transplants

Phases of a Transplant

Before Transplant

A successful transplant requires the patient be healthy enough to undergo the rigors of the transplant procedure. Age, general physical condition, the patient's diagnosis and the stage of the disease are all considered by the physician when determining whether a person should undergo a transplant.

Prior to a bone marrow transplant, a battery of tests is carried out to ensure the patient is physically capable of undergoing a transplant. Tests of the patient's heart, lung, kidney and other vital organ functions are also used to develop a patient "baseline" against which post-transplant tests can be compared to determine if any body functions have been impaired. The pre-transplant tests are usually done on an outpatient basis.

A successful bone marrow transplant requires an expert medical team - doctors, nurses, and other support staff - who are experienced in bone marrow transplants, can promptly recognize problems and emerging side effects, and know how to react swiftly and properly if problems do arise. A good bone marrow transplant program will also recognize the importance of providing patients and their families with emotional and psychological support before, during and after the transplant, and will make personal and other support systems readily available to families for this purpose.

Prior to conditioning, a small flexible tube called a catheter (sometimes called a "Hickman, Broviac" or central venous line) will be inserted into a large vein in the patient's chest just above the heart. This tube enables the medical staff to administer drugs and blood products to the patient painlessly, and to withdraw the hundreds of blood samples required during the course of treatment without inserting needles into the patient's arms or hands.

A patient admitted to the bone marrow transplant unit will first undergo several days of chemotherapy and/or radiation which destroys bone marrow and cancerous cells and makes room for the new bone marrow. This is called the conditioning or preparative regimen. The exact regimen of chemotherapy and/or radiation varies according to the disease being treated and the "protocol" or preferred treatment plan of the facility where the BMT is being performed.

The dosage of chemotherapy and/or radiation given to patients during conditioning is much stronger than dosages administered to patients with the same disease who are not undergoing a BMT. Patients may become weak, irritable and nauseous. Most BMT centers administer anti-nausea medications to minimize discomfort.

Donor or HLA Match

If bone marrow from a donor is used, the transplant is called an "allogeneic" BMT, or "syngeneic" BMT if the donor is an identical twin. In an allogeneic BMT, the new bone marrow infused into the patient must match the genetic makeup of the patient's own marrow as perfectly as possible. Special blood tests are conducted to determine whether or not the donor's bone marrow matches the patient's. If the donor's bone marrow is not a good match, it will perceive the patient's body as foreign material to be attacked and destroyed. This condition is known as graft-versus-host disease (GVHD) and can be life-threatening. Alternatively, the patient's immune system may destroy the new bone marrow. This is called graft rejection.

There is a 35 percent chance that a patient will have a sibling whose bone marrow is a perfect match. If the patient has no matched sibling, a donor may be located in one of the international bone marrow donor registries, or a mis-matched or autologous transplant may be considered.

In some cases, patients may be their own bone marrow donors. This is called an autologous BMT and is possible if the disease afflicting the bone marrow is in remission or if the condition being treated does not involve the bone marrow (e.g. breast cancer, ovarian cancer, Hodgkin's disease, non-Hodgkin's lymphoma, and brain tumors). The bone marrow is extracted from the patient prior to transplant and may be "purged" to remove lingering malignant cells (if the disease has afflicted the bone marrow).

Bone Marrow Harvest

Regardless of whether the patient or a donor provides the bone marrow used in the transplant, the procedure used to collect the marrow - the bone marrow harvest - is the same. The bone marrow harvest takes place in a hospital operating room, usually under general anesthesia. It involves little risk and minimal discomfort.

While the patient is under anesthesia, a needle is inserted into the cavity of the rear hip bone or "iliac crest" where a large quantity of bone marrow is located. The bone marrow a thick, red liquid - is extracted with a needle and syringe. Several skin punctures on each hip and multiple bone punctures are usually required to extract the requisite amount of bone marrow. There are no surgical incisions or stitches involved - only skin punctures where the needle was inserted.

The amount of bone marrow harvested depends on the size of the patient and the concentration of bone marrow cells in the donor's blood. Usually one to two quarts of marrow and blood are harvested. While this may sound like a lot, it really only represents about 2% of a person's bone marrow, which the body replaces in four weeks.

When the anesthesia wears off, the donor may feel some discomfort at the harvest site. The pain will be similar to that associated with a hard fall on the ice and can usually be controlled with Tylenol. Donors who are not also the BMT patient are usually discharged after an overnight stay and can fully resume normal activities in a few days.

For autologous transplants, the harvested bone marrow will be frozen (cryopreserved) and stored at a temperature between -80 and -196 degrees centigrade until the day of transplant. It may first be "purged" to remove residual cancerous cells that can't be easily identified under the microscope (see page 30).

In allogeneic BMTs, the bone marrow may be treated to remove "T-cells" (T cell depletion) to reduce the risk of graft-versus-host disease (see page 94). It will then be transferred directly to the patient's room for infusion.

During Transplant

During a bone marrow transplant the bone marrow is infused intravenously into the patient, in much the same way that any blood product is administered.

Even though the transplant lasts a short amount of time, the patient must stay in an isolated hospital room for two to four weeks after the transplant. The high-dose chemotherapy and/or radiation given to the patient during conditioning will have destroyed the patient's bone marrow, crippling the body's "immune" or defense system. As the patient waits for the transplanted bone marrow to migrate to the cavities of the large bones, set up housekeeping or "engraft," and begin producing normal blood cells, he or she will be very susceptible to infection and excessive bleeding. Multiple antibiotics and blood transfusions will be administered to the patient to help prevent and fight infection. Transfusions of platelets will be given to prevent bleeding. Allogeneic patients will receive additional medications to prevent and control graft-versus-host disease.

Extraordinary precautions will be taken to minimize the patient's exposure to viruses and bacteria. Visitors and hospital personnel will wash their hands with antiseptic soap and, in some cases, wear protective gowns, gloves and/or masks while in the patient's room. Fresh fruits, vegetables, plants and cut flowers will be prohibited in the patient's room since they often carry fungi and bacteria that pose a risk of infection. When leaving the room, the patient may wear a mask, gown and gloves as a barrier against bacteria and virus, and as a reminder to others that he or she is susceptible to infection.

A bone marrow transplant is a physically, emotionally, and psychologically taxing procedure for both the patient and family. A patient needs and should seek as much help as possible to cope with the experience. "Toughing it out" on your own is not the smartest way to cope with the transplant experience.

The bone marrow transplant is a debilitating experience. Imagine the symptoms of a severe case of the flu - nausea, vomiting, fever, diarrhea, extreme weakness. Now imagine what it's like to cope with the symptoms not just for several days, but for several weeks. That approximates what a BMT patient experiences during hospitalization.

During this period the patient will feel very sick and weak. Walking, sitting up in bed for long periods of time, reading books, talking on the phone, visiting with friends or even watching TV may require more energy than the patient as to spare.

Complications can develop after a bone marrow transplant such as infection, bleeding, graft-versus-host disease, or liver disease, which can create additional discomfort. The pain, however, is usually controllable by medication. In addition, mouth sores can develop that make eating and swallowing uncomfortable. Temporary mental confusion sometimes occurs and can be quite frightening for the patient who may not realize it's only temporary. The medical staff will help the patient deal with these problems.

Handling Emotional Stress

In addition to the physical discomfort associated with the transplant experiance there is emotional and psychological discomfort as well. Some patients find the emotional and psychological stress more problematic than the physical discomfort.

The psychological and emotional stress stems from several factors. First, patients undergoing transplants are already traumatized by the news that they have a life-threatening disease. While the transplant offers hope for their recovery, the prospect of undergoing a long, arduous medical procedure is still not pleasant and there's no guarantee of success.

Second, patients undergoing a transplant can feel quite isolated. The special precautions taken to guard against infection while the immune system is impaired can leave a patient feeling detached from the rest of the world and cut off from normal human contact. The patient is housed in a private room, sometimes with special air-filtering equipment to purify the air. The number of visitors is restricted and visitors are asked to wear gloves, masks and/or other protective clothing to inhibit the spread of bacteria and virus while visiting the patient. When the patient leaves the room, he or she may be required to wear a protective mask, gown and/or gloves as a barrier against infection. This feeling of isolation comes at the very time in a patient's life when familiar surroundings and close physical contact with family and friends are most needed.

'Helplessness" is also a common feeling among bone marrow transplant patients, which can breed further feelings of anger or resentment. For many, it's unnerveing to be totally dependent on strangers for survival, no matter how competent they may be. The fact that most patients are unfamiliar with the medical jargon used to describe the transplant procedure compounds the feeling of helplessness. Some also find it embarrassing to be dependent on strangers for help with basic daily functions such as using the washroom.

The long weeks of waiting for the transplanted marrow to engraft, for blood counts to return to safe levels, and for side effects to disappear increase the emotional trauma. Recovery can be like a roller coaster ride: one day a patient may feel much better, only to awake the next day feeling as sick as ever.

After Transplant

Leaving the Hospital

After being discharged from the hospital, a patient continues recovery at home (or at lodging near the transplant center if the patient is from out of town) for two to four months. Patients usually cannot return to full-time work for up to six months after the transplant.

Though patients will be well enough to leave the hospital, their recovery will be far from over. For the first several weeks the patient may be too weak to do much more than sleep, sit up, and walk a bit around the house. Frequent visits to the hospital or associated clinic will be required to monitor the patient's progress, and to administer any medications and/or blood products needed. It can take six months or more from the day of transplant before a patient is ready to fully resume normal activities.

During this period, the patient's white blood cell counts are often too low to provide normal protection against the viruses and bacteria encountered in everyday life. Contact with the general public is therefore restricted. Crowded movie theaters, grocery stores, department stores, etc. are places recovering BMT patients avoid during their recuperation. Often patients will wear protective masks when venturing outside the home.

A patient will return to the hospital or clinic as an outpatient several times a week for monitoring, blood transfusions, and administration of other drugs as needed. Eventually, the patient becomes strong enough to resume a normal routine and to look forward to a productive, healthy life.

Life after Transplant

It can take as long as a year for the new bone marrow to function normally. Patients are closely monitored during this time to identify any infections or complications that may develop.

Life after transplant can be both exhilarating and worrisome. On the one hand, it's exciting to be alive after being so close to death. Most patients find their quality of life improved after transplant.

Nonetheless, there is always the worry that relapse will occur. Furthermore, innocent statements or events can sometimes conjure up unpleasant memories of the transplant experience long after the patient has recovered. It can take a long time for the patient to come to grips with these difficulties.

Is It Worth It?

Yes! For most patients contemplating a bone marrow transplant, the alternative is near-certain death. Despite the fact that the transplant can be a trying experience, most find that the pleasure that comes from being alive and healthy after the transplant is well worth the effort.

Survivorship & Support

People who have undergone a stem cell transplant need to be assessed regularly by their physicians. The treatment team will closely monitor allogeneic transplant patients for signs of graft-versus-host disease (GvHD), and will monitor patients who have received either allogeneic or autologous transplants for immune system recovery, late complications arising from the agents used in the cytoreductive regimen, and any recurrence of cancer.

Physical Challenges

Patients may experience a range of physical symptoms while recovering from a marrow or stem cell transplant. These can include fatigue and weakness, distorted taste sensations, lingering nausea and diarrhea, hair loss or other changes in appearance, and loss of muscle tone. Because your immune system may not recover fully for some time, you will be at risk for infections and colds.

In some patients, cancer may recur following transplantation if the high-dose chemotherapy and radiation given before the transplant did not eliminate all the malignant cells, or if the autologous stem cells harvested before the cytotoxic therapy contained some cancer cells. In an allogeneic transplant, the graft-versus-leukemia process may not always protect the patient from relapse, especially if he or she had relapsed or advanced disease at the time of transplantation. When a patient's cancer recurs, physicians may shift their treatment strategy to a next-line therapy. In rare instances, patients may develop a secondary cancer or condition such as a myelodysplastic syndrome as a result of the high-dose treatment.

Our Allogeneic Stem Cell Transplant Survivorship Program

	Christine Iovino, NP

Memorial Sloan-Kettering Cancer Center has incorporated an Allogeneic Stem Cell Transplant Survivorship Program into the overall care plan for our patients. This program, part of Memorial Sloan-Kettering's institution-wide survivorship initiative, provides services that address the unique needs of survivors in the years following treatment, including:

• Review of recent medical history and physical examination
• Assessment to detect recurrence of cancer
• Identification and management of the effects of cancer and treatment
• Screening referrals for other cancers
• Health promotion recommendations, such as nutrition, exercise, and smoking cessation
• Development of treatment summary and follow-up care plan
• Communication with the primary care physician

Individuals in the Survivorship Program have regularly scheduled follow-up examinations with Christine Iovino, a nurse practitioner with special training in transplant survivorship issues. Ms. Iovino is a graduate of Columbia University's nurse practitioner program and has worked at Memorial Sloan-Kettering since 1998, including her five years as a nurse practitioner on the inpatient transplant service. She is a member of the Allogeneic Stem Cell Transplant Disease Management Team and leads our efforts to provide optimal care to patients following their treatment.

Emotional & Social Challenges

The period after a bone marrow or stem cell transplant can be a difficult and stressful time. To avoid infections, patients need to avoid crowds and people who are sick. They may feel somewhat isolated. Some people find it hard to get back to work. They may experience financial difficulties and may not be fully aware of their employment and insurance rights. Concerns about changes in their appearance and infertility may complicate social and sexual relationships and dating. These can all lead to depression and anger.

Our social workers counsel patients on how to cope with the stresses of illness and how to communicate with family members and children, and work with them on other fundamental concerns. During hospitalization, our inpatient bone marrow transplant social worker provides counseling for individuals, families, and couples, and facilitates a weekly support group for caregivers. During outpatient visits, a bone marrow transplant social worker can meet with you or your family members, or with everyone together in a family meeting. The outpatient social worker is also available to patients who have been discharged.

Other Aspects of Survivorship

To help patients manage some of the other issues that may come up in survivorship, Memorial Sloan-Kettering offers a wide range of programs for cancer survivors, including the following:

Memorial Sloan-Kettering Counseling Center

Our Counseling Center offers individual and family counseling sessions to help cancer survivors and their families address the problems they may encounter in adjusting to life after treatment.

Living Beyond Cancer

People facing the challenges of cancer survivorship can access a variety of support services through the Living Beyond Cancer section of our Web site. This section includes information about professionally led support groups and counseling; survivorship clinics to manage late effects of cancer and treatment; recommendations about screening and healthy living; and specialized services to address sexual health. We also offer a range of resources to help survivors, families, and friends better understand the complex emotional and social issues following treatment.

Additional services are offered through our Resources for Life After Cancer program, which provides a full range of educational support services, including individual and family counseling, periodic lectures or workshops to provide medical updates, and practical guidance on employment and insurance issues.

Integrative Medicine Service

Our Integrative Medicine Service is designed to enhance quality of life through healing regimens that address the body, mind, and spirit. Beneficial complementary therapies include various types of massage, acupuncture, hypnotherapy, meditation, visualization and other mind-body therapies, music therapy, and nutritional counseling, as well as classes such as yoga, t'ai chi, and chair aerobics. Patients may benefit from some of these services in the hospital, while others are better suited to recovering or recovered patients after discharge.

About Stem Cell Transplantation

In a stem cell transplant -- also called bone marrow transplant -- a patient's blood-forming stem cells are replaced by infusing new ones into his or her bloodstream. The procedure can be an effective form of treatment for certain forms of cancer, specific genetic diseases, and other types of blood disorders. For most patients, the aim of the transplant is to cure their disease.

What Blood-Forming Stem Cells Are and Where They Are Found

Blood-forming or hematopoietic stem cells are immature cells that can develop into any type of blood cells, including:

• white blood cells, which help the body fight infections
• red blood cells, which carry oxygen throughout the body
• platelets, which are important in blood clotting and the control of bleeding.

The stem cells are produced in the bone marrow, a sponge-like tissue that is found inside large bones (for example, in the breastbone, pelvis, ribs, and spine). Before a transplant, stem cells can be collected from a person's bone marrow or from the bloodstream, where so-called peripheral blood stem cells (PBSC) circulate.

Another source of stem cells is blood from a newborn's umbilical cord and placenta, which can be donated to a stem cell bank by the baby's mother.

The Two Main Types of Transplant and How They Work

The are two main types of transplant: autologous ("auto") transplant, in which a patient's own stem cells are collected and then transplanted back into the patient, and allogeneic ("allo") transplant, in which the stem cells are obtained from a person other than the patient, most commonly a brother or sister.

Before an autologous or allogeneic transplant, either chemotherapy or a combination of chemotherapy and radiation is given. This treatment, called the preparative regimen or conditioning, eradicates cancerous cells as well as the blood-forming stem cells in the bone marrow, causing the number of mature blood cells to drop.

Next, stem cells are infused into the patient's bloodstream through an intravenous catheter, in a procedure that is similar to a blood transfusion. No surgery is required. Over the following days, the transplanted stem cells travel to the bone marrow, where they will grow and develop into new mature blood cells, including red and white blood cells and platelets.

It usually takes several weeks before all the mature blood cells are replenished. During this time, special measures are taken to protect the patient from infections and bleeding.

Successful autologous and allogeneic transplants provide patients with a new, healthy bone marrow. Allogeneic transplants also give patients a new immune system, which is derived from the donor's stem cells and may give protection from cancer.

Diseases Treated with Transplantation

Different types of transplants are known to be more effective for some diseases than others. Your team of doctors will determine which type of transplant is best for you after considering the specifics of your disease and a number of other factors -- for instance, what other treatments you have received and your general health status. Your transplant doctor will discuss these factors in detail with you.

Here are some of the most common diseases treated with autologous or allogeneic transplantation:

Diagnosis	Allogeneic Transplant/ Autologous Rescue
Amegakaryocytosis/Congenital Thrombocytopenia	Allogeneic
Amyloidosis	Autologous
Aplastic Anemia/Refractory Anemia	Allogeneic
Germ Cell Tumors (Testicular Cancer)	Autologous
Paroxysmal Nocturnal Hemoglobinuria	Allogeneic
Hodgkin's Disease	Autologous or Allogeneic
Acute Leukemias	Allogeneic
Chronic Lymphocytic Leukemia	Allogeneic
Familial Erythrophagocytic Lymphohistiocytosis	Allogeneic
Non-Hodgkin Lymphoma	Autologous or Allogeneic
Multiple Myeloma	Autologous or Allogeneic
Osteopetrosis	Allogeneic
Myelodysplastic Syndrome/Other Myelodysplastic Disorders	Allogeneic
Solid Tumors	Autologous
Wiskott-Aldrich Syndrome	Allogeneic

Clinical Trials

A clinical trial is a type of clinical research study that tests a new medical approach in people to make sure it is safe and effective. A clinical trial is sometimes called a research protocol, a clinical study, or simply medical research.

Memorial Sloan-Kettering Cancer Center is typically involved in more than 900 clinical trials at a given time, covering a broad range of subjects. Some clinical trials study a drug, a medical device, or a new way of doing surgery. Others test new ways to prevent disease, diagnose cancer, improve quality of life, or help people with cancer manage difficult psychological and social issues. Some clinical trials are small, with just a few patients. Others are large and involve thousands of patients at Memorial Sloan-Kettering and other centers.

Clinical trials have played an important role in the fight against cancer. They are the only scientific way to prove whether a new treatment works better than current treatments. Most of the approaches that doctors use to treat cancer today would never have become available without clinical trials.

Why Consider a Clinical Trial?

Clinical trials may offer you access to a new experimental treatment before it is available to most patients. Joining a clinical trial is also a way to make a valuable contribution toward the progress against cancer. Many people with cancer are now living longer because of what was learned from past clinical trials.

Although every clinical trial is different, most are open to men, women, and/or children of all ethnic, racial, and socioeconomic backgrounds. Having a diverse group of participants is important to ensure that a new approach is safe and effective for all people, and that the latest discoveries about cancer improve the lives of everyone.

Clinical Trials at Memorial Sloan-Kettering Cancer Center

Memorial Sloan-Kettering is able to offer so many clinical trials because our doctors are experts in treating all kinds of cancers, both common and rare. Memorial Sloan-Kettering is also home to one of the world's top cancer research programs. Our scientists and physicians work together to turn scientific discoveries into new and improved cancer treatments.

The health of every patient who participates in a clinical trial is of the utmost importance at Memorial Sloan-Kettering. If you choose to participate in a clinical trial, the details of the study will be explained to you completely before you begin. A team of doctors, nurses, social workers, and other healthcare professionals will monitor your health closely to determine the safety and effectiveness of the treatment. They will also help you with any side effects you might experience.

Learn More about Clinical Trials

The more you know about clinical trials, the better informed you will be when deciding whether or not to join one. In this section, you will find general information about clinical trials. You will also find a listing of studies at Memorial Sloan-Kettering Cancer Center that are currently enrolling patients.

Adult Brain Tumors

• The brain is a soft mass of tissue that has three major parts, the cerebrum, cerebellum and the brain stem, all of which are effectively surrounded and protected by the bones of the skull; the brain is the tissue that controls people's voluntary and involuntary actions. 
  
• Cancer is the unregulated growth of abnormal cells in the body (cancer cells are also termed malignant cells).
  
• Malignant brain tumors contain cancer cells; benign brain tumors do not contain cancer cells but do contain abnormally replicating cells that do not metastasize (spread to other organs) but may still cause problems, often because of their size and are regulated to grow in a specific area. 
  
• Primary brain tumors are composed of abnormal types of brain cells with unregulated growth; the most common type is termed gliomas that arise from brain glial cells, but there are many other types (for example, astrocytomas, ependymomas, medulloblastomas and others).
  
• Secondary brain tumors are tumors that have spread to the brain tissue, but are composed of cancer cells from other organs (for example, breast, lung).
  
• The exact cause of brain tumors is unknown; however, people at higher risk for them are children and the elderly, white males, people with family members that have brain tumors, radiation exposure, and exposures to many different chemicals.
  
• Symptoms of brain tumors, many of which are non-specific and occur in other diseases, may include headaches, nausea, vomiting, speech, hearing or vision changes, memory problems, personality changes and paresthesias (an abnormal sensation of the skin such as numbness, tingling, prickling, burning, or creeping on the skin that has no objective cause).
  
• Brain tumors are diagnosed preliminarily by many methods including detailed physical exam, CT and/or MRI exams, angiograms, and X-rays; definitive diagnosis is by removing tissue from the tumor (tumor biopsy) and examining the cells microscopically.
  
• Treatment choices for a brain tumor depends on joint decisions made by the patient and the patient's physician team (team members may include oncologists, surgeons, therapists and others the patient may choose, including other doctors who may give a second opinion); treatment methods are based on the individual's disease and may consist of surgery, chemotherapy, radiation therapy, combinations of these methods or no treatment.
  
• Side effects of treatments are common and numerous but vary from patient to patient depending on the disease, method(s) used and the effectiveness of medications and other methods to reduce them; some of the most common side effects are weakness, nausea, edema, skin changes and hair loss but may include more serious problems such as infections, seizures, disabilities such as speech problems, mental changes and occasionally, death.
  
• Rehabilitation is frequently included in the treatment plan; specialists like physical, occupational and speech therapists can help the patient improve.
  
• Follow-up appointments are part of the treatment plan for brain tumors to catch any recurrent disease and to help with rehabilitation treatments.
  
• Support groups are available to patients with brain tumors and to patients who have and are undergoing treatments; for example, the American Cancer Society, American Brain Tumor Society.

What are adult brain tumors?

Adult brain tumors are diseases in which cancer (malignant) cells begin to grow in the tissues of the brain. The brain controls memory and learning, senses (hearing, sight, smell, taste, and touch), and emotion. It also controls other parts of the body, including muscles, organs, and blood vessels. Tumors that start in the brain are called primary brain tumors.

What are metastatic brain tumors?

Often, tumors found in the brain have started somewhere else in the body and spread (metastasized) to the brain. These are called metastatic brain tumors.

Why do cancers come back?

Sometimes cancer can come back. This can happen because tiny cancer cells, which can’t be seen with the naked eye or on scans, can be left behind after cancer treatment.

Over time these cancer cells can begin to divide again and form a tumour.

Treatment may be given to try to get rid of all the cancer so that it doesn’t come back. Many people may have an operation to remove the tumour. Often, to make sure all the cancer cells are taken away during the operation, some healthy tissue around the tumour will also be removed. To help reduce the risk of any cancer cells being left behind after surgery, other treatments such as chemotherapy, radiotherapy andbiological therapy may also be used.

For some types of cancer an operation isn’t an appropriate treatment. In this case chemotherapy, radiotherapy or biological therapy may be used. These treatments aim to destroy as many of the cancer cells as possible. Often a combination of treatments is given, which can be more effective.

Unfortunately, no treatment is guaranteed to be 100% effective. Sometimes cancer cells can remain and in some people the cancer might come back - sometimes many years later.

If cancer comes back in the same area of the body it’s known as a local recurrence. If cancer develops in a different part of the body, it’s called a metastasis or secondary cancer. A secondary cancer can develop if cancer cells break away from the original (primary) cancer and spread to other organs in the body through the bloodstream or lymphatic system. When these cells reach a new area of the body they grow to form a new tumour.

If cancer does come back it can often be treated again. Usually in this situation, treatment is given to control the cancer, but it may be possible to give treatment that aims to get rid of the cancer.

How is cancer treated?

There are several different types of cancer treatment.

Some are used to treat cancer in a particular (local) area of the body. These are called local treatments. They include surgery and radiotherapy. Other treatments can treat cancer in more than one part of the body at a time. These are called systemic treatments. Chemotherapy, hormonal therapy and biological therapy generally work in this way.

The main types of treatment for cancer are described here. It’s quite common for a combination of treatments to be used.

Surgery

An operation to remove the tumour is the main treatment for many types of cancer.

Surgery is most likely to be used if the cancer is only in one area of the body and has not spread. It can sometimes be used to remove a cancer that has spread to another area of the body, but this is less common.

Radiotherapy

Radiotherapy uses high energy x-rays to destroy cancer cells. The radiation is targeted at the area affected by cancer so that it does as little harm as possible to normal cells. One of the most common side effects is tiredness. Other side effects depend on the part of the body being treated.

Chemotherapy

Chemotherapy uses anti-cancer (cytotoxic) drugs to destroy cancer cells.

There are more than 50 different chemotherapy drugs. Sometimes treatment is with just one chemotherapy drug but often two or more drugs are given together. This is known as combination chemotherapy. The type of chemotherapy treatment you are given depends on the type of cancer you have.

Chemotherapy can cause various side effects depending on which drug (or combination of drugs) is used. However, there are many ways of preventing or reducing the side effects of chemotherapy.

Hormonal therapy

Hormones are natural substances made by the body that control the growth and activity of certain cells. Some cancers depend on particular hormones in order to grow. Hormonal therapies reduce the levels of these hormones in the body or block the hormones from reaching cancer cells. This shrinks the cancer and stops it growing.

Biological therapy

Biological therapies work in various ways to destroy cancer cells. They can:

• stimulate the body’s defences (immune system) to attack the cancer
• attach to particular types of cells (including cancer cells) in the body so that they die
• interfere with a cancer’s ability to grow
• stop a tumour from making its own blood supply so it can’t get the oxygen and nutrients it needs. 

Some biological therapies will do just one of these things while others may use two or more of these methods to attack the cancer.

Biological therapies can be given different names according to how they work. Some of the main types of biological therapies used to treat cancer are monoclonal antibodies,cancer growth inhibitors and angiogenesis inhibitors. Research is trying to discover whether other types of biological therapy such as vaccines and gene therapy can be used to treat cancer. This type of research is in the very early stages.

We have more information on cancer treatments and also on clinical trials.