Myelodysplastic syndrome

Research

Many patients at Fred Hutchinson Cancer Center receive promising therapies by taking part in clinical trials. These research studies are done by physician-scientists from both Fred Hutch and UW Medicine. They test new treatments or new ways to use current treatments.

Every advance in cancer treatment in recent years has come out of clinical trials. We offer more active clinical trials than anywhere else, which means more treatment options for patients like you.

Clinical trials

Most people with MDS receive treatment through clinical studies, also called clinical trials, because these offer a way to get the latest promising therapies. Access to clinical studies by researchers at both Fred Hutch and UW Medicine is one reason many patients come to us for care.

Your doctor can identify clinical trials that match your circumstances and talk with you about whether and how to participate. If you are eligible for a trial, we strongly recommend considering this option.

Clinical trial 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.

Medicines and chemotherapies

Agents that showed promise in pilot studies and are being tested in clinical studies may include:

AMG-531
Azacitidine given by mouth
Clofarabine (Clofarex, Clolar) given by mouth
Decitabine given by mouth
Rigosertib (Estybon)
Venetoclax (Venclexta)
Vorinostat (Zolinza)
Different combinations of chemotherapy agents
Different transplant regimens, such as radiolabeled BC8 (anti-CD45), which is an experimental transplant conditioning agent, and the chemotherapy medicine treosulfan

Doctors are also working on studies to:

Determine whether immune cells in a person with MDS can be modified so they will attack and kill the MDS cells (a type of immunotherapy)
Understand the chain of events that leads to severe MDS, with the hope of finding an agent that blocks one or more of these events to at least stabilize the disease

Chemotherapy Treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. It may be given alone or with other treatments. Treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Chemotherapy may be given by mouth, injection, infusion or on the skin, depending on the type and stage of the cancer being treated. It may be given alone or with other treatments, such as surgery, radiation therapy or biologic therapy. Clinical trial 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. Conditioning Treatments to prepare patients for stem cell transplantation. May include chemotherapy, monoclonal antibody therapy and radiation. The treatments used to prepare a patient for stem cell transplantation (a procedure in which a person receives blood stem cells, which make any type of blood cell). A conditioning regimen may include chemotherapy, monoclonal antibody therapy and radiation to the entire body. It helps make room in the patient’s bone marrow for new blood stem cells to grow; prevent the patient's body from rejecting the transplanted cells; and kill any cancer cells that are in the body. Immunotherapy A type of therapy that uses substances to stimulate or suppress the immune system to help the body fight cancer, infection and other diseases. A therapy that uses substances to stimulate or suppress the immune system to help the body fight cancer, infection and other diseases. Some immunotherapies only target certain cells of the immune system. Others affect the immune system in a general way. Types of immunotherapy include cytokines, vaccines, bacillus Calmette-Guerin (BCG) and some monoclonal antibodies.

Targeted radiotherapy

At Fred Hutch, researchers are also using antibodies to deliver radiation specifically to disease-causing cells. This lets doctors get more powerful doses of radiation where it’s needed to destroy the disease, while sparing most healthy tissue. People who receive targeted radiotherapy may be able to have low-dose transplant conditioning, which tends to cause milder side effects. This combination was first used successfully in high-risk MDS patients here who had no other curative options.

Antibody A protein made by immune system cells and released into the blood. Antibodies defend the body against foreign substances, such as bacteria. Conditioning Treatments to prepare patients for stem cell transplantation. May include chemotherapy, monoclonal antibody therapy and radiation. The treatments used to prepare a patient for stem cell transplantation (a procedure in which a person receives blood stem cells, which make any type of blood cell). A conditioning regimen may include chemotherapy, monoclonal antibody therapy and radiation to the entire body. It helps make room in the patient’s bone marrow for new blood stem cells to grow; prevent the patient's body from rejecting the transplanted cells; and kill any cancer cells that are in the body. Radiation therapy The use of high-energy radiation from X-rays, gamma rays, neutrons, protons and other sources to kill cancer cells and shrink tumors. 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 or brachytherapy). Systemic radiation therapy uses a radioactive substance, such as a radiolabeled monoclonal antibody, that travels in the blood to tissues throughout the body. Side effects A problem that occurs when treatment affects healthy tissues or organs. Some side effects of cancer treatment are nausea, vomiting, fatigue, pain, decreased blood cell counts, hair loss and mouth sores.

More research questions

Our researchers are working on a number of other topics related to MDS, such as:

How to identify subtypes of MDS more precisely, come up with more specific treatments and match each patient with the most effective medicines
What the best approach is for people who receive a bone marrow transplant, including chemotherapy intensity and transplant timing
What causes MDS, including whether changes in blood stem cells that occur naturally with age can provide clues and whether environmental factors play a role

Bone marrow The soft, spongy material in the center of your bones that produces all your blood cells, such as white blood cells, red blood cells and platelets. Chemotherapy Treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. It may be given alone or with other treatments. Treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Chemotherapy may be given by mouth, injection, infusion or on the skin, depending on the type and stage of the cancer being treated. It may be given alone or with other treatments, such as surgery, radiation therapy or biologic therapy. Stem cell A cell from which other types of cells develop. For example, blood cells develop from blood-forming stem cells.

Find open clinical trials for MDS at Fred Hutch, and visit our Patient Guide to Clinical Trials to learn about participating.

Patient Guide to Clinical Trials

Clinical Trials for Myelodysplastic Syndrome

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