Myelodysplastic syndromes
A group of blood cancers in which the bone marrow makes blood cells that do not grow into healthy ones.1 Too few healthy blood cells can lead to infection, anaemia or easy bleeding.2 Treatment ranges from blood transfusions and medicines to strong chemotherapy with a stem cell transplant, which can cure some people.1 It is rare and more likely after age 60, or after chemotherapy or radiotherapy for another cancer.2
Read more about Myelodysplastic syndromesTrial-stage treatments and research studies you can join
Filters
- Stem Cell Transplant Method Tested in Severe Anemia and Bone Marrow Failure
Tests a modified donor stem cell transplant in people with severe aplastic anemia or MDS.
Baltimore, United StatesAlso BethesdaAge 4 to 80 - Cladribine, idarubicin, cytarabine and venetoclax for acute myeloid leukemia and related blood cancers
Tests chemotherapy with venetoclax in adults with acute myeloid leukemia and related cancers.
Houston, United StatesAge 18 to 65 - Tests pacritinib in people with myelodysplastic syndromes
Tests the capsule pacritinib in people 12 or older with myelodysplastic syndromes.
Bethesda, United StatesAge 12 to 120 - ATRN-119 Plus Decitabine for TP53-Mutated Acute Myeloid Leukemia or Higher-Risk Myelodysplastic Syndrome
Tests ATRN-119 with decitabine in adults with TP53-mutated leukemia or high-risk MDS.
St Louis, United StatesAge 18+ - Arsenic trioxide to help prevent relapse after stem cell transplant for leukemia
Tests arsenic trioxide to lower relapse risk in adults with leukemia or MDS.
Ann Arbor, United StatesAge 21+ - Testing the safety of agenT-797 cells after a stem cell transplant for blood cancers
Tests whether agenT-797 is safe for adults after a donor stem cell transplant.
Madison, United StatesAge 18+ - Testing dexrazoxane to protect the heart during chemotherapy for blood cancers
Tests dexrazoxane to reduce heart side effects of chemotherapy in people with blood cancers.
Houston, United StatesAge 12+ - Ofirnoflast for Adults With Lower-Risk Myelodysplastic Syndromes Who Need Red Blood Cell Transfusions
Tests ofirnoflast in adults with myelodysplastic syndromes who need red blood cell transfusions.
Cleveland, United StatesAge 18+ - Tests whole genome sequencing alongside standard tests in leukemia and MDS
Tests whole genome sequencing with standard tests in adults with AML or MDS.
St Louis, United StatesAge 18+ - Testing New Treatment Combinations for Newly Diagnosed High-Risk Acute Myeloid Leukemia
Compares newer drug combinations with standard chemotherapy in adults with high-risk acute myeloid leukemia.
‘Aiea, United StatesAlso Albany, Albuquerque, Allentown, Anaconda, Ann Arbor, Appleton, Ashland, Augusta, …Age 18 to 59 - Axatilimab With or Without Azacitidine for Advanced Blood Cancers of the Bone Marrow
Tests axatilimab, alone or with azacitidine, in adults with advanced blood and bone marrow cancers.
Columbus, United StatesAlso PortlandAge 18+ - Tests emapalumab with other drugs to prevent graft-versus-host disease after transplant
Tests emapalumab with other drugs in adults with leukemia or myelodysplastic syndrome.
Duarte, United StatesAge 18 to 75 - Tests R289 in adults with lower-risk myelodysplastic syndromes
Tests the drug R289 in adults with lower-risk myelodysplastic syndromes.
Cleveland, United StatesAlso Columbus, Dallas, Durham, Hackensack, Houston, Los Angeles, Miami, New York, …Age 18+ - Tests elritercept in adults with myelodysplastic syndromes needing regular blood transfusions
Tests elritercept against placebo in adults with low-risk myelodysplastic syndromes needing transfusions.
Canton, United StatesAlso Cleveland, Columbia, Denison, Duarte, Glendale, Henderson, Houston, Issaquah, …Age 18+ - Elritercept Compared With Epoetin Alfa for Anemia in Lower Risk Myelodysplastic Syndromes
Compares elritercept with epoetin alfa for anemia in adults with myelodysplastic syndrome needing transfusions.
Atlanta, United StatesAlso Baytown, Boise, Cleveland, Denton, Duarte, Fort Myers, Fort Worth, Gilbert, …Age 18+ - Ropeginterferon alfa-2b for chronic myelomonocytic leukemia and related overlap blood cancers
Tests ropeginterferon alfa-2b in adults with chronic myelomonocytic leukemia or related blood cancers.
Los Angeles, United StatesAge 18+ - Testing enasidenib added to cedazuridine-decitabine for higher-risk IDH2-mutated myelodysplastic syndrome
Tests adding enasidenib to standard drugs in adults with higher-risk IDH2-mutated myelodysplastic syndrome.
Albany, United StatesAlso Albuquerque, Anaconda, Ankeny, Ashland, Baton Rouge, Berkeley, Bethesda, Billings, …Age 18+ - Tests cirtuvivint alone and with ASTX727 in leukemia and myelodysplastic syndromes
Tests cirtuvivint, alone or with ASTX727, in adults with leukemia or myelodysplastic syndromes.
Atlanta, United StatesAlso Baltimore, Basking Ridge, Boston, Chicago, City of Saint Peters, Commack, Creve Coeur, Crown Point, …Age 18+ - Tests natural killer cell therapy after cord blood transplant in blood cancers
Tests natural killer cells after chemotherapy and cord blood transplant in people with leukemia.
Houston, United StatesAge 15 to 80 - Venetoclax with chemotherapy for acute myeloid leukemia
Tests venetoclax with chemotherapy in adults with acute myeloid leukemia or related blood disorder.
Houston, United StatesAge 18+
Where treatment stands
Some patients can be cured with strong chemotherapy and a stem cell transplant from a donor. For others, treatment may slow the disease from getting worse.
Where research stands now
On 11 November 2026, 396 trials and studies of this disease were open. That day, 530 treatments were being tested in people; 46 had reached phase 3, the last large step before approval. From 5 November 2025 to 11 November 2026, articles reported 38 treatment studies in people, 15 in animals and 9 in the lab. In the EU, imetelstat and efbemalenograstim alfa were first approved in 2025 and 2024, not always for this disease. Health pages say strong chemotherapy with a donor stem cell transplant can cure some people.
We found 13 medicines approved for myelodysplastic syndromes in the EU, the US, Switzerland or France, and 11 being tested in people.
Approved: 13
Officially approved for this disease in the EU, the US, Switzerland or France.
Human testing stage: 11
Being tested in people. Many treatments at this stage are never approved.
Animal testing stage: 3
Worked in animals in a study published in the last 12 months. Most treatments at this stage never reach people.
Placebo: a dummy treatment without the medicine, used for comparison.
Phase 1, 2, 3: the steps of testing in people. Phase 1 checks safety, phase 3 is the last step before approval.
The EU medicines agency (EMA) and its list of medicines in the EU, US prescribing labels (DailyMed, openFDA), the Swiss medicines register (Swissmedic), the French medicines database (ANSM) and the Health Canada drug database, checked on 11 November 2026. Every medicine links to its own official text.
A computer made this list from the official texts: it kept only medicines whose own approval text names the disease, wrote "Who it is for" from that text and checked it against the text a second time. People have not reviewed it yet.
The trials: ClinicalTrials.gov and the EU trials register, as copied into our trial database, checked on 12 November 2026. Every treatment in trials links to its own trial records; the texts are written by AI from them and checked against them by a second AI. People have not reviewed them yet. Placebo, standard medicines used for comparison and procedures are left out.
Phase 3 trials are listed from every country; only some phase 2 trials are listed, so 128 other open phase 2 trials are not listed.
Animal and lab research: studies listed in PubMed and published in the 12 months before 12 November 2026, found by a search for myelodysplastic syndromes. AI chose the treatments from the study summaries and wrote the texts; a second AI checked each one against its study summary. People have not reviewed them yet. Treatments already in the approved or human testing lists are left out. A result in animals or cells does not show that a treatment works in people.
Not medical advice. Talk to your doctor before you change any treatment.
Support organisations
Patient organisations and helplines in United States, for you and your family.
Myelodysplastic syndromes organisations in United States
- National Cancer Institute – Cancer Information Service (CIS)
NCI's contact center: information specialists answer cancer questions from patients, families and the public by phone, chat and email.
- American Cancer Society
Nationwide US nonprofit helping people who face cancer: research, patient services, early detection, treatment information and education, with a cancer helpline on its site.
- Aplastic Anemia and MDS International Foundation
Foundation supporting patients and families with aplastic anemia, MDS and related bone marrow failure diseases.
- Cactus Cancer Society
Young adult cancer resource and community: support programs for people diagnosed with cancer as young adults.
- Cancer Hope Network
Nonprofit providing free one-on-one support to cancer patients and families by matching them with trained volunteers who have had a similar experience.
- Cancer Nation
Survivor-led US cancer advocacy organization (formerly the National Coalition for Cancer Survivorship) working on quality cancer care and survivorship resources.
- Genetic and Rare Diseases Information Center
NIH information center (GARD) providing reliable information on genetic and rare diseases to patients and families via website and contact center.
- Imerman Angels
Nonprofit offering free one-on-one cancer support and personalized mentoring that connects cancer fighters, survivors and caregivers with someone who has been there.
- MDS Foundation
US-based global nonprofit supporting patients with MDS and related diseases such as AML, their families and providers, with education, resources and support.
- Triage Cancer
National nonprofit providing free education on legal and practical issues (insurance, employment, finances) for people with cancer and caregivers.
General support in United States
- 988 Suicide & Crisis Lifeline
US national 24/7 free and confidential lifeline for anyone in suicidal crisis or emotional distress, reachable by calling or texting 988 or by chat.
- MedlinePlus
Free, easy-to-read health information service of the US National Library of Medicine (NIH) on diseases, conditions, drugs and wellness.
International organisations
- DKMS
Finds blood stem cell donors worldwide so that people with blood cancer can have a transplant.
Offer tissue or data
There are three ways to help research move faster.
Treatments approved outside the EU
We found no medicine for Myelodysplastic syndromes approved in the US or Japan that is not approved in the EU. These are the ones approved in the EU.
Checked on 11 Nov 2026 in the official lists of the US (FDA), Japan (PMDA, approvals since April 2004) and the EU (EMA). 6 uncertain matches are left out.
Your health insurer may pay part of the cost, sometimes all of it. This depends on your insurer, your plan and the country, so always ask your insurer before you go. For planned care in the EU, the EEA and Switzerland, ask about form S2.
“Miracle cure” fact checker
Social media spreads misinformation. Paste what you read here, and we'll tell you whether there is scientific evidence behind it.
What you paste is checked with AI (Anthropic, USA) and is not stored. Please leave out names and other personal details.
Before you pay for anything, ask three things
- Is there a published study in people?
- Can I have the full price in writing?
- What does my doctor say?
“Cures” others already fact-checked
- CBD (cannabidiol) & Myelodysplastic syndromes
- Vitamin C (incl. high-dose) & Myelodysplastic syndromes
- Ivermectin & Myelodysplastic syndromes
- Fenbendazole & Myelodysplastic syndromes
About Myelodysplastic syndromes
A group of blood cancers in which the bone marrow makes blood cells that do not grow into healthy ones.1 Too few healthy blood cells can lead to infection, anaemia or easy bleeding.2 Treatment ranges from blood transfusions and medicines to strong chemotherapy with a stem cell transplant, which can cure some people.1 It is rare and more likely after age 60, or after chemotherapy or radiotherapy for another cancer.2
What is it?
Myelodysplastic syndromes (MDS) are a group of blood cancers.1 Normally, stem cells in the bone marrow grow into healthy red cells, white cells and platelets.1,2 In MDS many of them do not mature and die in the marrow.2 This leaves too few healthy blood cells, which can lead to anaemia (too few red cells), infections and bleeding.2,1
MDS is rare and mostly affects older people: half are about 70 or older when they find out.2,3 In most people the cause is not known.1 The risk is higher after chemotherapy or radiotherapy for another cancer, and after contact with tobacco smoke, some chemicals (such as benzene, pesticides or fertilisers) or heavy metals such as lead and mercury.1
How doctors find out
Blood tests count the red blood cells, white blood cells and platelets.1 A bone marrow test takes a small sample of marrow with a needle, and a lab checks it for abnormal cells and chromosome changes.1 Some of these tests, such as checking the chromosomes, help plan treatment.1
Current treatment
Treatment depends on several factors. It can include transfusions, medicines that help make red blood cells, and antibiotics for symptoms; lenalidomide or azacitidine and decitabine for some people; and chemotherapy with a donor stem cell transplant for certain patients.Source: NCI PDQ
Will it get worse?
Prognosis (chance of recovery) and treatment options depend on certain factors.1 How it goes depends on how many immature cells (blasts) are in the marrow, how many kinds of blood cells are low, certain chromosome changes, whether it followed earlier cancer treatment, and your age and general health.1 Some types of MDS can turn into acute myeloid leukaemia (AML).1
Medicines: what helps, and what to watch for
Supportive care eases the problems caused by the disease. It can include red blood cell or platelet transfusions, medicines that help the body make more red blood cells, and antibiotics to fight infection.1 Azacitidine or decitabine can slow the change to AML.1 Lenalidomide helps some people with a certain chromosome change (del(5q)) need fewer transfusions.1 People at high risk of AML may get the same chemotherapy as for AML.1
Good to know:
A transplant, for some people
Some people can be cured with strong chemotherapy followed by a stem cell transplant from a donor, which gives them healthy blood-forming cells.1 It may not work as well when MDS was caused by earlier cancer treatment.1
Is there a cure?
For some people, yes: strong chemotherapy with a donor stem cell transplant can cure certain patients.1 There is no standard treatment when MDS comes back or stops responding.1 Clinical trials are testing new treatments.1
Median survival
About 2 years with azacitidine, in higher-risk MDSSource: The Lancet Oncology, 2009
Measured in: Higher-risk myelodysplastic syndromes: a phase III trial of azacitidine against conventional care (358 patients)
The figures behind it
Phase III trial in higher-risk myelodysplastic syndromes (358 patients): median overall survival 24.5 months with azacitidine vs 15.0 months with conventional care regimens. Lower-risk MDS is not part of this figure.
Sources
Written in plain words by Frontlight from these pages: