About 15,000 to 20,000 people in the US are diagnosed with myelodysplastic syndromes (MDS) each year, mostly people over 70. In MDS the bone marrow makes too few healthy blood cells, causing anemia and often the need for transfusions, and some cases turn into acute leukemia. Lower-risk MDS is now treated with newer drugs that reduce transfusions; higher-risk MDS is treated with azacitidine, and a stem-cell transplant remains the only cure.
- 486studies open or running
- 33phase 3 trials
- 1,164papers published this year
- 40NIH-funded projects
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Now
Nov 2026
A better first anemia drugHappened 60% transfusion-free 12+ wks
Luspatercept freed 60% from transfusions with a hemoglobin rise vs 35% with epoetin alfa.
60%: transfusion-free 12+ weeks with a hemoglobin rise, vs 35% with epoetin alfa.
A telomerase blocker approvedHappened 40% transfusion-free 8+ wks
Imetelstat kept 40% transfusion-free for at least 8 weeks vs 15% on placebo.
40%: transfusion-free for 8+ weeks, vs 15% with placebo.
Safer transplantsHappened 3.4% non-relapse deaths
Orca-T cut deaths not caused by relapse to 3.4% vs 13.2%; FDA approved June 2026.
3.4%: deaths not caused by relapse, vs 13.2% with standard transplant.
Venetoclax missed its survival goalHappened 22.2 mo median survival
In higher-risk MDS, adding venetoclax to azacitidine raised responses but median survival was 22.2 vs 21.7 months.
22.2 mo: median survival with venetoclax, vs 21.7 months with placebo.
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6 months
by May 2027
More subgroup data on venetoclaxPossible
Analyses of VERONA will clarify whether younger people or those heading to transplant benefit.
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12 months
by Nov 2027
An all-oral azacitidinePossible
Taiho's ASTX030 (azacitidine plus cedazuridine as a pill) is being compared with injected azacitidine; due November 2027.
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3 years
by late 2029
Treating anemia earlierPossible 60% transfusion-free (COMMANDS)
ELEMENT-MDS (402 people) tests luspatercept against epoetin alfa before transfusions are needed; due March 2028.
60%: transfusion-free 12+ weeks with a hemoglobin rise, vs 35% with epoetin alfa.
Elritercept phase 3 trialsPossible 41.3% transfusion-free (phase 2)
Takeda's elritercept is in two phase 3 trials, against placebo (due September 2028) and against epoetin alfa (due April 2029).
41.3%: transfusion-free for 8+ weeks in the first 24 weeks.
Oral azacitidine for lower-risk MDSLong shot
A Bristol Myers Squibb trial (230 people) tests oral azacitidine against placebo; due July 2028.
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5 years
by late 2031
Another BCL-2 pill for higher-risk MDSLong shot
Ascentage's lisaftoclax plus azacitidine (490 people) is in phase 3; due June 2029.
Treatment matched to gene changesLong shot
The National Cancer Institute's myeloMATCH screens up to 2,000 people and assigns treatment by their leukemia's gene changes; due May 2029.
The research paths
Anemia drugs for lower-risk MDS
60% transfusion-free 12+ wksDetails and 4 trialsHide
Luspatercept, imetelstat and now elritercept aim to end transfusion dependence.
WatchElritercept phase 3; ELEMENT-MDS.
- Elritercept vs epoetin alfa (ELRiSE MDS)Takeda · 300 people · enrolling Apr 2029
- Elritercept vs placeboTakeda · 225 people · enrolling Sep 2028
- Luspatercept vs epoetin alfa, before transfusions are needed (ELEMENT-MDS)Bristol Myers Squibb · 402 people · fully enrolled Mar 2028
- Oral azacitidine, lower riskBristol Myers Squibb · 230 people · fully enrolled Jul 2028
Higher-risk MDS combinations
22.2 mo median survivalDetails and 3 trialsHide
Azacitidine combinations have repeatedly failed to extend survival; BCL-2 inhibitors are still being tested.
WatchLisaftoclax phase 3; gene-matched trials.
Safer stem-cell transplants
3.4% non-relapse deathsDetailsHide
Engineered donor cells and better conditioning to reduce graft-versus-host disease.
WatchUse of Orca-T in older patients.
Borrowed from other cancers
From AML and CLL
Venetoclax
Phase 3 (missed goal)
A BCL-2 inhibitor that works well in AML, tested in higher-risk MDS.
From Myelofibrosis
Momelotinib
Phase 2
A JAK inhibitor that also improves anemia, being tested in lower-risk MDS.
Open to join now
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Lower risk, anemia
Elritercept vs epoetin alfa
Locations and eligibility → -
Lower risk, transfusion-dependent
Elritercept vs placebo
Locations and eligibility → -
Higher risk, newly diagnosed
Lisaftoclax plus azacitidine
Locations and eligibility → -
MDS or AML
myeloMATCH
Locations and eligibility → -
Lower risk, anemia
Momelotinib
Locations and eligibility →
See every recruiting MDS trial →
When the trials finish
Completion dates the trials registered. Results usually follow 3 to 12 months later.
- Heating up
- Maturing
- Early
- Cooling
- Fully enrolled, waiting on results
- Still enrolling
What just happened
FDA approves Orca-T for transplant
Precision-T, Blood.
VERONA: venetoclax misses survival goal
Higher-risk MDS.
FDA approves imetelstat
IMerge, The Lancet.
Standard treatment today
- Lower risk, anemiaLuspatercept or epoetin; imetelstat or lenalidomide (del 5q) when those stop working.
- Lower risk, no symptomsWatch with regular blood counts; early treatment is being tested.
- Higher riskAzacitidine or decitabine (including oral decitabine-cedazuridine).
- Higher risk, fit enoughAllogeneic stem-cell transplant, the only cure.
How to read this page, and sourcesHow to read this page, and sources
- Lower-risk and higher-risk MDS are treated very differently; check which group a result applies to.
- Forecasts are editorial judgments by Find a Trial, not predictions from sponsors or regulators.
See MDS trials recruiting now →