"Patients can be assured that MDS researchers all know their quality of life is paramount."
— Dr Shoshana Burke, May 2025

 

• Reducing transfusions is a major area of research. In MDS, staying independent of transfusions for as long as possible is key in maintaining a good quality of life. The level of transfusion dependency informs outcome, ie the fewer transfusions, the better their quality of life, and the more improved the prognosis.

 

• Reducing Inflammation. As a dominant pathway in MDS, and in the risk of progression to AML, inflammation is a popular target for research, which includes looking at Metformin (common treatment for diabetes) as a treatment for cancers, including MDS.

 

• Luspatercept and Imetelstat.  Both continue to show impressive results in reducing transfusion dependency, which should help obtain approval by the NHS. (Luspatercept is licensed in the UK but not yet available on the NHS, Imetelstat has just had European approval). Trials in combining ESAs (such as Epothrypoetin/EPO) with these two treatments are underway, and the signs are encouraging so far.

 

• Methylation (modifies gene expression).  This is increased in MDS and therefore a major target for research/treatments. Hypomethylating agents are showing positive results in trials, focusing on lower-risk MDS and potentially widening the scope for transplants.

 

• Vitamin C. Danish studies into the use of Vitamin C in MDS are very encouraging.

 

• More ‘prospective’ trials are being called for, aimed at low-risk and non-transfusion-dependent MDS, and into treatments which target specific gene mutations.

 

• What this means for MDS patients.   This year’s congress brought encouraging news for patients and families affected by MDS. From reducing transfusion reliance to targeting inflammation and gene expression, the field is moving toward more effective and personalised care.  We will be focusing on all these topics over the next few months and will bring you further news on our website and newsletters.