A Need for New Therapies in High-Risk MDS

The development of new treatments for myelodysplastic syndromes (MDS) has been slow, particularly for patients with high-risk disease. Despite the introduction of hypomethylating agents (HMAs) and lenalidomide (Revlimid) nearly 2 decades ago, few new drugs were approved until 2020.

According to Amer Zeidan, MBBS, associate professor at Yale School of Medicine, 2023 saw the approval of luspatercept-aamt (Reblozyl) and oral decitabine. However, most other trials evaluating other drugs for MDS have been unsuccessful. With limited treatment options available, especially for high-risk MDS, there is a pressing need for more effective drugs.

Here, Zeidan explains how the approval of imetelstat may impact patients with MDS.

Transcription:

0:09 | I think it would be a very important development because MDS has been a setting in which we have a lot of therapeutic failures, unfortunately, especially in high-risk disease. If you look at the last almost 20 years since HMAs were approved in 2004 and 2006 with azacitidine and decitabine, and lenalidomide in 2005, we did not get an approval in almost 20 years, except in the year 2020 where we had luspatercept as a second-line approval, and an oral version of decitabine. But we had many, many trials that tried [to look at] different agents and they were unsuccessful.

0:47 | This year, we have had an approval of ivosidenib [Tibsovo], which is an IDH1 inhibitor in the refractory/relapsed MDS setting after HMA failure, but that is a very small subset of patients who have IDH1 mutations. And [then there was] luspatercept with its frontline approval. But outside of this, we have had many failures, and clearly, having drugs being available to our patients is very important. We have seen more success in lower-risk than we have had in higher-risk. Hopefully that is going to extend to high-risk MDS, and we can keep the same momentum going.