This Drug May Reduce Your Dependence on Transfusions

Celgene Corporation, partnered with Acceleron Pharma Inc., announced at the beginning of this month that target enrollment of myelodysplastic syndromes (MDS) and beta thalassemia patients for the MEDALIST and BELIEVE Phase 3 clinical trials, respectively, for luspatercept has been completed – ahead of schedule.

This rapid enrollment proves that there are huge unmet needs in the rare blood diseases community.

Currently, patients suffering from MDS and beta thalassemia are extremely dependent on red blood cell transfusions to treat underlying anemia in these conditions, and there are limited treatment options outside of these transfusions to treat the complications that arise.

On the other hand, luspatercept is a protein that works to regulate red blood cell differentiation and maturation by promoting the production of healthy red blood cells (in layman’s terms).
Source: Giphy

The president of Hematology and Oncology for Celgene, Michael Pehl believes that

“Luspatercept may be a potentially paradigm-changing treatment option for patients and physicians alike.”

The final results from the Phase 3 studies are suspected to be announced in about a year, with the hope that patients in both trials achieve an overall reduction in red blood cell transfusion burden.

Hopefully, the MEDALIST and BELIEVE studies will be able to formally prove the efficacy and safety of luspatercept and help lessen the burden of MDS and beta thalassemia standards-of-care.

In the meantime, if you want to learn more about luspatercept, click here!

To learn more about MDS, visit our partner at

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