Myelodysplastic syndrome (MDS) can easily develop into acute myeloid leukemia (AML) if not treated in time. Protheragen boasts a talented team of researchers and scientists with extensive expertise in MDS. They are deeply committed to pioneering the development of cutting-edge therapies for MDS, aiming to address unmet therapeutic needs and advance targeted therapeutics in the field.
Myelodysplastic syndrome (MDS) refers to complex and rare disorders of blood which result from the bone marrow's failure to produce healthy, mature blood cells. Blood-forming cells in the bone marrow may lose functional competency, causing reduced levels of red and/or white blood cells and platelets. Therefore, individuals with MDS usually suffer from tiredness, recurrent infections, excessive and painless bleeding, and low levels of hemoglobin.
Fig. 1 Myelodysplastic syndrome originates from a clonal hematopoietic stem cell. (Fontenay, Michaela, et al., 2021)
The development of myelodysplastic syndrome (MDS) is caused by the build-up of genetic alterations in hematopoietic stem cells, which results in a failure of blood cells to adequately differentiate and mature. These changes lead to the disruption of the normal functioning of the bone marrow and also result in ineffective hematopoiesis, cytopenias as well as the production of blood cells which are both abnormal and dysfunctional. Significant underlying mechanisms include alterations to chromosomes, changes to the epigenome and immune system, all which facilitate the clonal expansion of defective cells.
Fig. 2 The multifaceted pathogenesis of myelodysplastic syndromes (MDS). (Lynch, Olivia F., and Laura M. Calvi., 2022)
The global myelodysplastic syndrome (MDS) treatment market is growing steadily. According to statistics, the global MDS market is estimated to be US$2.2 billion in 2023 and is expected to reach US$4.1 billion by 2030, with a compound annual growth rate of 9.1% from 2023 to 2030. This growth underscores the urgent need for innovative therapies, as current therapeutic options remain limited, especially for high-risk MDS individuals.
Table. 1 Drug development pipeline for myelodysplastic syndrome (MDS).
| Drugs | Types | Targeted | Developmental Stage |
|---|---|---|---|
| Luspatercept | Erythroid maturation agent | TGF-β superfamily signaling | Approved |
| Venetoclax | BCL-2 inhibitor | BCL-2 protein | Approved |
| Magrolimab | Anti-CD47 monoclonal antibody | CD47 | Phase III |
| Pevonedistat | NEDD8-activating enzyme inhibitor | NEDD8 pathway | Phase III |
| Ivosidenib | IDH1 inhibitor | IDH1 mutation | Approved |
| Enasidenib | IDH2 inhibitor | IDH2 mutation | Approved |
| Sabatolimab | Anti-TIM-3 monoclonal antibody | TIM-3 immune checkpoint | Phase III |
Disclaimer: Protheragen focuses on providing preclinical research services. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.
To tackle the unmet challenges in managing myelodysplastic syndrome (MDS), Protheragen offers end-to-end diagnostic and therapeutic development services. By focusing on the complex molecular mechanisms underlying MDS, we are dedicated to pioneering innovative therapies that deliver effective therapeutic solutions. Our team specializes in developing highly precise disease models, which facilitate thorough assessment of the safety, efficacy, and mechanisms of action of potential therapeutic candidates.
At Protheragen, we are committed to validating and optimizing therapies for myelodysplastic syndrome (MDS) through preclinical studies including pharmacodynamics, pharmacokinetics and toxicology to ensure their successful regulatory approval. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
References
Myeloproliferative Disorders (MPDs)
Bone Marrow Failure Syndromes (BMFS)