Megaloblastic anemia is a type of anemia distinguished by the existence of abnormally large and malformed red blood cells known as megaloblasts. To meet the challenges of therapy, Protheragen allocated its resources into advanced technologies and specialists working toward resolving the problem of megaloblastic anemia. With our complete support services, we can optimize your procedures from the development of the drug candidate to the commercialization of the drug.
Megaloblastic anemia is a type of macrocytic anemia marked by impaired DNA synthesis in the hematopoietic precursors, leading to ineffective erythropoiesis and the formation of megaloblasts-abnormally large erythroid cells with immature nuclei. This condition mostly results from a deficiency of vitamin B12 or folate, although some rare forms exist due to genetic factors or drugs.
Fig.1 Cold agglutinin disease (CAD) is an acquired autoimmune hemolytic anemia (AIHA). (Climent F, et al., 2022)
Megaloblastic anemia stems from the impaired synthesis of DNA resulting from a deficiency of either vitamin B 12 or folate, which interferes with the generation of thymidylate and leads to uracil being incorrectly inserted into the DNA, which triggers the apoptosis of erythroblasts along with asynchrony of the nucleus and cytoplasm in the developing erythrocytes. This leads to ineffective forming of red blood cells alongside the megaloblastic marrow morphology and peripheral blood findings of macrocytic anemia and hypersegmented neutrophils.
Fig.1 Cold agglutinin disease (CAD) is an acquired autoimmune hemolytic anemia (AIHA). (Climent F, et al., 2022)
The present management of megaloblastic anemia is predicated on B12 (injectable or high-dose oral) and folate supplementation, which reliably rectifies the hematologic abnormalities but does not restore neurological damage due to advanced B12 deficiency. This further emphasizes important unaddressed needs.
| Therapy | Targets | Mechanism of Action | Phase |
|---|---|---|---|
| Vitamin B12 | Methionine synthase, Methylmalonyl-CoA mutase | Serves as a cofactor for methionine synthase and methylmalonyl-CoA mutase. | Approved |
| Folate | Dihydrofolate reductase, Thymidylate synthase | Provides tetrahydrofolate (THF) for thymidylate synthase-mediated DNA synthesis, preventing uracil misincorporation. | Approved |
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.
Recognizing the challenges of diagnosing and treating megaloblastic anemia, Protheragen is committed to building a team of experts to provide cutting-edge diagnostic and therapeutic development solutions. Our commitment lies in providing a variety of customized therapy development services to meet the diverse research needs of our customers. We also excel in generating precise disease models that are carefully engineered to replicate the unique features of megaloblastic anemia. These models are valuable tools for validating the safety and efficacy of potential therapeutics.
Animal Model Development Services
At Protheragen, we are dedicated to supporting the development of innovative therapies through comprehensive preclinical research services . Our expertise spans pharmacodynamics (PD), pharmacokinetic (PK) and toxicology studies, ensuring a thorough evaluation of your therapeutic candidates. 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)