Pure Red Cell Aplasia
Pure Red Cell Aplasia (PRCA) is a rare hematological disorder characterized by a selective decrease in the production of red blood cells (RBCs) in the bone marrow. Through our comprehensive diagnostic development and therapeutic development services, we work to pave the way for pharmaceutical companies to develop their therapies.
Introduction to Pure Red Cell Aplasia
Pure Red Cell Aplasia is a hematological disorder characterized by a reduction or absence of erythroid precursors in the bone marrow, leading to a decrease in the production of mature RBCs. The condition can manifest as an isolated defect in erythropoiesis or as a part of a broader systemic disorder. PRCA can occur at any age, but it is more commonly diagnosed in adults. In acquired PRCA, autoimmunity plays a significant role, where the immune system targets and destroys erythroid progenitor cells in the bone marrow.
In some cases, PRCA can be associated with viral infections, notably B19 parvovirus. The virus infects and destroys erythroid progenitor cells, leading to a transient or persistent form of PRCA. Other viral infections, such as hepatitis C and HIV, have also been implicated in the development of PRCA.
Fig.1 Common pure red cell aplasia (PRCA)-associated disorders. (Gurnari C., et al., 2021)
Drug Discovery and Development for Pure Red Cell Aplasia
Immunosuppressive agents such as cyclosporine A have shown promising results in the management of PRCA. Cyclosporine A targets T-cell activation and has been found to induce remission in a significant number of patients. Other immunosuppressive agents, including azathioprine, cyclophosphamide, tacrolimus, rituximab, and anti-thymocyte globulin, may be considered in refractory cases or in PRCA associated with specific conditions.
For PRCA associated with viral infections, targeted antiviral therapy may be beneficial. In B19 parvovirus-associated PRCA, intravenous immunoglobulin (IVIG) has shown effectiveness in suppressing viral replication and improving erythropoiesis. IVIG can also be considered in cases where PRCA is refractory to other therapeutics or associated with immune dysregulation.
Our company is dedicated to advancing therapeutic options for PRCA. We are actively involved in the development of novel drugs and innovative therapeutic approaches. If you would like to customize your own therapy development solution, please click on the link below to learn more.
Our Services
At our company, we are at the forefront of diagnostics and therapy development for pure red cell aplasia. Our comprehensive services encompass a wide range of approaches to better understand and treat this challenging condition. We specialize in the development of animal models that accurately replicate the key features of PRCA. In addition, our dedicated research teams conduct in vitro experiments using patient-derived samples, cell culture models, and advanced molecular techniques to investigate the underlying mechanisms of PRCA.
PRCA Mouse Models
One of our well-established mouse models involves the disruption of erythropoietin (EPO) signaling, a key regulator of red blood cell production. By selectively targeting EPO-related genes we can induce PRCA-like symptoms in mice.
FeLV Infection Cat Models
By infecting cats with FeLV and monitoring their hematopoietic system, we can investigate how FeLV-C impairs erythroid progenitor cells' maintenance and differentiation.
Our company offers a comprehensive cell-based models development service. Through the isolation and culture of primary bone marrow cells from both healthy individuals and PRCA patients, our models enable the study of erythropoiesis and the identification of potential abnormalities in red blood cell production.
By differentiating pluripotent stem cells into erythroid lineage-specific organoids, we can recapitulate the process of red blood cell development in a controlled in vitro environment. These organoids exhibit similar characteristics to native bone marrow, allowing for the study of erythropoiesis and the identification of aberrations that contribute to PRCA.
Furthermore, our preclinical research services include evaluating the efficacy and safety of potential drug candidates for PRCA. In addition to the aforementioned services and models, we also provide customized solutions and develop disease models that cater specifically to your unique needs. If our services have piqued your interest, please do not hesitate to contact us.
Reference
- Gurnari C., et al. "How I manage acquired pure red cell aplasia in adults." Blood, The Journal of the American Society of Hematology 137.15 (2021): 2001-2009.