A recent study conducted by researchers at the Wake Forest Institute for Regenerative Medicine (WFIRM) has revealed the potential of bioengineered human placental cells as a cure for Hemophilia A, the most common inherited bleeding disorder. The study, published in Nature Communications, demonstrates that transplantation of placental stem cells before birth can result in the presence of the missing coagulation factor that is essential for treating Hemophilia A.
Hemophilia A affects over half a million individuals worldwide and leads to spontaneous and life-threatening bleeding. While significant advancements have been made in treating the disorder, the development of antibodies to current treatments and breakthrough bleeds remain significant challenges. The use of prenatal treatment using in-utero transplantation for Hemophilia A aims to provide curative levels of Factor 8, convert severe bleeding disorders to milder forms, and induce immune tolerance, thereby eliminating the risk of inhibitor formation.
The study explores the feasibility and safety of this innovative approach, particularly for individuals with a family history of Hemophilia A, as prenatal diagnosis and intervention are possible in approximately 70% of cases. The researchers hope that their work will lead to the cure of Hemophilia A before birth, allowing for the birth of healthy infants who would otherwise be affected by the disease.
Graca Almeida-Porada, M.D., Ph.D., the primary investigator on the study, highlighted the potential impact of a curative treatment for Hemophilia A, stating that it would be truly life-changing for patients and their families. The burden of the disease is high, and a treatment that can permanently convert a severe, life-threatening bleeding disorder into a milder issue would significantly improve the quality of life for Hemophilia A patients.
The pioneering research involved bioengineering human placental cells to produce Factor 8 coagulation protein, which was then transplanted into a preclinical sheep model at a gestational age equivalent to 16-18 weeks in humans. The results showed a substantial increase in Factor 8 plasma levels, with levels more than 48% higher than non-transplanted controls. Importantly, the transplanted cells successfully engrafted into major organs, and the recipients did not exhibit any immune responses to the transplanted cells or the Factor 8 protein they produced.
Anthony Atala, M.D., director of the Wake Forest Institute for Regenerative Medicine and author on the study, emphasized the feasibility and safety of prenatal treatment for Hemophilia A based on the study’s findings. The successful results provide evidence that prenatal treatment could be a viable option for managing Hemophilia A and improving the lives of patients.
The collaboration of multiple investigators enabled these groundbreaking studies, showcasing the potential of prenatal treatment for Hemophilia A. The researchers hope that their work will pave the way for the development of a cure for the disease before birth, offering a promising solution for individuals with Hemophilia A and their families.
1. Source: Coherent Market Insights, Public sources, Desk research
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