Prenatal stem cells treat hemophilia A in preclinical study

The study explores the feasibility and safety of prenatal treatment using in-utero transplantation for hemophilia A. Notably, approximately 70% of individuals with hemophilia A have a family history of the disorder, which provides an opportunity for prenatal diagnosis and intervention. This innovative approach aims to provide curative Factor 8 levels, convert severe bleeding disorders to milder forms, and induce immune tolerance, thereby eliminating the risk of inhibitor formation.

“The overall burden of disease in persons with hemophilia continues to be high. A therapeutic option that is curative, or can permanently convert a severe, life-threatening bleeding disorder to a mild issue, would be truly life-changing for Hemophilia A patients and their families,” explained Graca Almeida-Porada, M.D., Ph.D., primary investigator on the study.

“We hope our work will pave the way for the cure of hemophilia A prior to birth, thus enabling the birth of healthy infants who would otherwise have been affected by this disease.”

“These studies, that were possible due to the productive collaboration of multiple investigators, show that prenatal treatment of hemophilia A is feasible and safe,” said Anthony Atala, M.D., director of the Wake Forest Institute for Regenerative Medicine and author on the study.

In this pioneering research, human placental cells were bioengineered to produce Factor 8 coagulation protein and were then transplanted into a preclinical sheep model at a gestational age equivalent to 16–18 weeks in humans.

The results have shown a substantial elevation in Factor 8 plasma levels (>48% above non-transplanted controls) that persisted for the entire 3 years of follow-up. Most importantly, the cells successfully engrafted into major organs, and none of the recipients exhibited any immune responses to either the transplanted cells or the Factor 8 protein they produced.