In Utero Stem Cell Transplantation
Boost Brittle Bones Before Birth
Phase I/II clinical trial to test the safety and efficacy of pre- and/or postnatal MSC transplantation in the severest forms of OI (type III, severe type IV). Transplantation before birth at the onset of disease should lead to greater efficacy and engraftment with less rejection than transplantation after birth.
Phase I clinical trial to demonstrate the safety, feasibility and efficacy of performing in utero stem cell transplantation on fetuses affected with ATM. Performing stem cell transplantation at the same time as IUT minimizes any additional procedural risk to the fetus.
Fetal Research Intervention for Vein of Galen Malformation
While many vein of Galen patients do very well with conventional treatment after birth, there remains a significant fraction who will develop severe cardiopulmonary symptoms soon after delivery, and this subgroup has a high risk of morbidity and mortality, even with expert care.
An IRB and FDA-approved clinical research trial of fetal brain intervention/embolization for vein of Galen malformations at Boston Children’s Hospital is now open for recruitment.