Stem Cell Injections for Fetuses with "Brittle Bone" Disease

Many babies have a disease called osteogenesis imperfecta, that causes their bones and skeletons to be very brittle.  Their bones are so fragile that they fracture while in the womb.  Scientists and doctors are now performing stem cell therapy on fetuses in the womb to strengthen the skeletons of these babies before they are born.  Osteogenesis imperfecta is caused by a mutation in the gene that creates collagen--which is a tough, flexible, material that is crucial for bone strength.

Scientists are beginning a trial in Stockholm, Sweden that injects cells containing unmutated collagen copies of genes into 20 week old fetuses that were diagnosed with the brittle-bone disease.  The stem cells being injected into these fetuses come from the livers of terminated fetuses.  The way these stem cells help the fetuses in the womb is that they divide and move into the bone, strengthening and fixing parts of the bone that are fractured.  Stem cells will be more effective being transported into fetuses in their mother's wombs than after they are born because while in the womb, the fetus' immune system has not developed yet; so they are less likely to be rejected.  In born babies, the stem cells have a greater chance of being rejected since their immune systems are developed. A larger trial is being conducted in January with 15 fetuses and 15 babies with the condition.  Comparing the number of fractures in each group after the injection will be able to provide information as to whether early injection is more beneficial or not.

Stem cells are a newer concept in the field of medicine, especially being used in gene therapy for fetuses that lack the gene containing collagen.  They are unspecialized cells and have the ability to become specialized cells, as they do in fetuses with osteogenesis imperfecta.  So, they are essentially cells that "mold" into what the body needs them to be.  I believe this stem cell therapy has a good chance of being accepted by the fetuses, in comparison to babies already born due to the immune system development factor.  Stem cells are now being used in gene therapy, which is an important advancement to medicine and health.  If this trial has positive results in the fetuses after they are born, stem cells will most likely be researched further to use in similar diseases caused from gene mutations in fetuses.