First Clinical Trial of In Utero Stem Cell Transplant for Alpha Thalassemia
We have an opportunity to cure a disease before a person’s born. In our Fetal Treatment Center, we see many fetuses withbirth defects all the time. Some of those birth defects can actually be treated with a stem cell transplant. If you do the stem cell transplant after birth, you have to give the baby a lotof different toxic medicines to make room in the bone marrow. And, that can cause diseases on its own. This is the world’s first clinical trial of a blood stem cell transplantation in the fetus. If this treatment is successful, it could save the parents and the child from a lifetime of illness. (cello notes playing)
So in this first clinical trial, we’re focusing on a diseasecalled Alpha Thalassemia Major. And we focused on thatdisease because it is fatal. Alpha Thalassemia Majoris a disease in which the red blood cells don’t form correctly. And so, the fetus gets very sick. When we take blood stemcells from the mother and we put them into the fetus, the hope is that theywill go to the bone marrow and give rise to good red blood cells that can then carry oxygenand treat the anemia that the fetus is experiencing. (electronic notes popping) First, we have to harvest the stem cells from the mother. And then those cells are taken to the bone marrow stem cell processing lab where the blood stem cells are isolated and purified.
And then, they’re injected back into the umbilical vein. People used to think that the baby was enclosed in this impermeable space. But the truth is that the cells go back and forth between the mother and the fetus. And that trafficking it turns out, is actually really important in allowing the fetus’ immune system to tolerate the mother’s cells. (upbeat happy music) This is the first case in which we’re using the mother’s cells based onthe biology that we discovered and using a high doseof cells placed directly into the fetus’ blood stream. And we think that doing all three of those things will allow us to have more success compared to when it was tried before. If we find that this is safe and effective for this one disease, we hope that we can apply it to a host of other blood diseases, the most common of which would be Sickle Cell Disease.
One of the things about being a doctor is that you’re constantly seeing patients where you know that withthe developments in science, you could help them better. You could help them more effectively. But those therapies aren’tcurrently in the clinics. So I love being able to, in some weeks of the month, go into my research laband test out therapies that we can finally bring to patients. (electronic beeps)