Tippi MacKenzie, MD, is stopping disease before it starts – in the womb.
In 2017, she launched the world's first clinical trial of an in-utero stem cell transplant therapy. Her first patient, baby Elianna, was born just four months later.
Elianna's birth came after a second-trimester diagnosis of a normally fatal blood disorder. Her story hints at the lifesaving possibilities of fetal therapy for families who face similarly devastating diagnoses.
Refer to Fetal Treatment Center
this is Eliana and she is my patient without our help. She will most likely die before she is born. I came to U. C. S. F. Because fetal surgery was born right here and I love being able to look at parents in the eye and say we know what's going on with your child. We think we know how to fix it. There are also a lot of patients that we don't quite know how to help. What we're trying to do now is to bring fetal surgery to the next frontier. If this works we would be able to treat fetuses with a whole range of blood disorders before birth. This is the world's first clinical trial of a fetal stem cell therapy. Mhm. Mhm. One of the main things we've discovered is that there are cells trafficking between the mother and the fetus. If you transplant cells from the mother to the fetus will tolerate them and the transplant should be more successful. Elena's parents were the most lovely caring generous couple we could have had for our first patient. And they asked me Dr Mackenzie have you done this before? And I said no you would be the first. I can't think of a situation where you have more of a sense of responsibility. The day of the surgery was an incredibly surreal day where I tried very hard not to think of the fact that this day would be the culmination of more than a decade of research. The first step in this process is to harvest the bone marrow stem cells from the mother. Then the stem cells are taken to the bone marrow processing lab where they're isolated and purified. Mhm. We then inject the mothers healthy stem cells into the umbilical vein of the fetus along with a blood transfusion. Then we wait to see if the transplant works. Eliana is the first patient in our clinical trial to receive stem cells prior to birth. It was an amazing feeling to see Eliana wiggling around the incubator bed with her full head of black hair. This trial is the first step on a long journey. One thing that keeps me going is that we're so close, we're right there were almost able to treat fetuses and families with a whole host of birth defects. We're going to continue this work until birth defects are a thing of the past. Uh Oh yeah, yeah.
