To Believe: The Story of Fetal Therapy

To Believe

Alan M. Flake, MD: For most of human history the fetus has been really shrouded in mystery and multiple layers of the maternal abdomen obscuring anything that was wrong with the fetus.

N. Scott Adzick, MD: With the emergence of prenatal diagnostic techniques, particularly with the advent of maternal-fetal ultrasound, we could view into the womb for the first time.

Holly L. Hedrick, MD: We could see things we'd never seen before and, as those fetuses were followed, it became more clear what may or may not happen to them.

N. Scott Adzick, MD: There were a certain category of birth defects that were, for the most part, lethal. Where, when the baby was born, we were too late to do anything about it.

Holly L. Hedrick, MD: That was really the impetus to try to knock on the door sooner.

N. Scott Adzick, MD: Perhaps we can treat this new group of highly-selected unborn patients who have severe diseases that are progressive during the pregnancy. Maybe we could treat them before birth and that was a very radical and controversial concept.

Mary Kelly: At 20 weeks I had the Level II ultrasound, and everything was fine. About five weeks later, I went for a regular checkup with my OB, and he said, "You know, you're measuring a couple weeks ahead of schedule. Let's just get an ultrasound and just make sure everything's going okay." And that's when we found out that I was carrying a very, very sick baby.

Lori J. Howell, RN: I think about fetal surgery work being presented at national, international meetings and essentially being laughed out of the room.

N. Scott Adzick, MD: Most people thought we were crazy because there had to be at least 100 reasons why you couldn't operate on a fetus.

Alan M. Flake, MD: There was also a lot of skepticism about whether or not this was really beneficial.

N. Scott Adzick, MD: That's frequently the case with something that's new, and it was our obligation to not only convince the world, convince ourselves, but all that work had to be done prior to starting it clinically.

Lori J. Howell, RN: We had to know that, if we offered anything before birth, it had to be safe for mom.

N. Scott Adzick, MD: We had to go into the experimental laboratory and work principally with fetal sheep to develop the animal models and the techniques that we hoped some day could be used clinically.

Alan M. Flake, MD: We showed that those anatomic abnormalities cause the same sort of organ damage that was seen the human fetuses, and we showed in the animal model again that it could be corrected before birth.

N. Scott Adzick, MD: Then we had to apply this cautiously in the clinical realm, isolated cases, being brutally honest with the families about what we could and could not do, what our hopes were, and what our fears were.

Lori J. Howell, RN: And people began to believe-- Oh, in certain select instances it's not crazy to operate on the baby before they're born.

Mary Kelly: When they told us what fetal surgery meant, it was very surreal. It was something out of a sci-fi movie.

Jed Kelly: We sat down with the doctors, and they gave us their conclusions on what they believed Addison's condition was that she had a mediastinal teratoma, which is a large tumor in her chest that had compacted her heart and her lungs. So she actually was in heart failure. We were told it was a 50-50 chance of survival through the surgery, and we felt that was enough to proceed.

Alan M. Flake, MD: We spent a lot of time talking about the risks and the benefits and the fact that we had failures as well as successes.

Jed Kelly: They couldn't have been more professional and caring in making us feel like we were making the right decision. And no matter what decision we made, they would have been fully supportive of.

Lori J. Howell, RN: The Center for Fetal Diagnosis and Treatment at CHOP is one of a handful of centers throughout the United States and, in fact, the world and undoubtedly the largest.

Alan M. Flake, MD: CHOP was really the ideal place to develop a center.

N. Scott Adzick, MD: CHOP is an unbelievable place, so there's layers and layers of expertise.

Lori J. Howell, RN: What we know and are able to tell families because we've lived it is an amazing resource for families and, with the opening of the Garbose Family Special Delivery Unit, we have continually improved the outcome of mother and baby.

Alan M. Flake, MD: The SDU at CHOP is the first of its kind, where you deliver the fetus in a Children's Hospital, where all of this services that that fetus needs are immediately available.

Jed Kelly: Dr. Adzick described some of the early procedures to us and how much it had changed and just from where he was and where he had come to when he operated on Addison was an amazing change, an amazing improvement. And I expect it to continue.

Lori J. Howell, RN: Babies are still dying. We don't understand enough. But our lab here is entirely focused on learning everything we possibly can about the mysteries of the fetus.

Holly L. Hedrick, MD: There is incredible research endeavor that is always knocking on the next door.

Lori J. Howell, RN: From maternal safety, imaging techniques, to open fetal surgery, to now minimally invasive fetal surgery, and ultimately to stem cell and gene therapy.

Alan M. Flake, MD: In the next five years, we'll be treating a child with sickle cell disease in utero with a single injection of cells.

Mary Kelly: We're going to see kids who had diagnoses that there was no hope for. We're going to see these kids actually surviving and living great lives because of the interventions that they're doing and because of the research that they're doing.

Lori J. Howell, RN: We see teenagers now, when before we saw fetuses. And that work would not have been possible without the support from donors.

Alan M. Flake, MD: Philanthropy is an opportunity to have a tremendous impact.

N. Scott Adzick, MD: And what better thing to fund than babies? I mean, because you're funding a cure for a lifetime.

Jed Kelly: Truly they are saving lives and making families whole and happy.

Lori J. Howell, RN: The ultimate payoff is seeing mom come back with her child and how normal they are and how active they are and how smart they are.

Jed Kelly: Addison is everything a three-year-old little girl should be. She is nosy. She doesn't listen. She smiles. She's happy. She likes wearing her dresses.

Mary Kelly: Addison is a delight. People say to me, "She's so happy." And she has just got such a bright love of life.

Jed Kelly: And you watch her grow, and you watch her smile, and you watch how she changes, and how she's conquered the obstacles in her way, and you're amazed.

Mary Kelly: When she was born and so sick, I never would have imagined that she would be doing as well as she is today. And, if the Center for Fetal Diagnosis and Treatment was not there or if we were not made aware of it, Addison would not be here today.

Jed Kelly: People that know her, they look at her and they can't believe it. And all you can do is you sit there and you say, "Look at her now."

Addison: Look at me now.

Child 1: Look at me now.

Child 2: Look at me now. Look at me now.

Child 3: Look at me now.

Child 4: Look at me now.

Twins: Hello.

Twins' Mother: Say look at me now.

Twins: Look at me now.

Twins' Mother: Say hooray.

Twins: Yay.

Child 5: Look at me now.

Child 6: Look at me now.

Child 7: Look at me now.

Child 8: Look at me now.

Child 9: Look at me now.

Child 10: Look at me now.

Child 11: See me now.

Child 12: Look at me now.

Child 13: Look at me now.

Child 14: Look at me now.

Child 15: Look at me now.

Child 16: Look at me now.

Twins' Mother: Say bye.

Twins: Bye.