N. Scott Adzick, MD, MMM, is Surgeon-in-Chief at Children’s Hospital of Philadelphia (CHOP), and founder and director of the Richard D. Wood Jr. Center for Fetal Diagnosis and Treatment (CFDT), the largest fetal therapy center of its kind. He is also the C. Everett Koop Professor of Pediatric Surgery at CHOP and a Professor of Surgery, Obstetrics and Gynecology at the University of Pennsylvania School of Medicine.
Dr. Adzick has been an innovator in the field of fetal medicine since its inception, pursuing groundbreaking prenatal treatment for debilitating birth defects. He co-led the NIH-funded Management of Myelomeningocele Study (MOMS) at CHOP, breakthrough research proving fetal surgery for spina bifida results in significantly improved outcomes. His steadfast dedication to quality of surgical care and translational research has profoundly shaped the medical community’s approach to pediatric and fetal surgery.
In this piece, Dr. Adzick reflects on the impact the center he helped establish 30 years ago has had on the field and on families.
What makes the Center for Fetal Diagnosis and Treatment so special?
Innovation and teamwork. Also, a passion for excellence with regard to caring for patients and families. And we have the advantage — not only at the Center for Fetal Diagnosis and Treatment, but also across Children’s Hospital of Philadelphia — of having layers and layers of expertise to create seamless teams. It’s just wonderful.
How have research and innovations at CHOP shaped the field of fetal medicine?
Since our founding in 1995, we’ve dominated the field. We’ve been behind numerous firsts, including the first successful fetal surgery for sacrococcygeal teratoma, the first successful early gestation fetal surgery for myelomeningocele (MMC), the first fetoscopic surgery for multiples, the first successful fetal surgery for mediastinal teratoma, the first successful resection of a fetal pericardial teratoma, and the first special delivery unit exclusively dedicated to the comprehensive care of the mother and fetus with a birth defect. We've pioneered groundbreaking fetal surgical techniques. Notably, the development of in utero therapies, such as fetal surgery for MMC, has transformed care for high-risk pregnancies. We helped develop the ex utero intrapartum therapy (EXIT) procedure. We established fetal MRI for diagnosis of birth defects and developed better sonographic prognostic indicators for fetal lung lesions and congenital diaphragmatic hernia (CDH). The list goes on.
The field of fetal diagnosis and treatment is constantly evolving. What are you most excited about (at CHOP or beyond) and how soon do you think it will have an impact?
We tend to overestimate what will happen in the next year or two, and underestimate what will be accomplished 10 years from now. Our ongoing research continues to push the boundaries of prenatal care, offering new hope for treating and preventing birth defects before birth.
One of the biggest things on the horizon is fetal gene therapy and in utero gene editing using CRISPR technology to treat single gene disorders, which is happening right here in the labs at CHOP. It’s not ready for clinical primetime, but it’s going to happen. It has to be done ethically, there have to be all the safeguards in place. Once it’s ready, the field will just explode. It will transform our current approach of doing fetal surgery operations one by one, with big teams and huge efforts. It will affect thousands and thousands of unborn patients.
Other exciting advances in the works are:
- Clinical Outcomes Data Archive (CODA). This prospective data capture platform dissolves barriers between clinical care and research, allowing us to do research-grade data analysis to inform patient counseling about prognosis.
- Birth Defects Biorepository (BDB). This is a centralized place where we collect and store biological samples from mothers, fathers, babies, children and other family members to be used in future research studies. To date, we have whole genome sequencing available on nearly 3,000 total subjects.
- Artificial Womb. CHOP scientists are moving closer to clinical application of a unique fluid-filled environment that would provide a lifesaving bridge to take extremely premature babies safely from 23–24 weeks’ gestation with normal growth and development to 28–30 weeks’ gestation.
- Delivery Room of the Future Frontier Program. A team of CHOP physicians and scientists are using high-quality data and their own deep experience to create algorithms to address resuscitation of newborns with congenital anomalies and develop national care guidelines to optimize outcomes.
- Congenital Diaphragmatic Hernia Frontier Program. Leveraging CHOP’s standing as the largest CDH treatment program in the country, this initiative strives to eliminate mortality and morbidity through new research and the development of novel devices. Our team's research continues to translate to improved care: Promising early results have led to expanded criteria for fetoscopic endoluminal tracheal occlusion (FETO) for fetuses with CDH.
- Advancing Craniofacial Treatment with Genomics and Gene Therapy (ACTG) Frontier Program. This program is poised to harness genomic technologies to spearhead innovations in gene therapy, improve diagnostics, predict treatment outcomes, and develop new therapies for children with cleft and craniofacial conditions.
Why is experience so important — especially when it comes to fetal surgery and rare and complex conditions?
Experience and expertise minimize incremental risk factors and lead to superb outcomes. That’s the bottom line.
In the CHOP Department of Surgery as a whole, we’ve been guided by the following core principles:
- Attract and grow the best faculty
- Create the right clinical, educational, research and financial environment
- Establish multidisciplinary centers that draw regional, national and international referrals
- Innovate via new surgical techniques, translational research and clinical trials, being a creative force to overcome the unsolved problems and enhance the surgical care of children
- Lead in surgical education
- Cure, educate and discover, with a passion for excellence
Beyond your long list of medical achievements and accolades, what are you most proud of in your career?
There’s the quote, “When you operate on a child, you save not just a life, but a lifetime.” So, the No. 1 thing I’m proud of is saving lifetimes, whether it’s in terms of life or death, surviving, or improved quality of life for a long, fruitful, robust, wonderful life.
I’m also proud of what we’ve built, not only in the CFDT but in the CHOP Department of Surgery and beyond, through the establishment of multidisciplinary centers, some of which I noted earlier.
You've been a leading figure in the field of fetal medicine, pioneering groundbreaking advancements. What initially sparked your interest in this area of medicine?
When I was a general surgery resident at Massachusetts General Hospital, I learned about a young pediatric surgeon and one-of-a-kind creative genius named Michael Richard Harrison, MD, at the University of California, San Francisco (UCSF). At that time, maternal-fetal ultrasound was being refined, and for the first time we had the ability to diagnose birth defects in utero. As pediatric surgeons, Dr. Harrison and I were both frustrated by taking care of babies with life-threatening malformations causing such progressive and severe organ damage before birth that the babies didn't survive. He wanted to repair life-threatening defects before birth, and I instantly knew that needed to be done. I received some grant support from the NIH and the American College of Surgeons and set out more than 40 years ago to do a two-year research fellowship with Mike. The rest is history.
In looking back on a career, people focus on the successes. Were there failures and struggles, and how did they shape the work and your commitment to the field?
Open fetal surgical repair of CDH was tested and then abandoned because the most severely affected fetuses have liver herniation, and liver reduction into the fetal abdomen compromised umbilical blood flow during fetal surgery. That was enormously frustrating, but we learned a lot and simply kept going, never giving in or giving up with respect to patients. Those learnings, in turn, led to opportunities to enhance lung growth through FETO. Our CDH program is now the largest, most experienced of its kind in the world, with an unmatched level of ongoing collaboration.
What is your advice to a young scientist/surgeon/clinician/researcher who has an idea or research goal today that seems hard to believe?
My advice to young surgical research fellows and faculty is to follow your passion. It is very difficult to do both clinical work and research, but it is vitally important in order to advance the field.
As one of my teachers at Harvard Medical School and Boston Children’s Hospital, Dr. Judah Folkman, taught me:
As long as there is an unconquered disease, an injury that cannot be repaired, or a method of prevention that remains beyond reach, we have an obligation to conduct research. Research represents hope, and for many patients and families, hope is the best thing we have to offer. We pursue our investigations so that one day we can offer more than hope, we can offer health.
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N. Scott Adzick, MD, MMM, is Surgeon-in-Chief at Children’s Hospital of Philadelphia (CHOP), and founder and director of the Richard D. Wood Jr. Center for Fetal Diagnosis and Treatment (CFDT), the largest fetal therapy center of its kind. He is also the C. Everett Koop Professor of Pediatric Surgery at CHOP and a Professor of Surgery, Obstetrics and Gynecology at the University of Pennsylvania School of Medicine.
Dr. Adzick has been an innovator in the field of fetal medicine since its inception, pursuing groundbreaking prenatal treatment for debilitating birth defects. He co-led the NIH-funded Management of Myelomeningocele Study (MOMS) at CHOP, breakthrough research proving fetal surgery for spina bifida results in significantly improved outcomes. His steadfast dedication to quality of surgical care and translational research has profoundly shaped the medical community’s approach to pediatric and fetal surgery.
In this piece, Dr. Adzick reflects on the impact the center he helped establish 30 years ago has had on the field and on families.
What makes the Center for Fetal Diagnosis and Treatment so special?
Innovation and teamwork. Also, a passion for excellence with regard to caring for patients and families. And we have the advantage — not only at the Center for Fetal Diagnosis and Treatment, but also across Children’s Hospital of Philadelphia — of having layers and layers of expertise to create seamless teams. It’s just wonderful.
How have research and innovations at CHOP shaped the field of fetal medicine?
Since our founding in 1995, we’ve dominated the field. We’ve been behind numerous firsts, including the first successful fetal surgery for sacrococcygeal teratoma, the first successful early gestation fetal surgery for myelomeningocele (MMC), the first fetoscopic surgery for multiples, the first successful fetal surgery for mediastinal teratoma, the first successful resection of a fetal pericardial teratoma, and the first special delivery unit exclusively dedicated to the comprehensive care of the mother and fetus with a birth defect. We've pioneered groundbreaking fetal surgical techniques. Notably, the development of in utero therapies, such as fetal surgery for MMC, has transformed care for high-risk pregnancies. We helped develop the ex utero intrapartum therapy (EXIT) procedure. We established fetal MRI for diagnosis of birth defects and developed better sonographic prognostic indicators for fetal lung lesions and congenital diaphragmatic hernia (CDH). The list goes on.
The field of fetal diagnosis and treatment is constantly evolving. What are you most excited about (at CHOP or beyond) and how soon do you think it will have an impact?
We tend to overestimate what will happen in the next year or two, and underestimate what will be accomplished 10 years from now. Our ongoing research continues to push the boundaries of prenatal care, offering new hope for treating and preventing birth defects before birth.
One of the biggest things on the horizon is fetal gene therapy and in utero gene editing using CRISPR technology to treat single gene disorders, which is happening right here in the labs at CHOP. It’s not ready for clinical primetime, but it’s going to happen. It has to be done ethically, there have to be all the safeguards in place. Once it’s ready, the field will just explode. It will transform our current approach of doing fetal surgery operations one by one, with big teams and huge efforts. It will affect thousands and thousands of unborn patients.
Other exciting advances in the works are:
- Clinical Outcomes Data Archive (CODA). This prospective data capture platform dissolves barriers between clinical care and research, allowing us to do research-grade data analysis to inform patient counseling about prognosis.
- Birth Defects Biorepository (BDB). This is a centralized place where we collect and store biological samples from mothers, fathers, babies, children and other family members to be used in future research studies. To date, we have whole genome sequencing available on nearly 3,000 total subjects.
- Artificial Womb. CHOP scientists are moving closer to clinical application of a unique fluid-filled environment that would provide a lifesaving bridge to take extremely premature babies safely from 23–24 weeks’ gestation with normal growth and development to 28–30 weeks’ gestation.
- Delivery Room of the Future Frontier Program. A team of CHOP physicians and scientists are using high-quality data and their own deep experience to create algorithms to address resuscitation of newborns with congenital anomalies and develop national care guidelines to optimize outcomes.
- Congenital Diaphragmatic Hernia Frontier Program. Leveraging CHOP’s standing as the largest CDH treatment program in the country, this initiative strives to eliminate mortality and morbidity through new research and the development of novel devices. Our team's research continues to translate to improved care: Promising early results have led to expanded criteria for fetoscopic endoluminal tracheal occlusion (FETO) for fetuses with CDH.
- Advancing Craniofacial Treatment with Genomics and Gene Therapy (ACTG) Frontier Program. This program is poised to harness genomic technologies to spearhead innovations in gene therapy, improve diagnostics, predict treatment outcomes, and develop new therapies for children with cleft and craniofacial conditions.
Why is experience so important — especially when it comes to fetal surgery and rare and complex conditions?
Experience and expertise minimize incremental risk factors and lead to superb outcomes. That’s the bottom line.
In the CHOP Department of Surgery as a whole, we’ve been guided by the following core principles:
- Attract and grow the best faculty
- Create the right clinical, educational, research and financial environment
- Establish multidisciplinary centers that draw regional, national and international referrals
- Innovate via new surgical techniques, translational research and clinical trials, being a creative force to overcome the unsolved problems and enhance the surgical care of children
- Lead in surgical education
- Cure, educate and discover, with a passion for excellence
Beyond your long list of medical achievements and accolades, what are you most proud of in your career?
There’s the quote, “When you operate on a child, you save not just a life, but a lifetime.” So, the No. 1 thing I’m proud of is saving lifetimes, whether it’s in terms of life or death, surviving, or improved quality of life for a long, fruitful, robust, wonderful life.
I’m also proud of what we’ve built, not only in the CFDT but in the CHOP Department of Surgery and beyond, through the establishment of multidisciplinary centers, some of which I noted earlier.
You've been a leading figure in the field of fetal medicine, pioneering groundbreaking advancements. What initially sparked your interest in this area of medicine?
When I was a general surgery resident at Massachusetts General Hospital, I learned about a young pediatric surgeon and one-of-a-kind creative genius named Michael Richard Harrison, MD, at the University of California, San Francisco (UCSF). At that time, maternal-fetal ultrasound was being refined, and for the first time we had the ability to diagnose birth defects in utero. As pediatric surgeons, Dr. Harrison and I were both frustrated by taking care of babies with life-threatening malformations causing such progressive and severe organ damage before birth that the babies didn't survive. He wanted to repair life-threatening defects before birth, and I instantly knew that needed to be done. I received some grant support from the NIH and the American College of Surgeons and set out more than 40 years ago to do a two-year research fellowship with Mike. The rest is history.
In looking back on a career, people focus on the successes. Were there failures and struggles, and how did they shape the work and your commitment to the field?
Open fetal surgical repair of CDH was tested and then abandoned because the most severely affected fetuses have liver herniation, and liver reduction into the fetal abdomen compromised umbilical blood flow during fetal surgery. That was enormously frustrating, but we learned a lot and simply kept going, never giving in or giving up with respect to patients. Those learnings, in turn, led to opportunities to enhance lung growth through FETO. Our CDH program is now the largest, most experienced of its kind in the world, with an unmatched level of ongoing collaboration.
What is your advice to a young scientist/surgeon/clinician/researcher who has an idea or research goal today that seems hard to believe?
My advice to young surgical research fellows and faculty is to follow your passion. It is very difficult to do both clinical work and research, but it is vitally important in order to advance the field.
As one of my teachers at Harvard Medical School and Boston Children’s Hospital, Dr. Judah Folkman, taught me:
As long as there is an unconquered disease, an injury that cannot be repaired, or a method of prevention that remains beyond reach, we have an obligation to conduct research. Research represents hope, and for many patients and families, hope is the best thing we have to offer. We pursue our investigations so that one day we can offer more than hope, we can offer health.
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