Boston Children's Hospital Milestones


Boston Children’s Hospital opens as a 20-bed facility at 9 Rutland Street in Boston's South End.


Children's establishes the nation's first laboratory for the modification and production of bacteria-free milk.


Dr. William Ladd devises procedures for correcting various congenital defects such as intestinal malformations, launching the specialty of pediatric surgery.


Dr. James Gamble analyzes the composition of body fluids and develops a method for intravenous feeding that saves the lives of thousands of infants at risk of dehydration from diarrhea.


Dr. Louis Diamond characterizes Rh disease, in which a fetus's blood is incompatible with its mother's. Diamond later develops exchange transfusion to treat the disease.


Dr. Robert Gross performs the world's first successful surgical procedure to correct a congenital cardiovascular defect, ushering in the era of modern pediatric cardiac surgery.


Dr. Sidney Farber achieves the world's first successful remission of acute leukemia. He goes on to found the Dana-Farber Cancer Institute.


Dr. John Enders and his colleagues win the Nobel Prize for successfully culturing the polio virus in 1949, making possible the development of the Salk and Sabin vaccines. Enders and his team went on to culture the measles virus.


Dr. Judah Folkman publishes "Tumor angiogenesis: therapeutic implications" in the New England Journal of Medicine. It is the first paper to describe Folkman's theory that tumors recruit new blood vessels in order to grow.


Dr. Stuart Orkin develops restriction endonuclease mapping to diagnose thalassemia in utero.  A similar technique led to the development of prenatal tests for sickle cell anemia in 1982.


Children's physicians report the first surgical correction of hypoplastic left heart syndrome, a defect in which an infant is born without a left ventricle. The procedure is the first to correct what previously had been a fatal condition.


The Howard Hughes Medical Institute funds a major research program in molecular genetics, the first HHMI program at a pediatric hospital.

1986 Children's surgeons perform the hospital's first heart transplant.


Drs. Louis Kunkel and Stuart Orkin and their research teams develop the technique of positional cloning to identify the genes responsible for Duchenne muscular dystrophy and chronic granulomatous disease, respectively.

1987 The gene for a brain protein found in the degenerative nerve tissue of Alzheimer's patients is isolated and located on chromosome 21 by Dr. Rachael Neve. 


Researchers in Neurology and Genetics discover that beta amyloid, a protein that accumulates in the brains of people with Alzheimer's disease, is toxic to neurons, indicating the possible cause of the degenerative disease.


Dr. Joseph Murray, chief of Plastic Surgery emeritus, wins the Nobel Prize for his pioneering work in organ transplantation.

1993 A team led by Dr. Carlo Brugnara discovers that a common antifungal medication, clotrimazole, prevents dehydration in red blood cells, a factor in sickle cell disease. 


Boston Combined Residency Program (BCRP) formed.

1997 Endostatin, one of the most potent inhibitors of angiogenesis, is discovered by Drs. Michael O'Reilly and Judah Folkman.

1998 Dr. Anthony Atala successfully transplants laboratory-grown bladders into dogs, a major advance in the growing field of tissue engineering.  

1998 Dr. Evan Snyder clones the first neural stem cells from the human central nervous system.


Dr. Todd Golub first uses gene expression microarrays to differentiate cancers. 

1999 The FDA approves the use of CardioSEAL, a minimally invasive device invented by Dr. James Lock that closes holes in the hearts of the most seriously ill cardiac patients. 


Dr. Frederick Alt finds that end-joining proteins maintain the stability of DNA, helping to prevent the chromosomal changes that precede cancer. 


Children's performs the world's first successful fetal repair of hypoplastic left heart syndrome in a 19-week-old fetus.

2001 Dr. Lois Smith demonstrates that insulin-like growth factor 1 is critical to blood vessel growth in the eye, and that its loss in premature babies may lead them to develop retinopathy of prematurity. 


Dr. Nader Rifai co-authors a landmark study showing that a simple and inexpensive blood test for C-reactive protein is a more powerful predictor of a person's risk of heart attack or stroke than LDL cholesterol. 


Drs. Scott Pomeroy and Todd Golub use microarray gene expression profiling to identify different types of brain tumors and predict clinical outcomes. 


Drs. Heung Bae Kim and Tom Jaksic develop, test and successfully perform the world's first-ever serial transverse enteroplasty (STEP) procedure, a potential lifesaving surgical procedure for patients with short bowel syndrome. 


Children's surgeons perform New England's first multi-visceral organ transplant when an 11-month-old boy receives a stomach, pancreas, liver and small intestine from a single donor. 

2004 Dr. Marsha Moses and her colleagues show that ADAM 12, when found in urine, is a reliable indicator of the presence of breast cancer.


Dr. Stephen Harrison and colleagues show how a key part of the human immunodeficiency virus (HIV) changes shape, triggering other changes that allow the AIDS virus to enter and infect cells. 

Dr. Raif Geha discovers a gene mutation that causes common variable Immunodeficiency (CVID) and IgA deficiency. 


Dr. Scott Armstrong identifies self-renewal genes that turn a normal blood cell progenitor into a leukemic stem cell. 

2006 Dr. David Pellman discovers a set of genes whose loss is only lethal in hyperdiploid cells and are therefore therapeutic targets in hyperdiploid cancer cells. 

Dr. Dale Umetsu and colleagues characterize NKT cells, which may play an important role in causing asthma, even in the absence of conventional T-helper cells. 


Dr. Hannah Kinney links sudden infant death syndrome (SIDS) to abnormalities in the brainstem serotonin system, which regulates breathing, blood pressure, body heat and arousal. 

Children’s urologists successfully implant laboratorygrown bladders, the first completely tissue-engineered organs to be implanted in humans, in seven children with spina bifida. 

Dr. Doug Cowan creates a tissue-engineered, electrically conductive implant for the heart and shows that it functions well in mice. 

Drs. Sean Wu, Stuart Orkin and colleagues discover a type of stem cell that is the precursor to at least two main cell types that form the heart. 


Dr. Charles Nelson proves that abandoned children do much better cognitively if moved from institutions to foster care. 


Dr. Len Zon discovers that prostaglandin E2 greatly stimulates the growth of blood and probably other tissue stem cells.


Dr. Lois Smith finds that omega-3-polyunsaturated fatty acids reduce pathological retinal angiogenesis and are a potential therapy for retinopathy of prematurity. 

Dr. David Ludwig demonstrates that diets rich in rapidly digested carbohydrates not only expand waistlines, but may also cause fatty liver disease. 

Cardiac surgeons Drs. Virna Sales and John Mayer create living, growing heart valves in an animal model using tissue engineering techniques. 

Dr. Morris White and colleagues show that reducing insulin signaling specifically in the brain can prolong lifespan in mammals. 


Dr. George Daley discovers how to reprogram human somatic cells to pleuripotent stem cells with defined transcription factors. 


Dr. Chris Walsh and his colleagues identify several genetic loci that cause autism. 


Dr. Rani George finds that activating mutations in the receptor tyrosine kinase ALK cause some cases of neuroblastoma. 


Drs Vijay Sankaran and Stuart Orkin discover that the fetal hemoglobin to adult hemoglobin switch is controlled by the BCL11A transcription factor. This solves a decades old problem in hematology and has important implications for the treatment of sickle cell disease and thalassemias. 


Dr. Zhi He observes that stimulation of the mTOR pathway greatly increases axon regeneration after CNS injury. Subsequently, in 2012, Dr. He describes methods for achieving robust and sustained axon regeneration.

2008 A consortium led by Dr. Joel Hirschhorn discovered six new genetic variants linked to obesity. Most are active in the brain, suggesting that differences in appetite regulation contribute to obesity.
Neurobiology researchers at Children's successfully get damaged nerves to recover and regrow in a mouse model by temporarily silencing genes that normally prevent regeneration. 

2008 Dr. Scott Armstrong discovers MLL is caused by an epigenetic change that leads DOT1L to alter chromosome structure and activate normally silent genes. 

2008 Children’s neuroscientists identify Npas4, the first known "master switch" in brain cells to orchestrate the formation and maintenance of inhibitory synapses. 

2008 Dr. Takao Hensch and colleagues describe Otx2, a factor that triggers the learning by initiating a “critical period,” a window of heightened plasticity when the brain can readily make new connections. 

2008 Manton Center for Orphan Disease Research founded.


Immune Disease Institute joins Children's Hospital Boston as the Program in Cellular and Molecular Medicine. 


Drs. George Daley and Richard Gregory show that the microRNA, Lin 28, plays an important role in germ cell development and cancer. 


Drs. Len Zon and George Daley discover that blood flow triggers development of hematopoietic stem cells. 

2011 Drs. Stuart Orkin, Vijay Sankaran and their colleagues are able to correct sickle cell disease in mice by silencing BCL11A, which shows that the fetal hemoglobin switch can be reversed. 


Drs. Luigi Notarangelo, Sung-Yun Pai and David Williams achieve the first successful treatment of severe combined immunodeficiency by gene therapy in the US.


Dr. Heung Bae Kim develops novel method to stretch arteries in vivo for repair of arterial defects.


Standardized Clinical Assessment and Management Plans (SCAMPS) method developed for reducing costs and variability of care and improving outcomes. 

2012 Dr Gabriel Corfas shows that social experience regulates prefrontal cortex myelination and is essential for normal cognitive function. 


Drs. Amy Starmer, Ted Sectish and Chris Landrigan develop a patient handoff method (I-PASS) that greatly reduces medical errors and preventable adverse events

2013 Dr. Dan Bauer discovers an erythroid specific enhancer of BCL11A whose deletion raises fetal hemoglobin without affecting BCL11A in the brain and lymphocytes where it is needed. The discovery opens the door to gene editing of BCL11A as a treatment for sickle cell disease and thalassemia. 

Dr. Joseph Majzoub finds that MRAP2, a protein that regulates melanocortin signaling, is involved in body weight regulation in humans. 


Drs. Jeff Burns and Tracy Wolbrink launch OPENPediatrics, an innovative web-based digital learning platform linking physicians and nurses across the world

2014 Dr. Rani George discovers that neuroblastomas that overexpress MYC oncoproteins are selectively killed, without systemic toxicity, by inhibiting cyclin-dependent kinase 7 (CDK7). 

Dr. Fernando Camargo discovers that the Hippo- signaling pathway maintains the differentiated hepatocyte state. Loss of Hippo causes hepatocytes to revert to a progenitor state. 

2014 Dr. Derrick Rossi devises a procedure to reprogram myeloid cells into hematopoietic stem cells. 

Dr. Carla Kim identifies mechanisms that drive the differentiation of lung stem cells and contribute to alveolar repair after injury. 

2015  Dr. Joel Hirschhorn and others identify a large number of genes that contribute to obesity and body fat distribution. 

2015  Dr. Len Zon defines the perivascular hematopoietic stem cell niche and shows that epoxyeicosatrienoic acid lipids enhance HSC engraftment.  

2015 Dr Beth Stevens wins MacArthur “Genius” Award for defining the role of microglia in synapse pruning in development and Alzheimer’s disease.

2015 Dr Len Zon shows that reversion to a neural crest identity initiates the first cancerous cell in melanoma.

Dr Louis Kunkel and his colleagues show that overexpression of the Jagged protein ameliorates Duchenne muscular dystrophy suggesting a possible therapy for the disease. 

2015 Dr. Hao Wu visualizes the structure of the inflammasome, which activates innate immunity, and how it is assembled. 

Dr. Umut Ozcan discovers that Celastrol, a pentacyclic triterpene extracted from the roots of the thunder god vine plant, is a leptin sensitizer and a powerful anti- obesity agent. 

2016 Dr Len Zon shows that reversion to a neural crest identity initiates the first cancerous cell in melanoma. 

2016 Dr. Min Dong shows that the Frizzled proteins are the gastrointestinal receptors for C. difficile toxin. 

Dr. Beth Stevens reports the important discovery that the complement pathway and microglia, which prune excess synapses during normal brain development, are inappropriately activated and cause synaptic loss early in Alzheimer’s disease. 

Dr. Judy Lieberman discovers a new innate pathway for intracellular killing of bacteria by gasdermin D, which binds to the bacterial membrane and forms a lethal pore. 

Dr. George Daley and his colleagues find that loss of the let-7 microRNA family plays a key role in the development of neuroblastomas and is associated with a poor outcome. 


Dr. Seth Rakoff-Nahoum discovers that some gut bacteria cooperate by metabolizing nutrients for each other; likely the first of many examples of microbial symbiosis. 

2017 Researchers at Children’s and Beth Israel Deaconess Hospitals discover how the Ube3a gene impairs sociability in autism.