Neurology | Research & Innovation

Boston Children’s has the largest hospital-based basic neuroscience research enterprise in the United States. We also participate in a variety of clinical trials.

Active areas of investigation include developmental disorders, intellectual disability, autism, neuromuscular disorders, pain, epilepsy, neurometabolic disorders, eye-movement disorders, encephalopathy of prematurity, traumatic brain injury and more. The goal: to create better treatments.

Researchers in Neurology at Boston Children's are fortunate to have cutting edge tools and facilities close at hand to aid discovery. These include:

• genetics, genomics and gene therapy capabilities
• bioinformatics expertise
• tissue banks
• a Human Neuron Core
• a Neurobehavioral Developmental Core
• zebrafish models for drug discovery
• brain electrophysiology facilities
• an electromyography (EMG) laboratory
• the Developmental Neurophysiology Laboratory
• the Epilepsy Genetics Program
• state-of-the-art neuroimaging expertise.

Research Centers in Neurology at Boston Children’s

The Department of Neurology’s research program is focused on the developing brain and seeks to integrate clinical and basic neuroscience. In addition to research housed in the Department, we work closely with:

• The F.M. Kirby Neurobiology Center incorporates basic and translational neuroscience research to understand the nervous system, especially the developing nervous system, at the genetic, molecular, cellular and systems level. Its members aim to understand the brain’s circuitry, learn how disturbances can produce neurodevelopmental disorders and find new treatments.
• The Translational Neuroscience Center seeks to improve the lives of children and adolescents with nervous system disorders through innovative clinical programs, basic science and efficient translation of novel ideas into diagnostic tools and treatments. The Center aims to establish ties between laboratory researchers, clinicians and industry to accelerate translation.
• Intellectual and Developmental Disabilities Research Center (IDDRC): Boston Children’s is one of the oldest members of a national, NIH-funded network of some 20 centers seeking to prevent and treat intellectual and developmental disabilities through biomedical and behavioral research.

Clinical research and innovation in Neurology

Boston Children’s participates in a variety of drug trials, many of them initiated here, as well as clinical studies that track patients to continuously improve our care. We are also testing innovations such bracelets for detecting seizures or hand movements characteristic of neurologic disorders and conduct genetic research to help us better understand rare neurologic conditions.

Basic/translational neuroscience

Synapse development and brain plasticity

Several laboratories at Boston Children’s are investigating how connections or synapses between nerve cells in the developing brain are established, what enables connections to change over time and how synapse development can go awry to cause disease. The labs of Michela Fagiolini, PhD and Chinfei Chen, MD, for example, are using mouse models of Rett syndrome and the visual system to better understand this process. The lab of Beth Stevens, PhD, has revealed the role of microglia and immune molecules in synapse formation and elimination and has shown its relevance to normal brain development as well as disorders like schizophrenia and Alzheimer’s disease.

Brain connectivity and signaling

Several Boston Children’s labs, including those of Mustafa Sahin, MD, PhD, and Xi He, PhD, have investigated how biochemical signals regulate the process of circuit development in the brain. Sahin’s lab focuses on this question by studying tuberous sclerosis, a condition that frequently includes autism, through mouse models and structural brain imaging, aided by computational techniques. Takao Hensch, PhD, studies brain connectivity and the role played by specific “critical periods” in brain development.

Pain control

The lab of Clifford Woolf, PhD, is investigating new ways to selectively inhibit pain nerve fibers, or axons, and is modeling pain in neurons made from patients’ skin cells.

Nerve regeneration

Several labs, including those of Zhigang He, PhD, and Larry Benowitz, PhD, are looking for ways to regenerate nerve fibers in the central nervous system that could someday help patients with optic nerve trauma, glaucoma or spinal cord injury.

Precision medicine for brain tumors

The lab of neurologist-in-chief Scott Pomeroy, MD, PhD, is applying advanced genetics techniques, including genomic sequencing and genome-wide expression studies, to define biologically and clinically distinct subtypes of tumors such as medulloblastoma. Many of the mutations he’s identified are in epigenetic regulatory molecules. Based on his findings, the Children’s Oncology Group is now conducting next-generation clinical trials using molecular markers to stratify brain tumor patients’ disease risk.

Prenatal risk factors and brain development

In the ongoing Extremely Low Gestational Age Newborn (ELGAN) study, Alan Leviton, MD, and colleagues have identified inflammation and being born small for gestational age as risk factors for cerebral palsy.

Read more:

• Two-hit model of brain damage in the very preterm newborn
• Systemic inflammation on postnatal days 21 and 28 and indicators of brain dysfunction 2 years later among children born before the 28th week of gestation

A legacy of discovery in child neurology

From our origins in the 1920s, the Department of Neurology has been pushing for answers. Our first child neurologist-in-chief, Bronson Crothers, MD, began probing the mechanisms of cerebral palsy in the 1950s, and Randolph Byers, MD, who followed in his footsteps, was the first to identify lead poisoning as a cause of learning problems in the 1940s. William Lennox, MD, helped classify childhood epilepsies in the 1920s and went on to define their neurophysiology and genetics. Read more about our history.