Stories about: Pediatrics

‘Pull’ from an implanted robot could help grow stunted organs

Surgeons at Boston Children’s Hospital have long sought a better solution for long-gap esophageal atresia, a rare birth defect in which part of the esophagus is missing. The current state-of-the art operation, called the Foker process, uses sutures anchored to children’s backs to gradually pull the unjoined ends of esophagus until they’re long enough to be stitched together. To keep the esophagus from tearing, children must be paralyzed in a medically induced coma, on mechanical ventilation, for one to four weeks. The lengthy ICU care means high costs, and the long period of immobilization can cause complications like bone fractures and blood clots.

Now, a Boston Children’s Hospital team has created an implantable robot that could lengthen the esophagus — and potentially other tubular organs like the intestine — while the child remains awake and mobile. As described today in Science Roboticsthe device is attached only to the tissue being lengthened, so wouldn’t impede a child’s movement.

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Science and medicine in 2018: What’s the forecast?

weather predictions

Vector consulted its many informants to find out which way the wind will blow in 2018. Here are their predictions for what to expect in genetics, stem cell research, immunology and more.

GENETICS

Gene-based therapies mature

We will continue to see successes in 2018 reflecting the maturation of gene therapy as a viable, generalizable platform for curing many rare diseases. Also, we will see exciting new applications of other maturing platforms, like CRISPR/Cas9 gene editing and oligonucleotide therapies for neurologic diseases, building on the success of nusinersen for spinal muscular atrophy.

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2017 pediatric biomedical advances at Boston Children’s Hospital: Our top 10 picks

New tools and technologies fueled biomedicine to great heights in 2017. Here are just a few of our top picks. All are great examples of research informing better care for children (and adults).

1. Gene therapy arrives

(Katherine C. Cohen)

In 2017, gene therapy solidly shed the stigma of Jesse Gelsinger’s 1999 death with the development of safer protocols and delivery vectors. Though each disease must navigate its own technical and regulatory path to gene therapy, the number of clinical trials is mounting worldwide, with seven gene therapy trials now recruiting at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. In August, the first gene therapy won FDA approval: CAR T-cell therapy for pediatric acute lymphoblastic leukemia.

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One-time hydrocephalus operation, alternative to shunting, brings good outcomes for babies

infant hydrocephalus archival photos
(Flickr/Wikimedia Commons)

Hydrocephalus, literally “water on the brain,” is an abnormal build-up of cerebrospinal fluid in the brain cavities known as ventricles. In infants, it can be congenital (it often accompanies spina bifida, for example), or it can be caused by brain hemorrhage or infection. The usual treatment is surgery to implant a shunt, which drains the excess fluid into the abdomen, relieving pressure on the brain.

But over time, shunts nearly always fail, requiring emergency neurosurgery to repair or replace them. But emergency neurosurgery is not something that’s readily available outside of metropolitan areas. Untreated, hydrocephalus causes progressive brain damage and usually death.

What if a one-time operation could treat hydrocephalus permanently? In today’s New England Journal of Medicine, a randomized trial shows good results with a minimally invasive, relatively inexpensive shunt alternative called endoscopic third ventriculostomy with choroid plexus cauterization (ETV/CPC).

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Taking a sideswipe at high-risk neuroblastoma

Microscopy image of human neuroblastoma cells.
Human neuroblastoma cells.

Cancer and other diseases are now understood to spring from a complex interplay of biological factors rather than any one isolated origin. New research reveals that an equally-nuanced approach to treating high-risk neuroblastoma may be the most effective way to curb tumor growth.

One challenge in treating pediatric cancers like neuroblastoma is that they are not initiated from the same kinds of genetic mutations as adult cancers, which usually arise from mutations related to an accumulation of DNA replication errors or environmental factors. In contrast, childhood cancers more often stem from genetic duplications, deletions or translocations, the latter of which occurs when a gene sequence switches its location from one chromosome to another.

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Giving young organ transplant recipients a mobile reminder that might save their lives

Flow chart showing how the medication adherence app interacts with patients and a near-field-communication-enabled pillboxIn the U.S., more than 1,700 children receive organ transplants each year. Following transplantation, they must take immunosuppressants and steroids to protect their transplanted organ from being attacked by their own immune system.

But transplant teams know that kids are 60 percent more likely than adults to struggle with keeping a strict medication schedule. That puts the longevity of donated organs — and the lives of organ recipients — at unnecessary risk.

This challenge inspired a team of pediatric transplant experts at the Boston Children’s Hospital to develop a mobile application for smartphones that could serve as a portable reminder and a resource to support medication adherence.

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A “half-hearted” solution to one-sided heart failure

Illustration showing how the system supports a failing right ventricle
Illustration showing sectional view of a heart with the soft robotic system helping to draw blood into (left) and pump blood out (right) of the heart’s right ventricle.

Soft robotic actuators, which are pneumatic artificial muscles designed and programmed to perform lifelike motions, have recently emerged as an attractive alternative to more rigid components that have conventionally been used in biomedical devices. In fact, earlier this year, a Boston Children’s Hospital team revealed a proof-of-concept soft robotic sleeve that could support the function of a failing heart.

Despite this promising innovation, the team recognized that many pediatric heart patients have more one-sided congenital heart conditions. These patients are not experiencing failure of the entire heart — instead, congenital conditions have caused disease in either the heart’s right or left ventricle, but not both.

Read our Vector story on the soft robotic heart sleeve that mimics cardiac muscles.

“We set out to develop new technology that would help one diseased ventricle, when the patient is in isolated left or right heart failure, pull blood into the chamber and then effectively pump it into the circulatory system,” says Nikolay Vasilyev, MD, a researcher in cardiac surgery at Boston Children’s.

Now, Vasilyev and his collaborators — researchers from Boston Children’s, the Harvard John A. Paulson School of Engineering and Applied Sciences and the Wyss Institute for Biologically Inspired Engineering at Harvard University — have revealed their soft robotic solution. They describe their system in a paper published online in Science Robotics today.

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Breastfeeding protects against food allergy: We have evidence

Mother breastfeeding her baby. Is she helping her child avoid food allergy?

Eating allergenic foods during pregnancy can protect your child from food allergies, especially if you breastfeed, suggests new research. The findings, in a mouse model of allergy, underscore recent advice that pregnant or nursing mothers not avoid allergenic foods like eggs and peanuts.

The study is the first controlled investigation to demonstrate protection against food allergy from breast milk, while also pointing to a biological mechanism for inducing food tolerance. It was published online today in the Journal of Experimental Medicine.

“Whether mothers should eat allergenic foods during pregnancy or avoid them has been controversial,” says Michiko Oyoshi, PhD, of Boston Children’s Division of Allergy and Immunology, who led the study in collaboration with Richard Blumberg, MD, of Brigham and Women’s Hospital, her co-senior author.

“Different studies have found different results, in part because it’s hard in human studies to know when mothers and babies first encountered a specific food,” says Oyoshi. “But in a mouse model, we can control exposure to food.”

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Personalized care model enhances preterm babies’ development

NICU baby with his mother
Christian, born at 26 weeks gestation, has lived in the NICU since May. (Photos: Katherine C. Cohen, unless otherwise noted)

November 17, 2017 is World Prematurity Day.

From a cozy, dark and quiet existence, a preterm baby is forced out into a harsh, bright and noisy environment. Instead of being comforted and held securely by their parents, preemies are poked and prodded, hooked up to machines and exposed to jarring sights and smells as their developing brains struggle to realign.

Each year, an estimated 15 million babies around the world — 1 in 10 — are born prematurely. Medical advances enable more of them to live, but often with medical and developmental problems.

Heidelise Als, PhD, director of Neurobehavioral Infant and Child Studies at Boston Children’s Hospital, has worked for more than 30 years to create better outcomes, developing the Newborn Individualized Developmental Care and Assessment Program, or NIDCAP.

The NIDCAP model of care seeks to support the development of fragile newborns and reduce their stress. In a series of studies, Als and colleagues at other hospitals have documented its successes: improvements in lung function, feeding and growth; shorter lengths of stay; a reduction in brain hemorrhage and improved brain function and structure, with brain effects lasting until at least 8 years of age. Benefits have been documented even in medically fragile, very preterm infants and infants with severe intrauterine growth restriction.

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Meeting an unmet need: A surgical implant that grows with a child

Depiction of a growth-accommodating implant expanding in sync with a child's growing heart.
Artist’s rendering showing how a braided, tubular implant could grow in sync with a child’s heart valve. Credit: Randal McKenzie

Medical implants can save lives by correcting structural defects in the heart and other organs. But until now, the use of medical implants in children has been complicated by the fact that fixed-size implants cannot expand in tune with a child’s natural growth.

To address this unmet surgical need, a team of researchers from Boston Children’s Hospital and Brigham and Women’s Hospital have developed a growth-accommodating implant designed for use in a cardiac surgical procedure called a valve annuloplasty, which repairs leaking mitral and tricuspid valves in the heart. The innovation was reported today in Nature Biomedical Engineering.

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