Thank you for making Boston Children’s #1

The 2017-18 U.S. News & World Report “Best Children’s Hospitals” rankings were released this morning, and Boston Children’s Hospital has been named the #1 children’s hospital in the nation.

The U.S. News rankings rely most heavily on outcomes. In other words, were we able to make a difference?

At Boston Children’s, that goal is what drives our researchers and innovators to seek answers for all our patients, from the complex to the common. We seek out cures for children with the rarest disorders, whose needs are unimaginable, whose chances of survival may be heartbreakingly low. We also wrestle with everyday conditions like asthma, diabetes, even the flu: Can we prevent them, can we minimize their impact on children’s lives?

This is a moment worth celebrating — but it’s also a moment to give thanks. To our clinicians who challenge the status quo, and then push the boundaries of what’s possible. To parents who increasingly work alongside us, pushing for answers. To a rising tide of biomedical advances — CRISPR, next-generation sequencing, cell therapy, big data analysis. To the young scientists who work, usually behind the scenes, to make things better. To our donors and investors who believe in the power of science and support our efforts.

We created this video to give a taste of what can happen when all of you strive together to make the impossible possible.

Thank you for making Boston Children’s #1.

For more moments worth celebrating, visit bostonchildrens.org/GreatestStories.

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A new inlet to treating neurological disease

Image of brains showing red tracer dye, indicating passage of molecules through the blood-brain barrier
These brain images tell a story about the blood-brain barrier: At left, the brain before injection of red tracer dye. At center, an injection of tracer dye shows only a small amount of molecules can infiltrate the blood brain barrier. At right, a new approach for crossing the blood-brain barrier increases the tracer’s penetration into brain tissue.

The blood-brain barrier was designed by nature to protect the brain and central nervous system (CNS) from toxins and other would-be invaders in the body’s circulating blood. Made up of tightly-packed cells, the barrier allows nutrients to pass into the CNS and waste products from the brain to be flushed out, while blocking entry of harmful substances.

A dysfunctional blood-brain barrier can contribute to CNS diseases including Alzheimer’s and multiple sclerosis (MS). But, ironically, the same blood-brain barrier can keep out drugs intended to treat CNS disease. Scientists have long been seeking ways to overcome this obstacle.

Now, Timothy Hla, PhD, and members of his laboratory in the Boston Children’s Hospital Vascular Biology Program have found a way to selectively control openings in the blood brain barrier to allow passage of small drug molecules.

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Optic nerve regeneration: One approach doesn’t fit all

alpha retinal ganglion cells optic nerve regeneration
Alpha-type retinal ganglion cells (RGCs) in part of an intact mouse retina. The cell axons lead to the optic nerve head (top right) and then exit into the optic nerve. The alpha RGCs are killed by the transcription factor SOX11 despite its pro-regenerative effect on other types of RGCs. (Fengfeng Bei)

Getting a damaged optic nerve to regenerate is vital to restoring vision in people blinded through nerve trauma or disease. A variety of growth-promoting factors have been shown to help the optic nerve’s retinal ganglion cells regenerate their axons, but we are still far from restoring vision. A new study published yesterday in Neuron underscores the complexity of the problem.

A research team led by Fengfeng Bei, PhD, of Brigham and Women’s Hospital, Zhigang He, PhD, and Michael Norsworthy, PhD, of Boston Children’s Hospital, and Giovanni Coppola, MD, of UCLA conducted a screen for transcription factors that regulate the early differentiation of RGCs, when axon growth is initiated. While one factor, SOX11, appeared to be critical in helping certain kinds of RGCs regenerate their axons, it simultaneously killed another type — alpha-RGCS (above)— when tested in a mouse model.

At least 30 types of retinal ganglion cell message the brain via the optic nerve. “The goal will be to regenerate as many subtypes of neurons as possible,” says Bei. “Our results here suggest that different subtypes of neurons may respond differently to the same factors.”

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A new, much needed target for treating Candida albicans

Candida albicans

Fungal diseases commonly bring to mind the words “dangerous” or “difficult to cure.” Now, scientists might just be a step closer to treating diseases caused by one common, problematic fungus, Candida albicans, by targeting a key player unique to fungi in an important growth pathway.

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Caremap: Mobile app lets families track their children’s health, their way

Caremap
Carson Domey fires up Caremap

Family caregivers — as well as older children and adolescents — now have a powerful health data tracker. With a free iPhone app called Caremap, they can securely store and organize vital medical information, share it with health professionals, track health metrics important to them and gain insights to inform care.

For Michelle Domey, that means keeping close tabs on her son Carson’s Crohn’s disease. It means understanding early warning signs and what triggers a flare, like not getting enough sleep. “When he has a flare, the app is something we could take into an appointment,” she says. “We have historical data that can show us what may have triggered it.”

Available for free in the iTunes App Store, Caremap was developed by Boston Children’s Hospital’s Innovation & Digital Health Accelerator (IDHA) in collaboration with Duke Health System. It was built using Apple’s open source CareKit framework.

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Why is one twin sometimes smaller than the other? The answer may lie in the placenta

placental oxygen transport may help determine fetal size

When a baby is born small, it’s often chalked up to genetics or to maternal risk factors like poor nutrition or smoking. A study of twin pregnancies, published today in Scientific Reports, finds another factor that can be measured prentally: slower transport of oxygen from mother to baby across the placenta.

The study, part of the NIH-funded Human Placenta Project, is the first to make a direct connection between placental oxygen transport and birth outcomes. It relies on a new, noninvasive technique called Blood-Oxygenation-Level-Dependent (BOLD) MRI. Developed by P. Ellen Grant, MD, director of the Fetal-Neonatal Neuroimaging and Developmental Science Center at Boston Children’s Hospital and Elfar Adalsteinsson, PhD at MIT, it maps oxygen delivery across the placenta in real time.

“Until now, we had no way to look at regional placental function in vivo,” says Grant.

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A surprising new link between inflammation and mental illness — and a potential drug to protect the brain

A synapse being attacked by microglia, which causes neuropsychiatric symptoms in lupus
In the brain, a synapse (red – see diagonal “spine” across center of photo) is seen being wrapped around and attacked by immune cells called microglia (green), leading to synapse loss. Credit: Carroll lab / Boston Children’s Hospital

Up to 75 percent of patients with systemic lupus erythematosus — an incurable autoimmune disease commonly known as “lupus” —  experience neuropsychiatric symptoms.  But so far, our understanding of the mechanisms underlying lupus’ effects on the brain has remained murky.

“In general, lupus patients commonly have a broad range of neuropsychiatric symptoms, including anxiety, depression, headaches, seizures, even psychosis,” says Allison Bialas, PhD, a research fellow working in the lab of Michael Carroll, PhD, of Boston Children’s Hospital. “But their cause has not been clear — for a long time it wasn’t even appreciated that these were symptoms of the disease.”

Collectively, lupus’ neuropsychatric symptoms are known as central nervous system (CNS) lupus. Their cause has been unclear until now.

Perhaps, Bialas thought, changes in the immune systems of lupus patients were directly causing these symptoms from a pathological standpoint. Working with Carroll and other members of his lab, Bialas started out with a simple question, and soon, made a surprising finding – one that points to a potential new drug for protecting the brain from the neuropsychiatric effects of lupus and other diseases. The team has published its findings in Nature.

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Helping tissue grafts build a blood supply: Less is more

blood vessels in vivo

For a tissue graft to survive in the body — whether it’s a surgical graft or bioengineered tissue — it needs to be nourished by blood vessels, and these vessels must connect with the recipient’s circulation. While scientists know how to generate blood vessels for engineered tissue, efforts to get them to connect with the recipient’s vessels have mostly failed.

“Surgeons will tell you that when putting tissue in a new location in the body, the small blood vessels don’t connect at the new site,” says Juan Melero-Martin, PhD, a researcher in Cardiac Surgery in Boston Children’s Hospital. “If you want to engineer a tissue replacement, you’d better understand how the vessels get connected, because if the vessels go, the graft goes.”

Melero-Martin and colleagues have uncovered several strategies to help these connections form, as they describe online today in Nature Biomedical Engineering. The strategies could help improve the success of such procedures as heart patching, bone grafting, fat transplants and islet transplantation.

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Building a clinician-centric healthcare accelerator

busy doctors need a boost from healthcare accelerators

Who better to innovate in healthcare than doctors, nurses and others on the front lines? They know what’s broken. They want to fix it. And they understand healthcare’s complexity. Some have taken part in hackathons and pitch competitions. But once these events are over, most find they’re too busy to develop their ideas and that they lack the necessary business expertise.

In Harvard Business Review this week, leaders of the Innovation & Digital Health Accelerator (IDHA) at Boston Children’s Hospital, with Kevin Churchwell, MD, executive VP of health affairs, describe how (and why) the hospital formed an in-house accelerator program in 2016. In a single year, the program engaged more than 300 clinicians, researchers and administrators in more than 25 clinical departments, offering custom, “just in time” support. Nine projects were accelerated, including three new startups.

A central tactic is the “Opportunity SPRINT,” a 90-minute triage session that brings hospital teams together with business strategists, subject matter experts, technologists and, sometimes, parents and patients. Even when an idea isn’t immediately embraced, SPRINTs are designed to be educational and constructive, inspiring clinicians to reimagine their idea and come back with a better one.

Read more in HBR and check out IDHA’s portfolio.

 

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Under the hood of healthcare innovation: Jared Hawkins and the digital phenotype

Jared Hawkins Boston Children's Hospital

What does it take to change healthcare for the better? In the second of a two-part series on digital health innovators at Boston Children’s Hospital, we profile Jared Hawkins, MMSc, PhD. Like Gajen Sunthara, MSc, featured in part one, Hawkins was named among MedTech Boston’s 40 Under 40 Healthcare Innovators for 2017.

Jared Hawkins, director of informatics at Boston Children’s Innovation and Digital Health Accelerator (IDHA), brings a formidable skill set to his work. With a PhD in Immunology from Tufts University School of Medicine and an MMSc in Biomedical Informatics from Harvard Medical School, his background combines biomedical research (immunology, virology, oncology, genomics) with data science, visualization, computational modeling and software development.

His current work spans an equally diverse range of topics, touching on population and public health, patient experience, decision support and pharmacogenomics. A faculty member in the Computational Health Informatics Program, Hawkins is wired into the digital health ecosystem. He serves as a scientific advisor and co-founder of Raiing Medical (home temperature and fertility tracking) and is the head of engineering and co-founder of Circulation (non-emergency medical transportation via Uber).

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