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Giving thanks to scientists, innovators, parents and more

Thanksgiving table-shutterstock_326063243It’s been an exciting year for pediatric health care. As Thanksgiving draws near, Vector is taking a pause to acknowledge the inspiring people and ideas that are helping set the table for a better future.

What are we thankful for?

The growing cadre of citizen scientists — passionate parents pushing for answers for their kids, helping to move rare disease research forward through their own investigations and initiatives. They’re keeping academic researchers honest and on top of their game, and, in many cases, helping to fund them.

The growing inclination among clinicians to say, “the way things are isn’t good enough,” and then push the boundaries of what’s possible to improve sick children’s lives.

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An iPhone and a bucket help diagnose vestibular problems in dizzy children

dizziness vestibular bucket testDizziness is fairly common in children, but it can be very hard to diagnose the cause. Any number of conditions can produce dizziness, and children are a special challenge since they often can’t describe what they’re feeling.

“One of the toughest things to figure out is, is it a problem with the vestibular system, or is it part of something else, a heart problem or an eye problem?” says Jacob Brodsky, MD, director of the Balance and Vestibular Program at Boston Children’s Hospital. “Then, the next challenging part is determining whether it is an inner ear problem or a central vestibular disorder — a problem with the brain.”

A definitive answer often requires a battery of tests that few providers outside Boston Children’s can perform in children, as they require sophisticated and expensive equipment. But with an ordinary bucket, an iPhone, an $18 app and some Velcro, Brodsky can quickly get a good indication of whether a child has a vestibular problem—and specifically an inner ear problem.

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8 winning innovations at Hacking Pediatrics

general hackersHacking Pediatrics, now in its third year, continues to experiment with its format. 2015’s “Mashup” had a greater focus on partnerships, curation and delivering value to innovators at Boston Children’s Hospital. The brunt of the idea pitching and team formation took place in advance, allowing the event, on November 14, to be collapsed into one day.

The Hacking Pediatrics team (Kate Donovan, Mike Docktor, Meg McCabe, Cassandra Bannos and Leila Amerling) brokered collaborations with a dozen industry partners such as Microsoft, Cerner, Box, CVS Health and Boston Scientific. Over the course of a hectic 12-hour day, they worked with 17 teams of Boston Children’s innovators and experts from partner organizations who presented their final ideas to a panel of judges.

In another change for 2015, the Hacking Pediatrics team issued nine awards — but no immediate prizes. This was meant to incentivize teams to continue to work and meet milestones to earn real rewards, like a $10,000 design prize offered up by design firm Mad*Pow.

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An unusual dog, a new approach to muscular dystrophy: Stimulating a protective gene

Vieira with Ringo
Vieira with Ringo

Ringo was a golden retriever that defied the odds. Despite having the gene mutation for Duchenne muscular dystrophy (DMD), he remained healthy. And he’s provided a new lead for boosting muscle strength in DMD, one of the most common forms of muscular dystrophy. Unlike other dogs with the dystrophin mutation, who are weak and typically die by 2 years of age, Ringo was able to walk and run normally and lived to the age of 11, within the normal range for golden retrievers.

What made Ringo so resilient?

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The future of pediatric precision medicine: opportunities, barriers

“Precision medicine” looks to be heading down the same path as “big data” and “innovation”: The term is becoming so widely used that it threatens to detract from the real impact it is already having in patients’ lives.

But for children, who are still developing and have the most to gain, precision medicine is more than a bumper sticker. On the micro scale, early genetic testing—perhaps routinely, someday, in newborns—can help guide medical care, targeted therapies and preventive strategies based on a child’s genetic makeup. On a macro scale, big data from the larger population becomes a predictive tool, guiding medical decisions that could be life-altering in a still-malleable child.

“If you can make an early diagnosis, you can amplify the effects of what you do, rather than try to change the highways once they’re built,” said Wendy Chung, MD, PhD, of Columbia University Medical Center during a panel discussion last week at Boston Children’s Hospital’s Global Pediatric Innovation Summit + Awards (#PedInno15).

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Storify: The Global Pediatric Innovation Summit in tweets

Global Pediatric Innovation Summit AwardsWe’ve been covering the science and innovation news out of the Global Pediatric Innovation Summit + Awards all week. We close out with the event’s best one-liners and the attendees’ collective take on the talks, panels and announcements. Props go to @ldtimmerman and @cgcarlson for being the event’s top tweeters.

Read on for Day 1 and Day 2 highlights. For more, watch the video highlights and read the day-to-day blog coverage.

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Stuart Orkin honored for his lifetime research on blood development

Presenter Bill Evans, IBM Watson Health and Stuart H. Orkin, MD

When colleagues describe Stuart H. Orkin, MD, associate chief of hematology/oncology at Boston Children’s Hospital and chair of pediatric oncology at Dana-Farber Cancer Institute, the words “immeasurable,” “vanguard” and “mentor” quickly roll off the tongue.

In honor of his 35-year career and commitment to blood cell research, Boston Children’s Hospital presented Orkin with the 2015 Lifetime Impact Award, during Boston Children’s Global Pediatric Innovation Summit held this week. The award recognizes a clinician and/or researcher who has significantly impacted pediatric care through practice-changing innovations or discoveries and made extraordinary and sustained leadership contributions in health care throughout his or her career.

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Hematologist Vijay Sankaran receives Boston Children’s Hospital Rising Star Award


Before an audience of several hundred luncheon attendees, physician-scientist Vijay G. Sankaran, MD, PhD, received Boston Children’s Hospital’s 2015 Rising Star Award — recognizing the outstanding achievements of an up-and-coming innovator under the age of 45 in pediatric health care.

Sankaran, a board-certified pediatric hematologist/oncologist with Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, conducts innovative research on red blood cell disorders such as Diamond-Blackfan anemia, sickle cell disease and thalassemia.

The Rising Star Award and companion Lifetime Impact Award ceremony were held at the hospital’s Global Pediatric Innovation Summit + Awards on November 10.

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Two big wins for rare disease

Two new developments offer glimmers of hope to patients with rare, hard-to-diagnose conditions—validation of the power of crowd sourcing and the prospect of bringing cognitive computing to rare disease diagnosis. Both developments were announced at the Boston Children’s Hospital Global Pediatric Innovation Summit + Awards (#PedInno15).

The crowd-sourcing challenge, CLARITY Undiagnosed, yesterday announced the findings of 21 teams that competed from seven countries. The winning team, Nationwide Children’s Hospital (Columbus, OH), was awarded $25,000. Invitae Corporation (San Francisco) and Wuxi NextCODE Genomics (Cambridge, MA) were named runners-up.

Each team received DNA sequences and clinical data from five families whose illnesses had eluded many prior diagnostic attempts—in some cases, even prior genomic sequencing.

“These were the toughest of the tough,” says Alan Beggs, PhD, co-organizer of CLARITY Undiagnosed and director of the Manton Center for Orphan Disease Research at Boston Children’s.

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Rock Health, Boston Children’s Hospital join to speed pediatric digital health startups

Bridget Duffy MDBoston Children’s Hospital and Rock Health, a venture fund dedicated to digital health, announced a strategic partnership aimed at accelerating pediatric health technologies during the hospital’s 2015 Global Pediatric Innovation Summit + Awards. Bridget Duffy, MD, is chair of Rock Health, chief Medical Officer of Vocera and co-founder of the Experience Innovation Network.

As a physician, entrepreneur, and especially as a parent, I am excited and inspired by a partnership between Rock Health and Boston Children’s Hospital to identify promising companies and cultivate emerging technologies focused on transforming pediatric care. Together, Rock Health and Boston Children’s Hospital will seed bigger ideas, accelerate adoption of new solutions, and massively change the way medicine is delivered to every child.

A global leader in pediatric clinical care, Boston Children’s Hospital will lend its expertise to support Rock Health’s portfolio and help shape the next generation of innovations. While Rock Health already has a proven track record with companies in this space, such as Kurbo Health, Cellscope and Kinsights, this close relationship with Boston Children’s will greatly accelerate the design and evaluation of new solutions, improving the health care experience for children and their caregivers.

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