(Pyh2/Wikimedia Commons)

(Pyh2/Wikimedia Commons)

Geoffrey Horwitz, PhD, is a business development associate in the Technology and Innovation Development Office (TIDO) at Boston Children’s Hospital. Follow him on Twitter @GeoffHorwitz

At the recent 2014 Biotechnology Industry Organization (BIO) International Convention, the message was clear: Digital health is the new blockbuster. For the first time ever, BIO spotlighted digital health, with a specific focus on how digital health is influencing the pharmaceutical and biotech industries. Also featured was a digital health zone where companies and other exhibitors from all over the world could demo their products and services to thousands of attendees.

In pharmaceutical lingo, a blockbuster is a drug that generates revenues of at least $1 billion. Digital health certainly fits this definition. By 2018, reports suggest that revenues will exceed $6 billion for wearable wireless devices alone. A recent McKinsey study found that 75 percent of consumers surveyed, of various ages and located throughout the world, would like to use digital health devices. Full story »

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Mast cell food allergy

Mast cells don’t simply cause acute allergic reactions. They also turn off immune tolerance. But that could change. (Bruce Blaus/Wikimedia Commons)

Hans Oettgen, MD, PhD, is Associate Chief of the Division of Allergy and Immunology at Boston Children’s Hospital.  He leads a research group investigating mechanisms of allergic diseases.

Not long ago I received a wonderful email from “Sam,” an 18-year-old young man with peanut allergy. He was participating in a clinical trial of oral immunotherapy (OIT) being carried out by colleagues here at Boston Children’s Hospital.

In OIT, patients receive initially minute doses of the food to which they are allergic. Then, over many weeks, they ingest increasing amounts, under close medical monitoring at the hospital.

OIT’s goal is to get patients to tolerate previously allergenic foods by inducing their bodies to produce Treg cells, or regulatory T cells. These are the master controllers of our immune responses, and their actions include suppressing allergic responses to foods. Food ingestion, as in OIT, will eventually induce food-specific Treg cells, but it can be a long and cumbersome process. For Sam, ingesting escalating doses of peanuts proved difficult: His email described frequent reactions ranging from stomachaches and itchiness to difficulty breathing. Full story »

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401k drug financing

A new proposal suggests spreading drug development risk among many small investors.

Ed Anderson, CCRP, is a clinical research specialist for the Clinical Research Center’s Development and Operations Core at Boston Children’s Hospital.

There’s no way around it. Obtaining approval to market a new drug is lengthy, complex, costly and fraught with uncertainty and risk. Financial engineers at MIT propose a strategy to minimize that risk—one that deserves a close look.

In the last 10 years, the aggregate cost of pharmaceutical research and development has doubled, but the number of approved products has remained the same. To compound the problem, a $1.6 billion reduction in NIH funding, caused by the 2013 sequester, has stalled research projects at more than 2,500 research institutions supported by grants. Pressure from investors and stakeholders is pushing pharmaceutical companies to focus on projects with a greater chance of financial success.

As a result, translational studies—those that bridge the gap between basic research and clinical trials—continue to be neglected and account for less than 12 percent of total research funding. Full story »

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a wave of wearable devicesProjections that the global mobile health market will boom to nearly $50 billion have ignited interest among innovators. A pair of physician innovators from Boston Children’s Hospital peg wearables as the technology to watch and offer a sneak peek at what adoption might mean, while others ask about the pediatric market for wearables and point to a few potential stumbling blocks. Read on for their views. Full story »

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Judy Wang, MS, is a program manager in the Telehealth Program at Boston Children’s Hospital. She is currently serving on the Mayor’s ONEin3 Council, which works on projects dedicated to maximizing the positive impact that young people have on the City of Boston.

young health tech entrepreneurs

(ITU/Rowan Farrell creativecommons.org/licenses/by/2.0/legalcode)

If you Google the term “millennials,” you’ll see that Google automatically fills in such search terms as “millennials lazy,” “millennials spoiled,” “millennials trophy kids” and “millennials entitled.” Ouch.

As part of the Mayor’s ONEin3 Council and a Founding Hacker for MIT’s H@cking Medicine, I could not disagree more with this assessment of my generation. I’ve observed young people increasingly drawn to civically minded work with public impact—including work in health tech. Full story »

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Thomson Reuters most highly cited scientists

It’s not every day you wake up and find out you’ve been named one of the most influential people in science. But that day recently came to seven Boston Children’s Hospital researchers, when Thomson Reuters named them as some of the most highly cited scientists in the world.

Every few years, the information broker and news outlet combs through its Web of Science and InCites systems—which track the scientific literature—to see whose work other researchers consistently refer to in their papers. It then creates two categories: highly cited papers (those that “rank in the top 1 percent by citations for their field and year of publication”) and hot papers (ranking “in the top 0.1 percent by citations for their field”). Full story »

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Emmie was lucky enough to be diagnosed before age 3, but many families face a much longer journey.

Emmie Mendes was lucky enough to be diagnosed before age 3, but many families face a much longer journey.

At first, Corrie and Adam Mendes thought their daughter Emmie had an inner ear problem. She was late with several early milestones, including walking, and when she did walk, she often lost her balance. The family pediatrician sent them to a neurologist, who ordered a brain MRI and diagnosed her with pachygyria, a rare condition in which the brain is smoother than normal, lacking its usual number of folds.

Additionally, Emmie’s ventricles, the fluid-filled cushions around the brain, looked enlarged, so the neurologist recommended brain surgery to install a shunt to drain off fluid. He advised Corrie and Adam that Emmie’s life expectancy would be greatly reduced.

As Corrie recounts on her blog, Emmie’s Story, she went online and came across the research laboratory of Christopher Walsh, MD, PhD, at Boston Children’s Hospital. The lab does research on brain malformations and has an affiliated Brain Development and Genetics Clinic that can provide medical care.

After Walsh’s team reviewed Emmie’s MRI scan, genetic counselor Brenda Barry invited the family up from Florida. Full story »

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At TEDx Longwood this spring, Leonard Zon, MD, founder and director of the Stem Cell Program at Boston Children’s Hospital, took the stage. In his enthusiastic yet humble style, he took the audience on a journey that included time-lapse video of zebrafish embryos developing, a riff by Jay Leno and a comparison of stem cell “engraftment” to a college kid coming home after finals: “You sleep for three days, and on day 4, you wake up and you’re in your own bed.” Three takeaways:

1)   Stem cells made from our own skin cells can help find new therapeutics. With the right handling, they themselves can be therapeutics, producing healthy muscle, insulin-secreting cells, pretty much anything we need. (So far, this has just been done in mice.)

2)   Zebrafish, especially when they’re see-through, can teach us how stem cells work and can be used for mass screening of potential drugs. The Zon Lab boasts 300,000 of these aquarium fish, and can mount robust “clinical trials” with 100 fish per group.

3)   Drugs discovered via zebrafish are in human clinical trials right now: A drug to enhance cord blood transplants for leukemia or lymphoma, and an anti-melanoma drug originally used to treat arthritis.

Zon, who co-founded the biopharm company Fate Therapeutics, will be part of a judging panel of clinicians and venture capitalists for the Innovation Tank at Boston Children’s Global Pediatric Innovation Summit + Awards (Oct. 30-31). Don’t miss it!

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Clinical excellence is the foundation of patient care. But at a recent TEDx Longwood event, Elaine C. Meyer, PhD, RN, co-founder and director of the Institute for Professionalism and Ethical Practice at Boston Children’s Hospital and an Associate Professor of Psychology at Harvard Medical School, offered insight on the other half of the health care equation: the human connection and the power of conversation.

Meyer’s moving presentation makes clear how communication—listening and sharing words of comfort—profoundly impacts patient experiences, as does its absence. Through heartfelt stories, including her own experience as a patient, her talk empowers physicians, nurses, social workers, psychologists and other medical staff to “be present” and communicate with patients and families compassionately.

“Dig deep, find your inspiration to have conversations,” Meyer says, because patients remember the words spoken to them and how those words made them feel.

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ADHD long term drug safety rare adverse events FDA approval Kenneth Mandl Florence BourgeoisYou’d think drugs meant to be taken by children for years would be studied in children for a long time to measure their long-term safety.

You’d think drugs for a condition affecting millions of children would be tested in many, many children to catch any rare side effects.

You’d think all this would happen before the Food and Drug Administration, an agency known for its strict criteria, approved them for marketing.

But if a new PLoS ONE paper by Boston Children’s Hospital’s Florence Bourgeois, MD, MPH, and Kenneth Mandl, MD, MPH, is any indication, you’d be wrong.

In it, the pair reports that the FDA approved 20 attention deficit hyperactivity disorder (ADHD) drugs over the last 60 years without what would be considered sufficient long-term safety and rare adverse event data.

Their findings, they say, point to larger issues in how the FDA’s approval process addresses the long-term safety of drugs intended for chronic use in children. Full story »

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