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cell fate map Boston subway

Credit: Samantha Morris, PhD, Boston Children's Hospital

If you’ve lost your way on the Boston subway, you need only consult a map to find the best route to your destination. Now stem cell engineers have a similar map to guide the making of cells and tissues for disease modeling, drug testing and regenerative medicine. It’s a computer algorithm known as CellNet.

As in this map on the cover of Cell, a cell has many possible destinations or “fates,” and can arrive at them through three main stem cell engineering methods:

reprogramming (dialing a specialized cell, such as a skin cell, back to a stem-like state with full tissue-making potential)
differentiation (pushing a stem cell to become a particular cell type, such as a blood cell)
direct conversion (changing one kind of specialized cell to another kind)

Freely available on the Internet, CellNet provides clues to which methods of cellular engineering are most effective—and acts as a much-needed quality control tool. Full story »

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PTSD risk in adolescents after Boston Marathon bombingsDaniel Busso, MSc, is a doctoral student at the Harvard Graduate School of Education and a researcher in the Sheridan Laboratory at Boston Children’s Hospital.

More than 60 percent of teenagers have experienced a traumatic event in their lifetime, but only a minority will develop post-traumatic stress disorder (PTSD). For both researchers and clinicians, this raises an important question: Why are some youth at greater risk for mental health problems after trauma? As our lab reports in two recent studies, conducted after the 2013 Boston Marathon bombings, the answer may lie in our neurobiology.

PTSD, which includes intrusive memories, increased anxiety and difficulty concentrating or sleeping, has been linked to a variety of psychosocial and biological risk factors, such as prior experiences of trauma or a history of mental health problems. Other studies suggest that disruptions to the body’s stress response system, or in patterns of brain activity when responding to threat, may predispose people to the disorder.

However, a common problem in this research is that biological and mental health data are collected only once, usually long after the traumatic event itself, Full story »

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What's drawing lung cancer cells to migrate? (Juan Gaertner/Shutterstock)

What's drawing lung cancer cells to migrate? (Juan Gaertner/Shutterstock)

Ninety percent of lung cancer deaths are caused by the tumor’s spread—or metastasis—to other organs. Researchers have now discovered an approach to blocking metastasis in the most common type of lung cancer, adenocarcinoma, that potentially could be added to chemotherapy treatments aimed at shrinking the primary tumor.

Kerstin Sinkevicius, PhD, a research fellow at Boston Children’s Hospital, started with this question: Is there anything in a lung tumor’s environment that makes it metastasize? She sampled tissue from human lymph nodes—the first place cancers typically spread to—to see if the cells there were secreting anything that might lure cancer cells to migrate.

One chemical stood out: a growth factor called brain-derived neurotrophic factor, or BDNF. Secreted near maturing neurons, BDNF is best known for its role in stimulating the developing nervous system. Full story »

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(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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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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