Tripp Underwood contributed to this post.
Families with peanut-allergic children live in fear that their child will ingest peanuts—even minute amounts—accidentally. Now, a small pilot study published in the Journal of Allergy and Clinical Immunology offers hope.
In the year-long study, immunologist Dale Umetsu, MD, PhD, and colleagues in the Division of Allergy and Immunology at Boston Children’s Hospital were able to get some children to tolerate as many as 20 peanuts at a time. Their protocol combines a powerful anti-allergy medication with a methodical desensitization process.
While it’s not a cure, the protocol may enable children to weather trace amounts of peanuts that might lurk in baked goods or foods “manufactured in a facility that processes peanuts.” Even a small amount of peanut tolerance could be lifesaving. Full story »
In a one-two-three punch, a rapid screen in zebrafish can quickly identify a short list of drug candidates to test in mice and in patient-derived cells.
Scientists have had little success in growing skeletal muscle for patients with muscular dystrophy and other disorders that degrade and weaken muscle. Undertaking experiments in zebrafish, mouse and human cells, researchers have identified a way to do that, creating cells that Leonard Zon, MD
, hopes to see tested in patients in the next several years.
But what really excites Zon, director of the Stem Cell research program at Boston Children’s Hospital, is the power of the chemical screening platform he and his colleagues used. Described last week in the journal Cell, it found a cocktail of three compounds that induced human muscle cells to grow—in just a matter of weeks. Zon believes it could fast-track drug discovery for multiple disorders. Full story »
New research reinforces that inborn vulnerabilities can tip infants toward SIDS.
Epidemiologic studies have shown that infants who die suddenly, unexpectedly and without explanation—what’s referred to as sudden infant death syndrome, or SIDS—are often found sleeping face down with their face in the pillow, or sleeping next to an adult. These are environments that have the potential to cause smothering and asphyxiation. By advising parents to have infants sleep on their backs, in a separate crib or bed, the government’s Safe to Sleep
campaign (formerly known as Back to Sleep) has greatly reduced deaths from SIDS.
Hannah Kinney, MD, a neuropathologist at Boston Children’s Hospital, is clear that this campaign must go forward—it’s saved thousands of lives. But still, she receives calls from parents and grandparents haunted by their infants’ death, feeling at fault and wanting a second opinion.
And in many cases, she has been able to document abnormalities in brainstem circuits that help control breathing, heart rate, blood pressure and temperature control during sleep.
What’s lacking is early detection and treatment. Full story »
In mice, VEGF-A modRNA visibly improved blood supply to heart muscle (right image) as compared with no treatment.
Heart attacks cause the death of billions of the heart’s muscle cells. If these cardiomyocytes could be made to regenerate after an infarct, the heart could potentially be mended and its function restored.
Researchers have struggled to find the right approach to regeneration. Cell transplants have been tried, but the cells don’t engraft well long term and haven’t shown efficacy. Gene therapy to spur regeneration has been tested in animals, but dosage is hard to control and there’s a risk of genes going where they shouldn’t, causing tumors and other problems. Protein drugs have been tried, but they have short half-lives, being degraded or eliminated by the body before they can do much good. They are also hard to target to the heart.
A more recent approach to cardiac regeneration is to stimulate the body itself—and, specifically, progenitor cells— to repair the heart from within. Full story »
Wrapping up the National Pediatric Innovation Summit + Awards on Sept. 27, emcee Bruce Zetter, PhD, who runs a lab in Boston Children’s Vascular Biology program, remarked, “I thought I was going to learn about technology. What I learned about was communication.”
Surgeon, writer and public health researcher Atul Gawande, MD, MPH, laid bare this often overlooked element of medicine in his closing keynote. He eloquently made the point that communication—and more specifically systems—is where innovation is most needed and where it can have the most impact.
“We have emerged from the century of the molecule to the century of the system,” Gawande said.
Right now, these systems are broken, seemingly everywhere. Gawande recounted the sad tale of Duane Smith, a patient who survived a severe car crash that ruptured his spleen, only to lose his fingers, toes, nose and job from an ordinary strep infection. Full story »
There’s been an explosion of scientific research in autism—from mouse models of genetic syndromes involving autism to culturing neurons from stem cells derived from patients’ skin to tracking EEG patterns in infants whose brothers or sisters have autism.
So I expected yesterday’s panel on Piecing Together the Autism Puzzle, part of Boston Children’s National Pediatric Innovation Summit, to be about the science. I changed my seat just before it started, so I could better view the slides.
Instead, the conversation turned to the insurance, public health and social justice aspects of autism. Take, for example, the rising incidence of autism, which the CDC places at 1 in 88 (and 1 in 54 in boys). Panelist Ami Klin, PhD, director of the Marcus Autism Center at Children’s Healthcare of Atlanta, noted that between the CDC’s 2002 and 2008 reports on autism, there was close to a 101 percent increase in autism prevalence in Hispanics and a 96 percent increase in blacks.
Thousands of children didn’t suddenly develop autism in a six-year span; rather, more were diagnosed with autism as awareness of the disease increased. Even so, diagnoses often don’t occur until a child is 3 to 5 year old, and only 2.5 percent of diagnostic assessments of autism are using the field’s best standardized tools. While multiple diagnostic tests are being researched—like EEGs or blood tests looking at gene expression—they’re still experimental. Full story »
In Part 1 last week, Vector took a look at digital health apps, telemedicine, genomics, phenomics and new behavioral diagnostics as transformative trends in pediatrics. This week, we complete our list. These posts will also appear as an article in the fall issue of Children’s Hospitals Today magazine.
6. New pharma research and development (R&D) models
Academic medical centers have always worked with the pharmaceutical industry but never so closely as now. In the old model, industry drove therapeutic development. A company might fund an academic project or supply reagents, but the relationship generally ended with the project (and publication of a paper).
Now, with drug pipelines drying up and R&D costs rising, Big Pharma is under pressure to change. New industry-academia collaborations are forging creative partnerships, altering how both parties do business. The new models are allowing hospital researchers to do what they’ve never done before: take the lead in R&D. Full story »
An early innovation: Specially bred cows graze in front of Boston Children’s Hospital in 1919, providing safe, tuberculosis-free milk for patients.
Clinicians wanting to develop new devices and treatments for children face formidable barriers: regulators’ need to protect the most vulnerable coupled with a lack of commercial interest. But determined innovators do have options, including creative funding sources, says Thomas Krummel, MD
, director of surgical innovation at Stanford Medical School.
“Technology developed specifically for children has been a low priority,” Krummel began at a two-part talk at Boston Children’s Hospital this summer (read our coverage of the other part). “The FDA barriers are incredibly high, and ultimately, investors just demand returns that pediatric markets won’t necessarily deliver.”
As Krummel detailed, the FDA barriers are there for a reason: a past history of ethical abuses in human subjects research. In 1966, physician Henry Beecher, MD, exposed many examples in The New England Journal of Medicine, such as withholding effective treatment for the sake of research, proceeding with a treatment despite recognized hazards, or failing to disclose risk to patients. Institutional Review Boards (IRBs) arose in the mid-1970s to protect research subjects—protections that are especially strict when that research is done in children.
But there’s also a deep-seated reluctance to break with the status quo. Full story »
Since our “trends” posts at the top of the year are among our most viewed, Vector took time out this summer to take an interim snapshot of pediatric medicine’s cutting edge. Here we present, in no particular order, our first five picks. Check back next Friday for Part 2. If you want more, there’s still time to register for our National Pediatric Innovation Summit + Awards (September 26-27). The posts will also appear as an article in the fall issue of Children’s Hospitals Today magazine.
1. Digital health apps 2.0
The electronic revolution in health care continues. According to recent surveys, more than 90 percent of physicians have smartphones and more than 60 percent are using tablet devices like iPads for professional purposes. Dr. Eric Topol and others think these digital tools are the future of medicine.
Mobile apps keep proliferating, adding more and more features: high-quality image capture, voice-to-text capabilities and gaming techniques to motivate adherence, as well as sensors that gather physiologic data, like glucose levels and heart rate. Consumers are tracking and sharing data themselves, saving time in the clinic and helping physicians monitor their symptoms. Through the much-hyped Google Glass, it won’t be long before doctors can seamlessly call up patient data, look up a drug dosage and get decision support during a clinical visit without using a hand-held device.
One limiting factor in this “Wild West” scenario is the FDA’s ability to keep up with digital advances from a regulatory standpoint. Full story »