What we’ve been reading: Week of March 23, 2015

shutterstock_208124314

 

Single-Dose Cures for Malaria, Other Diseases (MIT Technology Review)
Pills that deliver a full course of treatment in one swallow could, or “super pills,” could simplify the treatment of diseases such as malaria and potentially produce cost savings that stretch into the $100 billion a year range, according to Bob Langer, PhD, from the Massachusetts Institute of Technology.

Read Full Story | Leave a Comment

CareAline: A mother’s road to SXSW

The CareAline wrap, modeled by Lochlan Fitzgerald
The CareAline wrap, modeled by Lochlan Fitzgerald. Below, the CareAline sleeve.

Our daughter, Saoirse, was diagnosed with cancer when she was 11 months old. Her care, safety and comfort were our first priorities. When she had a PICC line and later a central line placed to infuse drugs and fluids, we saw a need for a better way to keep these lines safe and secure without using skin-damaging tape and irritating mesh netting. Saoirse was tugging at her lines and trying to pull off the tape, so I handmade a fabric sleeve for her PICC line and a chest wrap for her central line, and she went back to playing and being a kid.

Initially we figured that would be the end of it.

Read Full Story | 1 Comment | Leave a Comment

Six emerging trends in vaccine development

boy receiving vaccine-shutterstock

Vaccines to protect against infectious disease are the single most effective medical product, but developing new ones is a challenging and lengthy process, limiting their use in developing countries where they are most needed. Once a new vaccine is developed, it undergoes animal testing, which is time-consuming and does not necessarily reflect human immunity.

“It can take decades from the start of vaccine development to FDA approval at huge cost,” says Ofer Levy, MD, PhD, a physician and researcher in the Division of Infectious Diseases at Boston Children’s Hospital. “We are working on making the process faster and more affordable.”

A variety of new strategies are emerging to facilitate vaccine development and delivery:

1. Modular approaches to vaccine production

The Multiple Antigen Presenting System (MAPS) is one innovative modular method to more efficiently produce vaccines that provide robust immunity.

Read Full Story | Leave a Comment

New Human Neuron Core to analyze ‘disease in a dish’

Human Neuron CoreLast week was a good week for neuroscience. Boston Children’s Hospital received nearly $2.2 million from the Massachusetts Life Sciences Center (MLSC) to create a Human Neuron Core. The facility will allow researchers at Boston Children’s and beyond to study neurodevelopmental, psychiatric and neurological disorders directly in living, functioning neurons made from patients with these disorders.

“Nobody’s tried to make human neurons available in a core facility like this before,” says Robin Kleiman, PhD, Director of Preclinical Research for Boston Children’s Translational Neuroscience Center (TNC), who will oversee the Core along with neurologist and TNC director Mustafa Sahin, MD, PhD, and Clifford Woolf, PhD, of Boston Children’s F.M. Kirby Neurobiology Center. “Neurons are really complicated, and there are many different subtypes. Coming up with standard operating procedures for making each type of neuron reproducibly is labor-intensive and expensive.”

Patient-derived neurons are ideal for modeling disease and for preclinical screening of potential drug candidates, including existing, FDA-approved drugs. Created from induced pluripotent stem cells (iPSCs) made from a small skin sample, the lab-created human neurons capture disease physiology at the cellular level in a way that neurons from rats or mice cannot.

Read Full Story | Leave a Comment

What we’ve been reading: Week of March 16, 2015

 

(government_press_office/Flickr)
(government_press_office/Flickr)

Scientists Call for a Summit on Gene-Edited Babies (MIT Technology Review)

Tools like CRISPR could give us the power to alter humanity’s genetic future. A group of senior American scientists and ethicists have called for a moratorium any attempts to create genetically engineered children using these technologies until there can be a robust debate.

Meet the healthcare company that won Mark Cuban’s heart at SXSW (MedCity News)

CareaLine, founded by the parents of a young girl who died of cancer, won over audience members’ hearts and investors’ wallets during SXSW 2015’s Impact Pediatrics competition.

Read Full Story | Leave a Comment

SXSW Interactive 2015: Our future selves, a maturing health tech industry and why failing is productive

SXSW Impact Pediatric HealthJudy Wang, MS, is a program manager in the Telehealth Program at Boston Children’s Hospital.

In 2012, when I attended the South by Southwest (SXSW) Interactive conference for the first time, health tech was still an emerging field. It was the first year the world’s leading conference for emerging technology and digital creativity made any effort to include health tech programming, and the first time its Accelerator pitch event included a category for health tech startups.

Only three years later, SXSW Interactive (March 13­–17, 2015) has grown to include almost 50 events related to health and medical technologies. Martine Rothblatt, CEO of the biotech company United Therapeutics, gave a keynote titled “AI, Immortality and the Future of Selves” that was both inspiring and provocative. She spoke to a world in which our 24/7 selves are increasingly being captured digitally. Audience questions captured by Twitter pondered the ethical implications of what Rothblatt called “mind clones”: future mechanical beings digitally programmed with our mannerisms, habits and memories.

This year also featured the Impact Pediatric Health pitch competition, captured in this Storify.

Read Full Story | 1 Comment | Leave a Comment

Yes, poor vaccination rates are fueling the 2015 measles outbreak

CDC measles outbreak map vaccination Disneyland
(CDC)

There’s been a lot of speculation about whether low vaccination rates are feeding the 2015 U.S. measles outbreak, which as I write this stands at 145 cases across seven states. Well, we can stop speculating, because the numbers are in, and measles is taking advantage of pockets of inadequately vaccinated people.

That’s the stark, unequivocal message from a study by epidemiologists at Boston Children’s Hospital, published this week in the journal JAMA Pediatrics.

Read Full Story | Leave a Comment

The emerging genetic mosaic of lymphatic and vascular malformations

somatic mosaic mutations vascular anomalies vascular malformations CLOVES Klippel-Trenaunay KTS fibroadipose FAVA lymphatic malformation

Our genes can mutate at any point in our lives. In rare cases, a mutation randomly occurs in a single cell of an embryo and gets carried forward only in the descendants of that particular cell, leaving its mark in some tissues, but not in others. This pattern of mutation, called somatic mosaicsm, can have complicated consequences down the road.

Take CLOVES, a rare syndrome combining vascular, skin, spinal and bone or joint abnormalities described by Ahmad Alomari, MD, co-director of Boston Children’s Hospital Vascular Anomalies Center (VAC). Four years ago, a research team including Alomari and Matthew Warman, MD, discovered that the growths in CLOVES patients had mutations in a growth-regulating gene called PIK3CA. Those mutations, they found, were spread through the affected tissues in a somatic mosaic pattern.

Now it turns out that CLOVES is not alone. In a recent paper in the Journal of Pediatrics, VAC researchers led by Warman proved that three other rare lymphatic and vascular anomalies and overgrowth syndromes often share the same somatic mosaic PIK3CA mutations: Klippel-Trenaunay syndrome (KTS), fibroadipose vascular anomaly (FAVA) and isolated lymphatic malformations.

Read Full Story | Leave a Comment

Embracing the future of pediatric quality measurement

Pediatric quality of care researchWhile studying quality of care in the 1990s, Mark Schuster, MD, PhD found that few studies on pediatric quality had been conducted. The typical explanation that he was given was that the federal government wasn’t funding research into quality measures because children on Medicaid don’t drive federal health-care costs nearly as much as adults on Medicaid and Medicare do.

But Schuster, chief of General Pediatrics at Boston Children’s Hospital and William Berenberg Professor of Pediatrics at Harvard Medical School, believes there have been other challenges in measuring care quality in children. In an acceptance speech upon receiving the 2014 Douglas K. Richardson Award for Perinatal and Pediatric Healthcare Research, published today in the journal Pediatrics (PDF), Schuster points to factors including the relative rarity of many pediatric conditions and that many of the benefits of excellent pediatric care are not observed until adulthood.

Read Full Story | Leave a Comment

How Skype and FaceTime inspired remote care for home-ventilated patients

Casavant telemedicine home ventilationFrom a series on researchers and innovators at Boston Children’s Hospital. At left, David Casavant demos TeleCAPE at a Boston Children’s Hospital Innovators Showcase.

It is said that necessity is the mother of invention, so when David Casavant, MD, observed his teenagers routinely using FaceTime and Skype to connect with friends, he had a lightbulb moment. Could videoconferencing help him support his patients—children and young adults who require mechanical ventilation in their homes?

“It just seemed obvious,” says Casavant, a physician in the Boston Children’s Hospital’s CAPE (Critical Care, Anesthesia and Perioperative Extension & Home Ventilation) program, part of the Division of Critical Care Medicine. “In my work we are always weighing the risk versus the benefit to the patient. It’s easy for ambulatory patients to swing by their primary care office, get a prescription or go for an x-ray, but that’s not the case for patients who have to have their oxygen, their suction or their ventilator. If you don’t have to put them on the road you are better off not to.”

Read Full Story | Leave a Comment