Microbiome therapeutics: 6 takeaways from a MassBio panel

microbiome therapeuticsSeeing the surprising success of “poop pills” in gastrointestinal C. difficile infection, pharma companies and startups are embracing the microbiome as a new therapeutic target for an astonishing range of maladies. To learn what pioneering companies in the space are thinking about the hope and the hype, Vector recently attended a panel on microbiome therapeutics at the MassBio Annual Meeting.

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The face of telehealth: Serving children in health care “deserts”

pediatric telehealthAt least 15 million children reside in Health Professional Shortage Areas (HPSAs) that average fewer than one health professional for every 3,500 people. In these health care deserts, time and transportation barriers prevent even children with health insurance have trouble getting timely care, particularly specialty care. Children in poor, rural areas are most at risk.

So health problems fester and get worse — and more expensive when finally addressed.

Telehealth can solve many of these problems. Through remote video/voice/data connections, dermatologists can view images of rashes and moles sent by primary care providers; cardiologists can patch into local emergency rooms and listen to heart sounds and read EKG tracings; critical care physicians and neonatologists can see and hear newborns in distress, listen to lung sounds, read their vital signs and view images. They can advise local clinicians and guide them through next steps.

However, pediatric telehealth hasn’t been adopted as widely as it could be. A white paper presented by the Children’s Health Fund at a Congressional briefing last week enumerated the obstacles:

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Probing the link between autism, GI disorders and the microbiome

autism microbiome
(Dubova/Shutterstock)

Sonia A. Ballal is an attending physician in the Division of Gastroenterology, Hepatology and Nutrition at Boston Children’s Hospital.

Eleven-year-old Lyle has autism and doesn’t speak, but his mother is used to reading his nonverbal cues. He prefers a routine, but has always been a generally cheerful child who enjoys school and playing with his little sister.

Several weeks before I met Lyle (not his real name), his mother observed a dramatic shift. He was agitated, at times hitting his head against the wall, not receiving his typical sunny reports from school.

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Reimagining connected health through home hubs

home health hubs

Through smart home hubs and the growing Internet of Things, people can now control lights, thermostats and other appliances and get information and entertainment with their always-connected digital devices. Consumers have widely adopted home automation products like Nest from Google and ecosystems like Apple’s HomeKit and Amazon’s Alexa.

But home hubs also have the potential to achieve the promise of connected health — access to health care services anywhere and anytime.

Home hubs can deliver enormous value as a means of health care delivery — not just helping casual consumers become familiar with their health and take preventive measures, but also helping manage complex care for patients with chronic illness and supporting timely decision making by clinical teams. Everybody involved with a person’s care can be plugged in, enabling coordination across providers and caregivers in a way that’s increasingly intuitive and meaningful.

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Can we supercharge vaccines? Added compound boosts T-cell production

supercharged vaccines oxPAPCBridging our innate and adaptive immune systems, dendritic cells are sentinels that circulate in the body searching out microbes and activating T-cells to destroy the invaders. They do this by presenting bits of the microbes on their surface—explaining why they’re often called antigen-presenting cells.

Reporting in Science this week, researchers describe a way to push dendritic cells into a “hyperactive” state, supercharging their ability to rally T-cells.

The key player, a fatty chemical called oxPAPC, is naturally found in damaged tissues and atherosclerotic plaques. It selectively targets dendritic cells and could, the researchers believe, enhance people’s immunity to a wide range of infections.

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Forty years waiting for a cure: ALD gene therapy trial shows early promise

Ethan and me, June 1977
Ethan and me, June 1977

A small piece of notepaper, folded twice, sits tucked in a slot of the secretary desk in the living room. Every so often, I pull it out, read it, then reread.

Addressed to my mom, the paper has a question and two boxes, one “yes” and one “no,” written with the careful precision of a 7-year-old.

I am sad of Ethan. You too?

A check marks the box.

Yes. Yes, I am sad too.

Learning about adrenoleukodystrophy

My brother Ethan Williams was 9 years old in the fall of 1976, when he began to lose his sight. For my parents, that winter brought an endless round of doctor visits, therapists and lab tests.

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Give childhood cancer a place on the cancer ‘moonshot’

cancer moonshot

Cancer remains the leading disease-related cause of death in children in the United States. Yet, when it comes to cancer research funding and drug development, pediatric cancer is often left behind, writes David A. Williams, MD, president of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, in Boston Globe Media’s STAT.

Although there are more than 150 types of childhood cancer, pediatric cancer receives only a small fraction of NIH and National Cancer Institute funding, Williams writes. Yet, he points out, just as breakthroughs in adult cancer research can help children, breakthroughs in pediatric cancer can also benefit adults.

Williams and other members of the Coalition for Pediatric Medical Research recently met with the staff of Vice President Joseph Biden, leader of the federal government’s cancer moonshot. Their message? Make sure that pediatric cancer is represented on the moonshot.

Read Williams’s STAT piece.

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From NICU dad to citizen scientist: Creating a smart pulse oximeter

Morris Family
Jon and Sarah Morris with 7-year-old twins Drew and Emma

When Sarah and Jon Morris’ twins were born nine weeks early, they embarked on a journey largely dictated by their children’s medical needs. While son Drew was thriving, daughter Emma was severely compromised and was transferred to Boston Children’s Hospital’s Neonatal Intensive Care Unit (NICU). “We felt powerless,” remembers Jon. “Every time we thought we had made progress, we had a setback. It’s always two steps forward, one step back in the NICU. That backwards step always hit the hardest.”

After 296 days at Boston Children’s, Emma went home tethered to breathing and feeding tubes. The Morrises had a pulse oximeter at home to regularly test Emma’s blood oxygen level.

There were frustrating limitations to Emma’s oximeter:

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The science of marathon running

Running the Boston Marathon takes months of training, and it can take a toll on elite marathoners and novice athletes alike. Vector checked in with some of the experts in the Boston Children’s Hospital’s Sports Medicine Division to learn more about how science can aid marathon runners, prevent injury and illness and facilitate recovery. Hover over the icons below for some basics.

Chocolate milk: A no-brainer for maximizing recovery

The most rapid way to maximize recovery after an endurance run is to consume carbohydrates in a 4:1 ratio within 30 to 60 minutes of completing the marathon. The enzyme glycogen synthase is most active within this time frame; it synthesizes glycogen — the body’s main source of energy during activity — from glucose in the body. Chocolate milk has become a favorite of runners and athletes since it provides the perfect 4:1 ratio of carbohydrate to protein. —Laura Moretti, MS, RD, clinical nutrition specialist

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Arsenic and neural tube defects: Lessons from Bangladesh?

arsenic neural tube defects

Spina bifida and other neural tube defects have become fairly rare in the United States, thanks in part to folic acid added to foods and campaigns to get childbearing women to take folic acid. But in Bangladesh, spina bifida is a common occurrence on maternity wards; in fact, it is considered to be epidemic.

“No surveillance is done, so it’s not clear how many cases there are,” says Maitreyi Mazumdar, MD, MPH, a neurologist at Boston Children’s Hospital who conducts environmental health research. “Children may die in delivery, or they may die before seeing a surgeon.”

Although folic acid supplementation isn’t widespread in Bangladesh, Mazumdar thinks there is another factor in play: the country’s ongoing epidemic of arsenic poisoning.

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