Stories about: Innovation

Light-activated nanoparticles could avoid painful eye injections for ‘wet’ macular degeneration

Could intravitreal injections become a thing of the past?
(PHOTO: ZKALILA1998 / WIKIMEDIA COMMONS)

There are two standard treatments for “wet” age-related macular degeneration (AMD), in which abnormal, leaky blood vessels in the back of the eye lead to fluid buildup and vision loss. The first, injection of medication directly into the eye, can be painful and can cause inflammation, infection and detachment of the retina. The second, ablation therapy, uses lasers to destroy the leaky blood vessels. It, too, is unpleasant to undergo, and the lasers can also destroy surrounding healthy tissue, causing further vision loss.

In today’s Nature Communications, the lab of Daniel Kohane, MD, PhD, provides proof-of-concept of a more tolerable alternative: tiny, drug-carrying nanoparticles that can be injected intravenously, but deliver medication only to the eye.

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Two new options for treating esophageal damage in children

Rusty Jennings, MD, and Michael Manfredi, MD (CREDIT: MICHAEL GODERRE)

A child’s esophagus can become damaged through physical trauma or ingestion of toxic chemicals or foreign objects — such as oven and drain cleaners, lye, laundry and dishwasher detergents and batteries. Depending on the substance and the amount ingested, children can develop esophageal strictures (scar tissue that narrows the esophagus) or esophageal perforations (holes in the esophagus). These problems can also be complications of surgery for esophageal atresia, in which a baby is born without part of the esophagus.

Children with esophageal perforations have traditionally been treated with long courses of antibiotics and not eating by mouth. More recently, perforations have been treated with stents, and strictures with a combination of dilation and stenting. But stenting, while it can be effective, requires up to eight weeks of therapy and can have complications such as pain, retching and local pressure necrosis, a type of ulcer that may worsen perforation. Such concerns have led researchers to investigate alternative treatments for perforation and strictures.

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Can we mass-produce platelets in the lab?

Lab-grown platelets could someday be given to patients
Activated platelets (IMAGE: ADOBE STOCK)

Most of us have somewhere around a trillion tiny platelets zooming around our bloodstreams. Joseph Italiano, PhD, of Boston Children’s Hospital’s Vascular Biology Program, calls them the “Swiss Army knives of the blood.” In addition to their key role in clotting, platelets are important in immunity, wound healing, chemical delivery, blood vessel development and more.

At healthcare facilities, platelets are in constant demand for patients with blood diseases, or those receiving radiation or chemotherapy for cancer. But unlike other blood products, platelets can’t be stored for more than a few days. If there’s a snowstorm or other emergency preventing donors from giving platelets, a hospital can easily run out. So researchers have been trying to make platelets in a lab setting.

Two teams at Boston Children’s Hospital are tackling the problem in slightly different ways.

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Gene panel helps investigate sudden unexpected death in children

Thousands of cases of sudden unexplaied death (SUDP) occur each year.
(PHOTO: ADOBESTOCK)

Almost 10 percent of pediatric deaths occur suddenly and without explanation. In this terrible situation, the first question many parents have is “Why?” For most, answers never come.

Childhood deaths that cannot be explained by traditional autopsy and death-scene investigation are referred to as sudden unexplained deaths in pediatrics (SUDP). In children, these deaths are more common than those from either cardiac disease or cancer and typically occur in infancy or early childhood.

Robert’s Program at Boston Children’s Hospital, co-founded by Richard Goldstein, MD, and Hannah Kinney, MD, is adding a genetic approach to the search for answers.

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New technique images whole brains with incredible resolution

Overview of brain structures captured by new imaging technique
Combined expansion microscopy and lattice light-sheet microscopy allows for highly detailed images to be taken over large sections of the brain. (IMAGES COURTESY SRIGOKUL UPADHYAYULA, RUIXUAN GAO, AND SHOH ASANO)

Decades ago, discoveries about the brain’s intricate anatomy were made with careful dissection and drawings. Today, they’re made with super-resolution imaging and massive computing power capable of handling hundreds of terabytes of data.

In this week’s Science, a team out of the Massachusetts Institute of Technology (MIT), the Janelia Research Campus of the Howard Hughes Medical Institute (HHMI), Harvard Medical School (HMS) and Boston Children’s Hospital, describes a technique capable of imaging whole brains at exquisitely high resolution, allowing scientists to distinguish tiny sub-cellular structures.

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Using multiple data streams and artificial intelligence to ‘nowcast’ local flu outbreaks

(PHOTO: ADOBESTOCK)

Because influenza is so contagious, it’s been challenging to track and forecast flu activity in real time as people move about and travel. While the CDC continuously monitors patient visits for flu-like illness in the U.S., its information can lag by up to two weeks. A new study led by the Computational Health Informatics Program (CHIP) at Boston Children’s Hospital combined multiple approaches, providing what appear to be the most accurate local flu predictions to date.

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Overriding resistance to epigenetic inhibitors in neuroblastoma: Targeting PI3K

(IMAGE COURTESY NATIONAL CANCER INSTITUTE)

Children’s cancers pose unique challenges. They’re not caused by the same kinds of genetic mutations that cause adult cancers, and only a minority of their mutations can be targeted with drugs. In a recent study, Kimberly Stegmaier, MD, at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and her colleagues systematically deleted every gene in the genome in a number of childhood cancers. This led them to previously unknown — and targetable — genes that help drive tumor growth.

But Stegmaier is also interested in epigenetic regulators — proteins that help control the regulation of genes and contribute to many pediatric cancers. They’re a hot subject of research: Child cancers tend to arise in developing tissues, and epigenetic regulators are active during early development. Clinical trials are starting to test drugs that inhibit epigenetic cancer-promoting factors.

There’s a problem, though: Cancers often become resistant to targeted inhibitors, including epigenetic inhibitors. So, again using genome-wide approaches, Stegmaier set out to find ways to overcome this resistance.

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3D human tissue construct could de-risk vaccine development

Ofer Levy (L) with Guzman Sanchez-Schmitz (PHOTO: MICHAEL GODERRE)

Immunization is one of modern medicine’s greatest success stories. Yet we still lack vaccines for common diseases such as HIV and respiratory syncytial virus. Other vaccines are only moderately effective, like those against tuberculosis or pertussis. The average vaccine can take a decade or more to develop, at a cost of hundreds of millions of dollars, and vaccines that worked flawlessly in mice regularly fail in clinical trials. As a result, many companies are reluctant to enter into vaccine development.

“We need a way to rapidly assess vaccine candidates earlier in the process,” says Ofer Levy, MD, PhD, a physician-scientist in the Division of Infectious Diseases at Boston Children’s Hospital and director of the Precision Vaccines Program. “It’s simply not possible to conduct large-scale, phase 3, double-blind, placebo-controlled studies of every potential vaccine for every pathogen we want to protect against.”

In a paper published today in Frontiers in ImmunologyLevy’s team describes the first modeling laboratory system for testing human immune responses to vaccines — outside the body.

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Alexa: How are smart speakers helping patients, caregivers and clinicians?

Susan Bennett, the voice of Siri, emcees the Voice.Health Summit
Susan Bennett, the original voice of Apple’s Siri, emcees the 2018 Voice.Health Summit. (PHOTOS COURTESY IDHA/BOSTON CHILDREN’S HOSPITAL)

Sarah Lindenauer is product and portfolio manager for the Innovation & Digital Health Accelerator (IDHA) at Boston Children’s Hospital.

Where can you hear the voice of Siri introducing a keynote speaker? Or see the developers of the first healthcare skill for voice present alongside leading pharmaceutical and health insurance companies? Experience demos of cutting-edge voice technologies from 20+ startups from around the world, in simulated healthcare environments?

It all went down October 17th in Boston at the Voice.Health Summit, presented by Boston Children’s Hospital’s Innovation and Digital Health Accelerator (IDHA) as a run-up to the Connected Health Conference. More than 300 leading innovators in voice tech in healthcare came from around the world for a day of immersion and to answer the question, “What’s next?”

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Science Seen: Using Twitter to map hospitals’ stance toward LGBT patients

Hswen Y et al. Social Science & Medicine Oct 2018. DOI: 10.1016/j.socscimed.2018.08.031  

How sensitive are hospitals to the needs of lesbian, gay, bisexual and transgender (LGBT) patients? In a 2010 survey by Lambda Legal, 70 percent of transgender patients and 56 percent of gay/lesbian/bisexual patients reported discrimination from health care providers. Clinicians refused to provide needed care, refused to touch them or used excessive precautions, blamed them for their health status, were verbally abusive or were physically violent.

A new exploratory study, published in the October issue of Social Science & Medicine, turned to social media for a view from the ground. The researchers, Yulin Hswen of Harvard T.H. Chan School of Public Health and Jared Hawkins, PhD, MMSc, of Boston Children’s Hospital’s Informatics Program, analyzed 1,856 publicly available tweets from 2015-2017.

“Information from social media and other online sources can help us gain authentic and unsolicited accounts from vulnerable patient groups, like LGBT individuals who are not typically represented,” says Hswen.

Based on the tweets, the team determined which hospitals were more supportive of LGBT patients (the blue dots in the above map) and which were less supportive (the red dots).

The identified tweets included Twitter handles from 653 hospitals and contained LGBT-related terms: LGBT, transgender, trans, intersex, sex change, transisbeautiful, tranny, drag queen, preferred pronoun, transhealth, genderodyssey, cis, gay, lesbian, queer, rainbowhealth, gender fluid, homosexual, bisexual, homo, homophob and transphobe. A tweet classed as supportive might read, “@Hospital is hosting a LGBT resource fair;” a negative tweet might read: “Having sex with men does not mean I deserve less @Hospital.”

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