Flipping the switch on tumor growth

Pictures of angiogenic tumor cells
Time-lapse imaging can reveal tell-tale changes in cellular behaviors associated with tumor growth.

Without a blood supply, a tumor can remain dormant and harmless. But new blood vessel growth from an existing vessel, a process called angiogenesis, is a hallmark of both benign and malignant tumors. During angiogenesis, blood vessels invade tumors and activate them, fueling their growth.

Now, Marsha A. Moses, PhD, who directs the Vascular Biology Program at Boston Children’s Hospital, and members of her laboratory have revealed that a specialized imaging system can detect changes in cell behaviors. These changes predict when tumors are leaving a state of dormancy and becoming more likely to grow.

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An unclassified tumor — with a precisely targeted therapy

Jesus (who received targeted therapy for his tumor) pictured with his father
Jesus and his father, Nathaneal

Early last year, at his home in San Juan, Puerto Rico, Jesus Apolinaris Cruz’s leg hurt so much he could barely sleep. “All day,” the 13-year-old recalls. “It was constant pain.” His parents took him to two local pediatricians, who examined him, drew blood, tested his platelets. No diagnosis. Finally, in April 2016, a physician ordered an MRI. No wonder Jesus’s leg hurt. He had a large, cancerous tumor lodged in his hip.

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Rare disease therapies: Three strategies to bridge the gap between research and industry

Rare disease research: DNA helix pictured here
Genetic mutations underpin many rare diseases.

Right now, there are about 7,000 rare diseases affecting 10 percent of Americans. Only five percent of these diseases have any FDA-approved treatment options.

Panelists:
David Williams, MD: President, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center; Senior VP, Chief Scientific Officer and Chief of Hematology/Oncology, Boston Children’s
Wayne Lencer, MD: Chief of Gastroenterology, Hematology and Nutrition, Boston Children’s
Phil Reilly, MD, JD: Venture Partner at Third Rock Ventures
Alvin Shih, MD, MBA: Chief Executive Officer at Enzyvant

Even at a place like Boston Children’s Hospital, where doctors regularly see children with rare diseases from all over the world, there are big challenges when it comes to drug discovery and treatment.

“Roughly 70 percent of drugs to treat children are used off-label,” says David Williams, Boston Children’s chief scientific officer. “That’s because these drugs were initially developed for adults and have not been tested formally in children.”

In order to cure rare diseases in children and adults, scientists must bridge the gap between research and industry. On May 25, Boston Children’s Technology and Innovation Development Office (TIDO) and MassBio held a candid panel discussion about what it will take to advance the development of rare disease therapies. Here are three of the biggest takeaways

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Under the hood of healthcare innovation: Gajen Sunthara and leveraging EHRs

Gajen Sunthara
(Photo: Greg Weintraub)

What does it take to be an innovator changing our healthcare system for the better? This two-part series profiles two digital health innovators at Boston Children’s Hospital who were named among MedTech Boston’s 40 Under 40 Healthcare Innovators for 2017.

Gajen Sunthara, MSc, has two innovation passions: healthcare policy and electronic health records (EHRs). With professional experiences spanning technology, business and government, he finds himself in a position to effect change in a way that few others can.

“Gajen’s passion for healthcare is evident from the moment that you meet him,” says Farhanah Sheets, a software engineer at Boston Children’s Innovation and Digital Health Accelerator (IDHA) who reports to Sunthara. “No matter how big or small the idea, he brings a level of excitement to each project that is contagious.”

As director of Innovation R&D for IDHA, Sunthara is leading significant efforts around EHR interoperability — the ability of healthcare information systems to exchange and use each other’s data. He’s also focused on creating applications that can easily be integrated into any EHR system.

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Precision drug delivery systems could ‘trigger’ an age of nanomedicine

Nanoscale drug delivery systems could target and destroy diseases in our body with increased precision and reduced side effects.

What if we could deliver biocompatible nanoparticles into the body and then activate them to release drugs exactly where they are needed, without causing side effects elsewhere?

Scientists like Daniel Kohane, MD, PhD, of Boston Children’s Hospital, are developing nanoscale drug delivery systems to do just that, using a variety of materials and triggers that are sensitive to a range of specific stimuli.

“Triggerable drug delivery systems could improve the treatment of many diseases by reducing side effects and increasing the effectiveness of therapeutics,” says Kohane, who directs the Laboratory for Biomaterials and Drug Delivery at Boston Children’s. He is the senior author on a recent article about the topic in Nature Reviews Materials.

One potential use of nanoscale drug delivery systems is of special interest to Kohane and his lab members

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Study: Repeal of the individual mandate could undercut health care for youth under 26

individual mandate
Diagnosis: No insurance coverage

As the U.S. Senate takes up the proposed American Health Care Act (AHCA), a three-state study provides evidence that eliminating the individual mandate could jeopardize young adults’ health care coverage — even with laws allowing people under 26 to be covered under their parents’ plan.

Researchers led by Lauren Wisk, PhD, of the Division of Adolescent and Young Adult Medicine at Boston Children’s Hospital, analyzed insurance data from 131,542 adolescents and young adults whose family was covered by Harvard Pilgrim Health Care between January 2000 and December 2012.

“With an individual mandate, many more young adults used the dependent coverage provision, and people who were previously dropped from their parents’ plan were more likely to get back on,” Wisk reports.

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Could fecal transplants heal Crohn’s and colitis in children? Two trials are set to find out

two trials test fecal transplant in Crohn's and colitis in children

Could an exciting potential treatment for inflammatory bowel disease (IBD) be found in the gastrointestinal tract itself? That’s the theory behind a pair of new studies by Stacy A. Kahn, MD, which will investigate the potential role of fecal microbial transplant (FMT) in the treatment of Crohn’s disease and ulcerative colitis in children.

In IBD, the immune system attacks healthy cells in the digestive tract, triggering symptoms such as abdominal pain, fatigue, poor growth and bloody diarrhea. Children with IBD can also experience problems elsewhere in the body, including joint pain, liver disorders and eye inflammation.

Known colloquially as the “poop pill,” or “stool transplant,” FMT harnesses growing knowledge about the gut microbiota, the collection of bacteria and other microbes that populate our GI tract.

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Two resilient dogs point to new targets for Duchenne muscular dystrophy

Duchenne muscular dystrophy protective genes
Suflair, at right, is alive and well at 11 years despite having the DMD mutation (courtesy Natássia Vieira)

Two golden retrievers that had the genetic mutation for Duchenne muscular dystrophy (DMD), yet remained healthy, have offered up yet another lead for treating this muscle-wasting disorder.

For several years, Natássia Vieira, PhD, of the University of São Paolo, also a fellow in the Boston Children’s Hospital lab of Louis Kunkel, PhD, has been studying a Brazilian colony of golden retrievers. All have the classic DMD mutation and, as expected, most of these dogs are very weak and typically die by 2 years of age. That’s analogous to children with DMD, who typically lose the ability to walk by adolescence and die from cardiorespiratory failure by young adulthood.

But two dogs appeared unaffected. Both ran around normally. The elder dog, Ringo, lived a full lifespan, and his son Suflair is still alive and well at age 11.

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Predicting influenza outbreaks faster with a digitally-empowered wearable device

Influenza viruses. Outbreaks can be predicted using digital health tools like Thermia.The Thermia online health educational tool, developed at Boston Children’s Hospital, has enabled one-month-faster prediction of seasonal influenza outbreaks in China, via its digital integration with a commercially-available wearable thermometer. The findings appear in a new study published in the American Journal of Public Health.

 “The fact that we were able to predict influenza outbreaks faster than China’s national surveillance programs really shows the capacity for everyday, wearable digital health devices to track the spread of disease at the population level,” says the study’s lead author Yulin Hswen, who is a research fellow in Boston Children’s Computational Epidemiology Group and a doctoral candidate at the Harvard T. H. Chan School of Public Health.

China has 620 million mobile internet users who can theoretically access the standalone Thermia application from any computer, smartphone or even the Amazon Alexa assistant.

Although the Boston Children’s team has previously demonstrated that social media can be used to track disease, this is the first time they’ve shown that outbreaks can be predicted through an integrated wearable device and online tool.

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Webchat to highlight what’s new in pediatric brain tumors

pediatric brain tumors, child MRI

Last September, the National Center for Health Statistics reported that brain tumors have overtaken the much more common leukemia as the leading cause of death from pediatric cancer. Although progress has been made and the promise of more progress is on the horizon, the cure rate for childhood brain tumors lags behind a number of other pediatric cancers.

As pediatric neuro-oncologist Peter Manley, MD, of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center told Live Science, new research on cancer genomics “is so impressive that my feeling is that we will continue to see a decline in deaths.”

To mark Brain Tumor Awareness Month, Mark Kieran, MD, PhD, clinical director of the Brain Tumor Center at Dana-Farber/Boston Children’s, will host a webchat on Monday, May 22 (3:30 p.m. ET). The live chat will highlight the latest research and treatments for pediatric brain tumors. Here’s a look back at some recent developments:

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