Stories about: cardiology

Can we teach heart cells to grow up?

normal and mutant cardiomyocytes
A mutant heart muscle cell (in green) surrounded by normal cells. The mutant cell lacks Srf, a master maturation gene. It is unable to grow in size and lacks the fine membrane invaginations that help coordinate muscle contractions (appearing as vertical striations in the normal cells). (IMAGE: GUO Y; ET AL. NAT COMMS 9 #3837 (2018).]

Scientists around the world have been trying to replace damaged heart tissue using lab-made heart-muscle cells, either injecting them into the heart or applying patches laced with the cells. But results to date have been underwhelming.

“If you make cardiomyocytes in a dish from pluripotent stem cells, they will engraft in the heart and form muscle,” says William Pu, MD, director of Basic and Translational Cardiovascular Research at Boston Children’s Hospital. “But the muscle doesn’t work very well because the myocytes are stuck in an immature stage.”

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Trial shows chemotherapy is helping kids live with pulmonary vein stenosis

Magnification of pulmonary vein tissue showing signs of pulmonary vein stenosis (plump abnormal cells stained dark magenta).
Magnification of pulmonary vein tissue showing signs of pulmonary vein stenosis (plump abnormal cells stained dark magenta). Credit: Boston Children’s Hospital Department of Pathology

Pulmonary vein stenosis (PVS) is a rare disease in which abnormal cells build up inside the veins responsible for carrying oxygen-rich blood from the lungs to the heart. It restricts blood flow through these vessels, eventually sealing them off entirely if left untreated. Typically affecting young children, the most severe form of PVS progresses very quickly and can cause death within a matter of months after diagnosis.

Until recently, treatment options have been limited to keeping the pulmonary veins open through catheterization or surgery. Yet this approach only removes the cells but does nothing to prevent their regrowth. Now, a clinical trial shows that adding chemotherapy to a treatment regimen including catheterization and surgery can deter abnormal cellular growth and finally give children with PVS a chance to grow up.

Results of the trial, run by the Boston Children’s Hospital Pulmonary Vein Stenosis Program, were recently published in the Journal of Pediatrics.

“Through this approach, we’ve created the first-ever population of survivors who are living with severe PVS,” says Christina Ireland, RN, MS, FNP, who has managed enrolling patients in the trial and treating new patients since the trial ended. “We’ve changed this disease from an acute killer to a chronic, manageable condition.”

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Five devices for pediatrics get help in advancing to market

kids with pediatric devices playing doctor

Medical devices for children tend to have small markets, so development can lag up to a decade behind similar devices for adults. The Boston Pediatric Device Consortium (BPDC), formed through an FDA initiative, aims to change that math.

This month, the BPDC and the Innovation and Digital Health Accelerator at Boston Children’s Hospital announced five winners of a national pediatric device challenge. Each winner will receive a combination of up to $50,000 in funding per grant award and/or in-kind support from leading medical device strategic partners, including Boston Scientific, CryoLife, Edwards Lifesciences, Health Advances, Johnson & Johnson Innovation, Medtronic, Smithwise, Ximedica and the Boston Children’s Simulator Program. These organizations will provide mentorship, product manufacturing and design services, simulation testing, business plan development, partnering opportunities and more.

“We have a major unmet need for pediatric medical devices that are specifically designed to address the demands of a growing, active child,” said BPDC leader Pedro del Nido, MD, chief of Cardiac Surgery at Boston Children’s, in a press release. “We are pleased to support these teams as they work toward accelerating their technologies from concept to market.”

The five Challenge winners are:

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Pediatric innovators showcase highlights inventions

Innovators Showcase Boston Children's HospitalSome great inventions were on view this week at the second annual Boston Children’s Hospital Innovators Showcase. Hosted by the hospital’s Innovation Acceleration Program and Technology & Innovation Development Office, the event featured everything from virtual reality goggles with gesture control to biomedical technologies. Below are a few new projects that caught Vector’s eye (expect to hear more about them in the coming months), a kid-friendly interview about the SimLab and list of inventions kids themselves would like to see. (Photos by Katherine Cohen except as noted)

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PVNH: Could this genetic disorder have a ‘butterfly’ effect?

The butterfly effect is defined as “the sensitive dependence on initial conditions, where a small change at one place in a deterministic nonlinear system can result in large differences to a later state.” In medicine, the identification of a rare disease or a genetic mutation may provide insights that spread well beyond the initial discovery.

And in genetics, scientists are learning just how widespread the effects are for mutations in one gene: filaminA (FLNA).

FLNA is a common cause of periventricular nodular heterotopia (PVNH), a disorder of neuronal migration during brain development. The syndrome was first described by the late Peter Huttenlocher, MD, and the gene was identified by Christopher Walsh, MD, PhD, of Boston Children’s Hospital.

In normal brain development, neurons form in the periventricular region, located around fluid-filled ventricles near the brain’s center, then migrate outward to form six onion-like layers. In PVNH, some neurons fail to migrate to their proper position and instead form clumps of gray matter around the ventricles.

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Modified RNA offers drug-like approach to regenerating heart tissue

In mice, VEGF-A modRNA visibly improved blood supply to heart muscle (right image).
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 cardiac 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.

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Scaling up quality improvement: How do we motivate providers?

Engaging clinicians in change will require a cultural shift. (David Oliva/Wikimedia Commons)
Engaging clinicians in change will require a cultural shift. (David Oliva/Wikimedia Commons)
Alyssa Bianca Velasco, ScB, is a clinical data specialist for the Standardized Clinical Assessment and Management Plans (SCAMPs) program at Boston Children’s Hospital.

Reducing health care costs doesn’t have to involve making sacrifices in patient safety or quality of care or holding clinicians to rigid guidelines. Over the past several years, Boston Children’s Hospital has rolled out a methodology known as Standardized Clinical Assessment and Management Plans (SCAMPs). Described in the May issue of Health Affairs, SCAMPs are based on the idea that clinicians should be able to diverge from established medical best practices, provided they document the reasons and track the results—in essence making continual data-driven modifications to practice.

The success of SCAMPs in reducing practice variability and costs and improving outcomes at Boston Children’s has led other institutions, one by one, to adopt them. In the next phase, we plan to expand SCAMPs much more broadly, creating a network of hospitals that will pool pertinent clinical data into a centralized non-profit institution, the Institute for Relevant Clinical Data Analytics (IRCDA).

I am part of a team that is providing training, analytics and IT support to help make that large-scale implementation happen.

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Pediatric hospitals challenged by new adult heart population

Adult and child-mikecogh-FlickrAdvances in medical care sometimes present challenges on the flipside. Case in point: Over the past three decades, progressive developments in pediatric cardiac care have allowed many babies born with congenital heart disease (CHD) to survive. And longevity continues to improve. This progress, however, has brought hospitals a burgeoning patient population with tremendously complex and varied disease states.

About 90 percent of children born with heart defects now survive to adulthood, thanks to diagnostic, interventional and critical care improvements. Specifically, one-year survival has improved from 67.4 percent from 1979 to 1993, to 82.5 percent from 1994 to 2005.

“The number of pediatric hospital admissions for congenital heart disease is increasing relatively slowly, but as the patients live longer and develop common adult medical issues, adult patient admissions are increasing much more rapidly,” says Alexander Opotowsky, MD, MPH, cardiologist at the Boston Adult Congenital Heart (BACH) and Pulmonary Hypertension services at Boston Children’s Hospital.

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Turning heart growth on and off: MicroRNAs could point to new treatments

In mice, boosting amounts of a microRNA family called miR-17-92 led to dramatic enlargements of embryonic and postnatal hearts, with thicker ventricle walls.

 

Challenging accepted wisdom about the heart, Boston Children’s Hospital cardiologist Bernhard Kühn, MD, recently showed that infants, children and adolescents are capable of generating new heart muscle cells, or cardiomyocytes. That work raised the possibility that scientists could stimulate regeneration to repair injured hearts.

Now, we have a potential therapeutic target to accomplish this: a family of microRNAs called miR-17-92 that regulates cardiomyocyte proliferation. In Circulation Research earlier this month, a team led by Kühn’s research colleague Da-Zhi Wang, PhD, demonstrates its potential.

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Raising an early warning in the ICU: T3

How can ICU clinicians manage the data from all these monitors?

With the Internet’s meteoric rise in the last 20 years—to the point of being available 24/7 in your pocket—technology pundits, psychologists and sociologists have been sounding ever louder warnings about information overload: the constant onslaught of communication, information and media coming at us all the time, and in ever greater volume.

Now imagine you’re a doctor or nurse in an intensive care unit (ICU). For you, information overload isn’t just a daily reality—it’s a necessary one. To make the right decisions at the right time for each patient, you must keep tabs on numerous bedside monitors—in the ICUs at Boston Children’s Hospital, that’s 10 or more for each child.

Melvin C. Almodovar, MD, medical director of Boston Children’s Cardiac Intensive Care Unit (CICU), and his colleagues wanted a better way to assess the patient’s physiologic state and catch crises before they happen.

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