Author: Nancy Fliesler

Delivered through amniotic fluid, stem cells could treat a range of birth defects

Transamniotic stem cell therapy, or TRASCET, is like amniocentesis is reverse.
Amniotic fluid is routinely withdrawn for prenatal testing. It could also be a delivery route for fetal cell therapy to treat congenital anomalies, with broader applications than once thought.

The amniotic fluid surrounding babies in the womb contains fetal mesenchymal stem cells (MSCs) that can differentiate into many cell types and tissues. More than a decade ago, Dario Fauza, MD, PhD, a surgeon and researcher at Boston Children’s Hospital, proposed using these cells therapeutically. His lab has been exploring these cells’ healing properties ever since.

Replicated in great quantity in the lab and then reinfused into the amniotic fluid in animal models — a reverse amniocentesis if you will — MSCs derived from amniotic fluid have been shown to repair or mitigate congenital defects before birth. In spina bifida, they have induced skin to grow over the exposed spinal cord; in gastroschisis, they have reduced damage to the exposed bowel. Fauza calls this approach Trans-Amniotic Stem Cell Therapy, or TRASCET.

New research findings, reported this month in the Journal of Pediatric Surgery, could expand TRASCET’s therapeutic potential.

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Can rare pain syndromes point the way to new analgesics?

analgesic drug discovery could reduce prescription opioid use
Boston Children’s Hospital and Amgen will collaborate to discover and accelerate non-addicting pain drugs.

As the opioid epidemic deepens and drug overdoses increase, effective non-addicting painkillers are desperately needed. Efforts to discover new pain pathways to target with new drugs have thus far had little success. Other promising research is investigating triggerable local delivery systems for non-opioid nerve blockers, but it’s still in the early stages.

A new collaboration between Boston Children’s Hospital and the biopharmaceutical company Amgen is aimed at accelerating new pain treatments. Announced yesterday, it will revolve around patients with rare, perplexing pain syndromes. The scientists hope that the genetic variants they find in these patients will shed new light on pain biology and lead to new ways of controlling pain. 

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What do hospitals want from prospective digital health partners?

how digital health startups can better approach hospitals
How digital health startups can better approach hospitals.

How can the growing number of digital health startups sell their products to large-scale healthcare enterprises? Earlier this year, Rock Health, a San Francisco-based venture fund dedicated to digital health, conducted 30-minute interviews with executives at multiple startups and a few large healthcare organizations. They identified several key sticking points: navigating the internal complexities of hospitals, finding the right buyer, identifying the product’s value proposition and relevance to the hospital and avoiding “death by pilot.”

Now, in a Rock Health podcast, John Brownstein, PhD, Chief Innovation Officer at Boston Children’s Hospital’s Innovation and Digital Health Accelerator and Adam Landman, MD, MS, MIS, MHS, Chief Information Officer at Brigham and Women’s Hospital and part of its Innovation Hub, offer further tips from the inside. They were hosted by Rock Health’s director of research, Megan Zweig.

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Dock Health’s shared ‘to do’ list for clinical teams — so basic, so necessary

Dock Health - a shared to-do list for clinical teams - could ease clinical burnout

While something as simple as a “to-do list” might seem trivial, a secure hub to store, prioritize and assign clinical and administrative tasks could be game-changing in healthcare.

Michael Docktor, MD, of Boston Children’s Hospital made this case yesterday at the Health 2.0 Conference in Santa Clara, Calif. He demonstrated Dock Health, a secure iOS mobile and web application that helps medical teams manage the numerous tasks that fall under clinical care. The idea was born in his gastroenterology practice at Boston Children’s and was incubated by the hospital’s Innovation and Digital Health Accelerator (IDHA).

“In an average day in clinic, I might see 15 patients and get 75 emails, 10 secure messages, three pages and five [electronic medical record] messages in my inbox,” Docktor writes on Medium. “Not too long ago, some emails were from frustrated colleagues, asking me to do something for a second or third time. Sadly, some were from parents of my patients, kindly reminding me that they were sitting in the lab waiting for the orders I forgot to place or trying to book their colonoscopy, for which I had forgotten to submit the form.”

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How do we sense moonlight? Daylight? There’s a cell for that

environmental light sensing must span a wide spectrum of light intensities

To run our circadian clocks, regulate sleep and control hormone levels, we rely on light-sensing neurons known as M1 ganglion cell photoreceptors. Separate from the retina’s rods and cones, M1 cells specialize in “non-image” vision and function even in people who are blind.

Reporting in today’s Cell, neuroscientists at Boston Children’s Hospital describe an unexpected system that M1 cells use to sense changing amounts of environmental illumination. They found that the cells divvy up the job, with particular neurons tuned to different ranges of light intensity.

“As the earth turns, the level of illumination ranges across many orders of magnitude, from starlight to full daylight,” says Michael Do, PhD, of the F.M. Kirby Neurobiology Center at Boston Children’s Hospital, senior author on the paper. “How do you build a sensory system that covers such a broad range? It seems like a straightforward problem, but the solution we found was a lot more complex than expected.”

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New hope for X-linked myotubular myopathy as gene therapy clinical trial begins

gene therapy myotubular myopathy

Boys born with X-linked myotubular myopathy (XLMTM) face a grim prognosis. Extreme muscle weakness leaves many ventilator-dependent from birth, and most infants need feeding tubes. About half pass away before 18 months of age.

Last week, the biotechnology company Audentes Therapeutics announced the dosing of the first patient in a gene-therapy clinical trial — 21 years after the MTM1 gene was first cloned.

Hopes are high. Gene therapy has already shown striking benefits in dogs with XLMTM in studies co-authored by Alan Beggs, PhD, director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital, and colleagues at Généthon and the University of Washington. In the most recent study, 10-week-old Labrador retrievers already showing signs of the disease showed improvements in breathing, limb strength and walking gait after a single dose of the gene therapy vector.

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Monitoring mitochondria: Laser device tells whether oxygen is sufficient

Shining a laser-based device on a tissue or organ may someday allow doctors to assess whether it’s getting enough oxygen, a team reports today in the journal Science Translational Medicine.

Placed near the heart, the device can potentially predict life-threatening cardiac arrest in critically ill heart patients, according to tests in animal models. The technology was developed through a collaboration between Boston Children’s Hospital and device maker Pendar Technologies (Cambridge, Mass.).

“With current technologies, we cannot predict when a patient’s heart will stop,” says John Kheir, MD, of Boston Children’s Heart Center, who co-led the study. “We can examine heart function on the echocardiogram and measure blood pressure, but until the last second, the heart can compensate quite well for low oxygen conditions. Once cardiac arrest occurs, its consequences can be life-long, even when patients recover.”

In critically ill patients with compromised circulation or breathing, oxygen delivery is often impaired. The new device measures, in real time, whether enough oxygen is reaching the mitochondria, the organelles that provide cells with energy.

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Pediatric devices wanted: Boston Children’s Hospital and the Boston Pediatric Device Consortium launch $250,000 challenge

Boston Pediatric Device Strategic Partner Challenge opens

There’s generally little incentive for industry to develop medical devices for children: The pediatric market is small (most children are healthy) and clinical trials are harder to do in children.

“Innovation in medical devices with the potential to improve the health of children and adolescents continues to lag in comparison to those for adults,” says Pedro del Nido, MD, leader of the Boston Pediatric Device Consortium and Chief of Cardiac Surgery at Boston Children’s Hospital. 

This week, the Innovation and Digital Health Accelerator (IDHA) at Boston Children’s Hospital and the Boston Pediatric Device Consortium (BPDC) announced a national challenge to try to remedy this problem. The Boston Pediatric Device Strategic Partner Challenge will award up to $50,000 to entrepreneurs and innovators seeking to create novel pediatric medical devices, from a total pool of up to $250,000.

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3D organoids and RNA sequencing reveal the crosstalk driving lung cell formation

lung disease
A healthy lung must maintain two key cell populations: airway cells (left), and alveolar epithelial cells (right). (Joo-Hyeon Lee)

To stay healthy, our lungs have to maintain two key populations of cells: the alveolar epithelial cells, which make up the little sacs where gas exchange takes place, and bronchiolar epithelial cells (also known as airway cells) that are lined with smooth muscle.

“We asked, how does a stem cell know whether it wants to make an airway or an alveolar cell?” says Carla Kim, PhD, of the Stem Cell Research Program at Boston Children’s Hospital.

Figuring this out could help in developing new treatments for such lung disorders as asthma and emphysema, manipulating the natural system for treatment purposes.

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Bad to the bone: New light on the brain’s venous system… and on craniosynostosis

cerebral veins and skull development in a normal child
Normal skull and brain venous development in a young child (courtesy Tischfield et al).

A recent study rocked the neuroscience world by demonstrating what in retrospect seems obvious: the brain has its own lymphatic system to help remove waste. A new study, from the laboratory of Elizabeth Engle, MD, at Boston Children’s Hospital, sheds light on another critical, little-studied part of the brain’s drainage system: the dural cerebral veins that remove and reabsorb excess cerebrospinal fluid.

The story of these vessels, the cover article in the next Developmental Cell, is a great example of lab scientists and physicians joining to make fundamental discoveries in biology. Strangely, critical clues come from children with craniosynostosis, a congenital malformation in which the skull plates fuse together too early in prenatal development, resulting in abnormal head shapes and, often, neurologic complications.

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