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Nancy Fliesler

At TEDx Longwood this spring, Leonard Zon, MD, founder and director of the Stem Cell Program at Boston Children’s Hospital, took the stage. In his enthusiastic yet humble style, he took the audience on a journey that included time-lapse video of zebrafish embryos developing, a riff by Jay Leno and a comparison of stem cell “engraftment” to a college kid coming home after finals: “You sleep for three days, and on day 4, you wake up and you’re in your own bed.” Three takeaways:

1)   Stem cells made from our own skin cells can help find new therapeutics. With the right handling, they themselves can be therapeutics, producing healthy muscle, insulin-secreting cells, pretty much anything we need. (So far, this has just been done in mice.)

2)   Zebrafish, especially when they’re see-through, can teach us how stem cells work and can be used for mass screening of potential drugs. The Zon Lab boasts 300,000 of these aquarium fish, and can mount robust “clinical trials” with 100 fish per group.

3)   Drugs discovered via zebrafish are in human clinical trials right now: A drug to enhance cord blood transplants for leukemia or lymphoma, and an anti-melanoma drug originally used to treat arthritis.

Zon, who co-founded the biopharm company Fate Therapeutics, will be part of a judging panel of clinicians and venture capitalists for the Innovation Tank at Boston Children’s Global Pediatric Innovation Summit + Awards (Oct. 30-31). Don’t miss it!

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A restored cornea grown from human ABCB5-positive limbal stem cells

A restored, clear cornea grown from ABCB5-positive limbal stem cells. (Image courtesy of the researchers)

Severe burns, chemical injury and certain diseases can cause blindness by clouding the eyes’ corneas and killing off a precious population of stem cells that help maintain them. In the past, doctors have tried to regrow corneal tissue by transplanting cells from limbal tissue—found at the border between the cornea and the white of the eye. But they didn’t know whether the tissue contained enough of the active ingredient: limbal stem cells.

How cancer research led to a regenerative treatment for blindness.

Results have therefore been mixed. “Limbal stem cells are very rare, and successful transplants are dependent on these rare cells,” says Bruce Ksander, PhD, of the Massachusetts Eye and Ear/Schepens Eye Research Institute. “If you have a limbal stem cell deficiency and receive a transplant that does not contain stem cells, the cornea will become opaque again.”

Limbal stem cells have been sought for over a decade. That’s where a “tracer” molecule called ABCB5—first studied in the context of cancer—comes in. Full story »

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spina bifida myelomeningocele

Prenatal cell therapy could avoid the need for invasive surgery to repair myelomeningocele.

The neural tube, which becomes the spinal cord and brain, is supposed to close during the first month of prenatal development. In children with spina bifida, it doesn’t close completely, leaving the nerves of the spinal cord exposed and subject to damage. The most common and serious form of spina bifida, myelomeningocele, sets a child up for lifelong disability, causing complications such as hydrocephalus, leg paralysis, and loss of bladder and bowel control.

New research from Boston Children’s Hospital, though still in animal models, suggests that standard amniocentesis, followed by one or more injections of cells into the womb, could be enough to at least partially repair spina bifida prenatally.

Currently, the standard procedure is to operate on infants soon after delivery. Full story »

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music and executive functionMy daughter just surprised me by signing up for fifth grade band starting this fall. To my further delight, some new research—using both cognitive testing and brain imaging—suggests that as she practices her clarinet, she also may be honing her executive functions.

Like a CEO who’s on top of her game, executive functions—separate from IQ—are those high-level brain functions that enable us to quickly process and retain information, curb impulsive behaviors, plan, make good choices, solve problems and adjust to changing cognitive demands. While it’s already clear that musical training relates to cognitive abilities, few previous studies have looked at its effects on executive functions specifically.

The study, appearing this week in PLOS ONE, compared children with and without regular musical training, as well as adults. To the researchers’ knowledge, it’s the first such study to use functional MRI (fMRI) of brain areas associated with executive function and to adjust for socioeconomic factors. Full story »

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Classic Penfield motor homunculus could be different in soccer playersOne of my very favorite images in science, Dr. Wilder Penfield’s classic motor homunculus, shows how much brain real estate is devoted to controlling movement of different parts of the body. Notice the huge hands and the tiny feet. As the World Cup gets underway, soccer fan Jeffrey Holt, PhD, also a Boston Children’s Hospital neuroscientist, writes that soccer is more than just a great sport, it’s “a triumphant display of the incredible plasticity of the human brain… because the soccer player is limited by one simple rule: no hands!”

Though no one’s actually taken a look, Holt imagines that the brains of great soccer players like Cristiano Ronaldo, Lionel Messi or Neymar would have much expanded neural representation of the feet. Read more in his post on WBUR-Boston’s Cognoscenti blog.

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Mila-cap-ball-2014-05-08 12.24.14Is 9-month-old Mila Goshgarian at risk for developing autism spectrum disorder (ASD)? Her 4-year-old twin brothers are both on the spectrum, so statistically her chances are at least 20 percent.

Her mother, Tonia, brought her into Boston Children’s Hospital for the Infant Sibling Project, which works with babies who are at increased risk of developing ASD in hopes of discovering early brain biomarkers for the disorder. This is Mila’s fifth visit; she’s been coming to the Labs of Cognitive Neuroscience for testing since the age of 3 months. Full story »

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boy with cerebral palsyThe start of what promises to be a lengthy, multi-part endeavor has begun unfolding on Capitol Hill. It’s an attempt to reform the Medicaid program so that children with medical complexity (those with a single, serious medical condition, or multiple chronic conditions) can receive higher quality care with fewer emergency department visits and fewer hospital admissions.

When you think of medically complex children, think of children living with conditions such as spina bifida or cerebral palsy, children dependent on ventilators or feeding tubes, or children with genetic disorders. They represent just 6 percent of the 43 million children on Medicaid—yet they account for about 40 percent of Medicaid’s spending on children. Their care is often fragmented and poorly coordinated.

The reform effort, led by more than 60 participating pediatric hospitals and supported by the Children’s Hospital Association (CHA), focuses on Medicaid because it’s the single largest insurance provider for children. The backdrop is a cost-conscious Congress that’s the most politically polarized ever, passing the fewest bills ever. Full story »

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Macrophage therapy IBD

Giving patients the right kind of immune cells could curb their IBD, research suggests.

Inflammatory bowel disease (IBD) is miserable for anyone, but when it strikes a child under age 5, it’s much more severe, usually causing bloody diarrhea, wrenching abdominal pain and stunted growth. Early-onset IBD is rare, but on the rise: For reasons unknown, its incidence is increasing by about 5 percent per year in some parts of the world.

A recently identified form of early-onset IBD shows up within months of birth, causing severe inflammation in the large intestine and abscesses around the anus. Recently linked to genetic mutations in the cellular receptor for a signaling protein, interleukin-10 (IL-10), it can also lead to lymphoma later in life.

As with all early-onset IBD, IL-10-receptor deficiency has no good treatment. A bone marrow transplant is actually curative, but carries many risks, especially in infants.

“We’ve been trying to understand why IBD in these children is so severe and presents so early,” says Dror Shouval, MD, a pediatric gastroenterologist at Boston Children’s Hospital and a fellow in the lab of Scott Snapper, MD, PhD. The beginnings of such an understanding—detailed recently in the journal Immunity—could lead to a new treatment approach for this and perhaps other kinds of early-onset IBD. Full story »

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Preliminary findings suggest steroid treatment could improve language and behavior in children with ASD.

Preliminary findings suggest that steroid treatment could improve language and behavior in children with regressive ASD.

In as many as a third of cases of autism spectrum disorder (ASD), children’s language and social skills develop normally at first. Then, between 1 and 2 ½ years of age, they begin to regress, losing words and retreating from interactions with parents—often abruptly. There has been some anecdotal evidence that steroid treatment can help these children, and a small retrospective study agrees—finding improved language and behavior and reversal of a characteristic abnormality in the language processing area of their brains.

Researchers led by Frank H. Duffy, MD, of the Epilepsy Center at Boston Children’s Hospital, looked back at 20 children 3 to 5 years old with documented regressive ASD who had received steroid therapy (prednisolone) under a neurologist’s supervision, generally starting several months after their regression was noted. For comparison, the team also reviewed data from 24 similar autistic children who did not receive steroids. Full story »

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ICISS screengrab2What happens when you try to scale up a successful quality initiative? Eric Fleegler, MD, MPH, and Eugenia Chan, MD, MPH, are facing that challenge with ICISS, their web-based system that went quickly from ideation to adoption by 3,000 patients with attention deficit hyperactivity disorder (ADHD) and their families.

ICISS enables parents, teachers and patients to give online updates on medications, symptoms and school performance in close to real time, then packages that data for clinicians in a visual, actionable fashion. But tasked with introducing ICISS into four other clinics at Boston Children’s Hospital—autism, asthma, depression and epilepsy—a raft of practical, legal and philosophical questions came up about how to handle these patient-generated health data. For example:

  • How should we inform families that they need to contact their provider directly with immediate concerns?
  • What if a parent indicates that a child is at risk of self-harm, and how can we manage this in a timely manner?
  • How can clinics afford to hire additional staff to screen and manage alerts from ICISS when this activity is non-reimbursable?
  • What is the obligation of the provider if actionable data show up months in advance of the scheduled visit?

Fleegler and Chan discuss the challenges and lessons learned in our sister publication, Innovation Insider. We’d be interested to hear from others facing similar questions in handling patient-generated health data.

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