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Tom Ulrich

Urine Vascular Biology Program netrin brain tumor biomarker Edward Smith Michael Klagsbrun

A good biomarker is one whose levels go up or down as a patient’s disease worsens or wanes. A great biomarker also gives key insights into disease development. A really great biomarker does both of these things and also serves as a treatment target.

With a protein called netrin-1, Edward Smith, MD, and Michael Klagsbrun, PhD, seem to have hit the trifecta. In a recent paper in Cancer Research, they report a clear relationship between urine netrin levels and medulloblastoma, the most common malignant brain tumor of children.

And show that netrin fuels the tumor’s invasion into healthy brain tissue.

And that blocking netrin may, at least in the laboratory, check the tumor’s spread. Full story »

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Wearable health gadgets fitness trackers Apple HealthKit Google fit

Fitbit, Jawbone, Nike, Withings…a lot of companies are already in the wearable/mobile health technology and data tracking game. But a couple of really big players are stepping on to the court.

At their most recent Worldwide Developers Conference, Apple announced both an app and a framework—Health and HealthKit—that will tie in with various wearable technologies and health apps. HealthKit will also feed data into electronic medical record (EMR) systems like Epic, which runs at some of the largest hospitals in the country. And rumors abound that an upcoming Apple smartwatch (iWatch? iTime? Only Tim Cook knows right now) will carry a host of sensors for tracking activity and health data.

Google also wants to get into the game with a health data framework called Fit that they announced at their I/O conference in June. Unlike Apple, its strategy seems more focused on providing a standard way for trackers, devices and apps from different manufacturers to talk to Android Wear devices.

What will entry of these big players mean? We asked Michael Docktor, MD, clinical director of Boston Children’s Hospital’s Innovation Acceleration Program. Full story »

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Ebola response ethics experimental treatment therapy ZMapp

Guinean Red Cross volunteers prepare to decontaminate a hospital in the capital, Conakry. (European Commission DG ECHO/Flickr)

The world paused for a moment when the news broke last week that two Ebola-infected American missionaries working in Liberia had received an experimental therapy called ZMapp. As I write this, both patients are back on U.S. soil, and seem to be responding well to the treatment.

But was it ethical?

That difficult question can be divided into two. First is the question of whether it was ethical to give the two patients a drug that, up to that point, had never been tested in people. The second—in some ways thornier—question is: Was it ethical to give the treatment to two Americans but not the nearly 1,850 West Africans infected in the outbreak (as of August 11)? Full story »

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This interactive map of the Ebola outbreak, produced by HealthMap, paints a picture of the epidemic's course from its first public signs in March. Mouse around, scroll down, zoom and explore. And click play to see how events have unfolded thus far.

Sobering news keeps coming out of the West African Ebola outbreak. According to numbers released on August 6, the virus has sickened 1,711 and claimed 932 lives across four nations. The outbreak continues to grow, with a high risk of continued regional spread, according to a threat analysis released by HealthMap (an outbreak tracking system operated out of Boston Children’s Hospital) and Bio.Diaspora (a Canadian project that monitors communicable disease spread via international travel).

“What we’ve seen here—because of inadequate public health measures, because of general fear—is [an outbreak that] truly hasn’t been kept under control,” John Brownstein, PhD, co-founder of HealthMap and a computational epidemiologist at Boston Children’s Hospital, told ABC News. “The event started, calmed down and jumped up again. Now, we’re seeing movement into densely populated areas, which is highly concerning.”

If you’re interested in keeping tabs on the outbreak yourself, there are several tools that can help. Full story »

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Thomson Reuters most highly cited scientists

It’s not every day you wake up and find out you’ve been named one of the most influential people in science. But that day recently came to seven Boston Children’s Hospital researchers, when Thomson Reuters named them as some of the most highly cited scientists in the world.

Every few years, the information broker and news outlet combs through its Web of Science and InCites systems—which track the scientific literature—to see whose work other researchers consistently refer to in their papers. It then creates two categories: highly cited papers (those that “rank in the top 1 percent by citations for their field and year of publication”) and hot papers (ranking “in the top 0.1 percent by citations for their field”). Full story »

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ADHD long term drug safety rare adverse events FDA approval Kenneth Mandl Florence BourgeoisYou’d think drugs meant to be taken by children for years would be studied in children for a long time to measure their long-term safety.

You’d think drugs for a condition affecting millions of children would be tested in many, many children to catch any rare side effects.

You’d think all this would happen before the Food and Drug Administration, an agency known for its strict criteria, approved them for marketing.

But if a new PLoS ONE paper by Boston Children’s Hospital’s Florence Bourgeois, MD, MPH, and Kenneth Mandl, MD, MPH, is any indication, you’d be wrong.

In it, the pair reports that the FDA approved 20 attention deficit hyperactivity disorder (ADHD) drugs over the last 60 years without what would be considered sufficient long-term safety and rare adverse event data.

Their findings, they say, point to larger issues in how the FDA’s approval process addresses the long-term safety of drugs intended for chronic use in children. Full story »

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Wong-Baker pain faces subjective objective pain assessment systems neuroscience David Borsook

Subjective measures of pain, like the Wong-Baker face scale (above), are useful in assessing patients' pain, but objective measures would be far better.

“How much pain are you in?” It’s a harder question than you think. Tools for assessing patients’ pain—be they children or adults—rely on their perception: a subjective measure that eludes quantification and can change in response to any number of emotional, psychological or physiological factors.

Being able to objectively quantify pain could open the door to better pain management (especially for patients with chronic or neuropathic pain), better anesthetic dosing during surgical procedures, better understanding of addiction (and how to avoid it) and more.

To do so, we need measurable markers: physiologic parameters that reliably and quantitatively change during the experience of pain. But according to pain researcher David Borsook, MD, PhD—of Boston Children’s Hospital’s departments of Anesthesiology, Perioperative and Pain Medicine and Radiology—discovering such markers requires a better understanding of the larger context and of events that trigger pain, a perspective he refers to as “systems neuroscience.” Full story »

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Old car aging blood cell hematopoietic stem cell blood disorder Derrick Rossi

Like an old, unused car, our aging blood stem cells can accumulate damage over time that they can't fully repair.

My first car was my grandfather’s 1980 Chevrolet Malibu. For about two years before my family gave it to me, it sat unused in Grandpa’s garage—just enough time for all of the belts and hoses to rot and the battery to trickle down to nothing.

Why am I telling this story? Because it’s much like what happens to the DNA in our blood-forming stem cells as we age.

Hematopoietic stem cells (HSCs) spend very little of their lives in an active, cycling state. Much of the time they’re quiescent or dormant, keeping their molecular and metabolic processes dialed down. These quiet periods allow the cells to conserve resources, but also give time an opportunity to wear away at their genes.

“DNA damage doesn’t just arise from mistakes during replication,” explains Derrick Rossi, PhD, a stem cell biology researcher with Boston Children’s Hospital’s Program in Cellular and Molecular Medicine. “There are many ways for damage to occur during periods of inactivity, such as reactions with byproducts of our oxidative metabolism.”

The canonical view has been that HSCs always keep one eye open for DNA damage and repair it, even when dormant. But in a study recently published in Cell Stem Cell, Rossi and his team found evidence to the contrary­­—which might tell us something about age-related blood cancers and blood disorders. Full story »

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Surgical Sam beating heart pediatric trainer mannequin simulation Simulator Program The Chamberlain GroupWe often see medical magic in Hollywood, but it’s not often we see Hollywood magic brought into medicine. Now, Boston Children’s Hospital’s Simulator Program and special-effects collaborators at The Chamberlain Group (TCG) have done just that.

Simulation has become a key component in team training, crisis management, surgical practice and other medical training activities. With simulation, medical teams can add to and hone their skills in an environment where people can make mistakes without risking patient harm—”practicing before game time,” says Boston Children’s critical care specialist Peter Weinstock, MD, PhD, who runs the Simulator Program.

Mannequins are a key part of simulation, and Weinstock’s team, working together with companies, designers and engineers, has developed eerily lifelike ones that can bleed and “respond” to interventions based on computer commands from a technician.

But there are some things Weinstock’s mannequins haven’t been able to capture up to now, like the movements of a beating heart.

That’s where TCG and a new mannequin called Surgical Sam come in. Full story »

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John Kheir, MD, first envisioned an injectable form of oxygen eight years ago, the night one of his patients, a nine-month-old girl, died after catastrophic lung failure. Kheir, a cardiac intensive care specialist at Boston Children’s Hospital, spoke last night to WBZ-TV’s Mallika Marshall, MD, about his efforts to try to buy precious time for children whose lungs stop working:

Want to know more? Read Kheir’s own words about his hopes and challenges for intravenous oxygen in a post he penned for Vector.

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