Author: Tom Ulrich

My work, my life, my innovations: Katherine Janeway, MD

“It’s all about the patients,” says Katherine Janeway, MD, when asked about the motivations behind her efforts to bring precision medicine to pediatric oncology. But it’s more than that; the drive to combine science and care is in her blood. A solid tumor specialist and cancer genomics researcher at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Janeway is the sixth generation of her family to choose a scientific or medical path—not just as a career, but also as a form of service.

Janeway will bring her patient-centered perspective to the Cancer Genomics + Care panel at Boston Children’s Global Pediatric Innovation Summit + Awards 2015. Read more about her life, work and innovations—and inspirations—by hovering over the objects that surround her everyday.

Learn more about the Boston Children’s summit and register to attend.

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The MERS death rate in Saudi Arabia is double that in South Korea. Why?

map South Korea Saudi Arabia MERS
(Wikimedia Commons)

The Middle East respiratory syndrome (MERS) virus outbreak in South Korea is essentially over. (Not so in Saudi Arabia, where the virus first emerged, though—authorities there have reported a major uptick in new MERS cases in recent days.) And while the country gets back on its feet, some interesting data are starting to come out, especially about the outbreak’s case fatality rate (CFR; the percent of patients infected with the virus who died from it).

John Brownstein, PhD, and Maimuna Majumder, MPH, from Boston Children’s HealthMap team just reported in the journal Emerging Infectious Diseases that MERS’ CFR in South Korea (22 percent) is fully half that seen in Saudi Arabia (44 percent).

This infographic about Brownstein and Majumder’s MERS paper gives a snapshot of the data the analyzed, and what they think those data mean:

MERS virus South Korea Saudi Arabia infographic

Read Brownstein and Majumder’s analysis and check out Boston Children’s Hospital’s news release about the MERS paper.

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Progress in the treatment of childhood leukemia

Although treatments for childhood cancer patients are improving, cancer remains the leading cause of death by disease in children. Doctors and researchers are also focused on decreasing the toxicity of these treatments, which can have side effects years after a child finishes treatment.

“The war against childhood cancer is hardly over,” says Kimberly Stegmaier, MD, a pediatric oncologist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. “We need to do better.”

Stegmaier, who focuses her research on identifying new drug targets and new drugs for childhood leukemiaEwing sarcoma, and neuroblastoma, recently discussed advances in childhood cancer treatment in a Science, Innovation, and Discovery Talk (SID Talk) at Dana-Farber. During the TED Talk-style presentation, Stegmaier explained some of her research in the treatment of sub-microscopic acute lymphoblastic leukemia (ALL) as well as genetic targets in childhood cancers.

“What you can do in an environment where you have chemists, biologists, and clinicians adjacent and working collaboratively is very powerful,” says Stegmaier. “That’s why I’m here today—we need to cure 100 percent of kids, and we can’t do this alone.”

This story originally ran on Dana-Farber Cancer Institute’s Insight blog.

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So far, so good for gene therapy patient Emir Seyrek

Emir Seyrek gene therapy Wiskott-Aldrich ThrivingRemember Emir Seyrek, the Turkish boy who last year was the first patient in gene therapy trial for a genetic immunodeficiency called Wiskott-Aldrich Syndrome? Emir traveled back to the U.S. earlier this month for an annual follow-up visit with his care team at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. The news was quite good.

“Emir is the star of the trial,” Sung-Yun Pai, MD—a Dana-Farber/Boston Children’s gene therapy and immunodeficiency transplant specialist and lead (along with David Williams, MD, and Luigi Notarangelo, MD) of the U.S. arm of the trial—tells our sister blog, Thriving. “He has the highest platelet count of all of the children who have gone through gene therapy with this vector so far. His immune function is excellent, and we have no worries whatsoever from a bleeding standpoint. He’s perfectly safe to play like a normal child.”

Learn more about Emir’s progress on Thriving.


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My work, my life: John Brownstein, PhD

Boston Children’s Hospital’s new chief innovation officer, John Brownstein, PhD, is an epidemiologist by training and a founding father of the growing field of digital epidemiology—the use of digital (especially social and mobile) data from a variety of sources to detect and track disease and promote health. As co-founder of HealthMap and director of the Computational Epidemiology Group in the hospital’s Computational Health Informatics Program, he infuses his work into many aspects of his life—along with a healthy helping of hot sauce.

Hover over the icons in the photo below to learn about the things in Brownstein’s phone, office and life that keep him going.

Brownstein will be one of four panelists discussing Patient Engagement in a Big Data World at Boston Children’s Global Pediatric Innovation Summit + Awards 2015. Learn more about the summit and register to attend.

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Daniel Kohane, MD, PhD, at TEDMED: “Smart vehicles for safer medications”

“The drugs that I take don’t just go to the places in my body they’re supposed to go to do the things they do. They actually go everywhere. And what they do in those other places can be whatever.”

With those words, Daniel Kohane, MD, PhD—director of the Laboratory for Biomaterials and Drug Delivery at Boston Children’s Hospital—launched into a TEDMED talk about technologies that get drugs to where they need to go with much greater precision, like:

“Progress in this field is limited only by the imagination of the investigators and, to some degree, by reality,” says Kohane, who also sees patients in Boston Children’s Department of Critical Care Medicine. “You can achieve really big things by thinking really small.”

Click the image above to watch his whole talk.

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So, what’s your digital phenotype?

Ideally, we’re all supposed to see our doctor once a year for a checkup. It’s an opportunity to see how we’re doing from a health perspective, address any concerns or issues that we may have and catch any emerging issues before they become true problems.

But those visits are really only one-time, infrequent snapshots of health. They don’t give a full view of how we’re doing or feeling.

Now, think for a moment about how often you post something to Facebook or Twitter. Do you post anything about whether you’re feeling ill or down, or haven’t slept well? Ever share how far you ran, the route you biked or your number of steps for the day?

Every time you do, you’re creating a data point—another snapshot—about your health. Put those data points together, and what starts to emerge is a rich view of your health, much richer than one based on the records of your occasional medical visit.

As John Brownstein, PhD—director of the Computational Epidemiology Group (CEG) in Boston Children’s Hospital’s Computational Health Informatics Program and the hospital’s new Chief Innovation Officer—explains in this episode of the Harvard Medical School (HMS) Labcast (click the image above to hear it), this view has a name: your digital phenotype.

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Targeting inflammation in sickle cell disease with fatty acids

sickle cell disease red blood cells
(OpenStax College/Wikimedia Commons)

Painful, tissue-damaging vaso-occlusive crises (a.k.a. pain crises) are one of the key clinical concerns in sickle cell disease (SCD). The characteristic C-shaped red blood cells of SCD become jammed in capillaries, starving tissues of oxygen and triggering searing pain. Over a patient’s life, these repeated rounds of oxygen deprivation (ischemia) can take a heavy toll on multiple organs.

There’s some debate as to why these crises take place—is the sickled cell’s shape and rigidity at fault, or are the blood vessels chronically inflamed and more prone to blockage? Either way, doctors can currently do little to treat vaso-occlusive crises, and nothing to prevent them.

The inflammation view, however, is leading some researchers to ask whether omega-3 fatty acids—which can alleviate inflammation—might be part of the solution. A recent mouse study in the journal Haematologica, led by Mark Puder, MD, PhD, of Boston Children’s Vascular Biology Program, and Carlo Brugnara, MD, of the hospital’s Department of Laboratory Medicine reveals some molecular clues and suggests that human trials of omega-3s might be a good next step.

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How our neutrophils might sabotage wound healing in diabetes

When you get a cut or a scrape, your body jumps into action, mobilizing a complicated array of cells and factors to stem bleeding, keep the wound bacteria-free and launch the healing process.

For most of us, that process is complete in a couple of weeks. But for many people with type 1 and type 2 diabetes, delayed wound healing can have permanent consequences. For example, between 15 and 25 percent of diabetes patients develop chronic foot ulcers. Those ulcers are the root cause of roughly two-thirds of lower limb amputations related to diabetes.

Why don’t these wounds close? Blame a perfect storm of diabetic complications, such as reduced blood flow, neuropathy and impaired signaling between cells. According to research by Denisa Wagner, PhD, of Boston Children’s Hospital’s Program in Cellular and Molecular Medicine, a poorly understood feature of our immune system’s neutrophils may be one more ingredient in the storm.

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Sun Hur, PhD: Overcoming barriers to reveal innate immunity’s secrets

Self-discovery is a theme that unites Sun Hur’s life and work. Growing up with a passion for physics, Hur pursued a scientific career in chemistry before launching her own research group in biology. Today, Hur, an investigator in Boston Children’s Hospital’s Program in Cellular and Molecular Medicine (PCMM), uses her considerable intellectual gifts to uncover how the immune system distinguishes self from non-self.

In the video above, produced by the Vilcek Foundation (which honors and supports foreign-born scientists and artists who have made outstanding contributions to society in the United States), Hur talks about her personal and scientific journey since coming to the U.S. from her native South Korea in 2000. Overcoming cultural and language barriers, she has turned her childhood fascination with order and chaos toward exploring how the innate immune system recognizes invaders, in particular disease-causing viruses that generate a double-stranded RNA during replication.

These studies, which could open doors to new treatments for cancer and inflammatory diseases, recently garnered her the Vilcek’s 2015 Prize for Creative Promise in Biomedical Science.

Adapted from announcements originally published by the Vilcek Foundation and the PCMM.

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