Daniel Kohane of Boston Children's Hospital is developing drug delivery technologies that rely on nanoparticles and the spectrum of light.Getting drugs where they need to be, and at the right time, can be more challenging than you think. Tumors, for example, tend to have blood vessels that are tighter and twistier than normal ones, making it hard for drugs to penetrate them. Despite decades of research on antibodies, peptides and other guidance methods, drug makers struggle to target drugs to specific tissues or cell types.

And even once a drug arrives at the right place, the ability to fine-tune the dose so that the drug is released at the right time and in the right amount remains an elusive goal.

What’s needed is some kind of trigger, a stimulus that a clinician can turn on and off to guide when a drug is available and where it goes to make sure it does its job with the fewest side effects.

Daniel Kohane, MD, PhD, a critical care specialist and director of the Laboratory for Biomaterials and Drug Delivery at Boston Children’s Hospital, thinks he’s hit upon a promising trigger, one that’s all around us: light. Full story »

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Alprolix FDA approval recombinant factor IX rFIXFc hemophilia B coagulation

(Courtesy Biogen Idec)

A few weeks ago Vector brought you the backstory of how a clotting factor for hemophilia was made to last longer in the blood, allowing injections to be pared to once every week or two, rather than two to three per week.

Today we bring more good news: Following a successful Phase III trial, rFIXFc recently received the green light for marketing from the FDA and from Health Canada.

Developed by Biogen Idec under the trade name Alprolix™, rFIXFc—a modified version of clotting factor IX—is the fruition of a technology first envisioned by three researchers—gastroenterologists Wayne Lencer, MD, of Boston Children’s Hospital, and Richard Blumberg, MD, of Brigham and Women’s Hospital, and immunologist Neil Simister, DPhil, of Brandeis University—for large protein drugs. Their idea: to extend the drugs’ half-lives by protecting them from being ground up by cells. Full story »

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Ibeziako,PatriciaPatricia Ibeziako, MD, directs the Boston Children’s Hospital Global Partnerships for Psychiatry Observership Program and the Psychiatry Consultation Service at Boston Children’s Hospital.

Children and adolescents constitute almost a third of the world’s population—2.2 billion individuals—and almost 90 percent live in low-income and middle-income countries, where they form up to half of the population. Yet, for many years, child mental health has largely been glossed over—with long-term negative effects on educational attainment in addition to chronic disability and lost productivity.

Major international non-governmental organizations and United Nations agencies work in settings where children are at risk for mental health difficulties. However, with the exception of the World Health Organization (WHO), these agencies often fail to acknowledge or focus on child mental health issues. In 2005, the WHO Atlas of Child and Adolescent Mental Health Resources reported that less than one third of 66 countries surveyed had an entity with sole responsibility for child mental health programming, and that national budgets rarely had identifiable funding for child mental health services. Full story »

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Cameron with Galina Lipton, MD

By the time Cameron Shearing arrived at the South Shore Hospital Emergency Department (ED) during a December snowstorm, he wasn’t breathing. He didn’t have much time. The two-year-old had aspirated a chocolate-covered pretzel, which sent tiny bits of material into his lungs.

The odds of a good outcome were not high. Pretzel is one of the worst foods to aspirate for two reasons: The small pieces can block multiple small airways, and the salt, which is very irritating, causes a lot of inflammation.

“Cameron was one of the sickest patients I ever cared for as an emergency physician. I did everything I could within my scope of practice, but he needed the tools and expertise of pediatric subspecialists,” recalls Galina Lipton, MD, from Boston Children’s Department of Emergency Medicine, who was staffing the South Shore Hospital emergency room that evening. Full story »

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Last week, Boston Children’s Hospital’s Innovation Acceleration Program hosted a jam-packed Innovators’ Showcase where teams from around the hospital networked, traded ideas and showed off their projects. Here are a few Vector thinks are worth watching.

isotropic diffusion reveals information on axons on DTI1. An imaging ‘biomarker’ after concussion

Thirty percent of people who suffer a mild traumatic brain injury—a.k.a. concussion—have ongoing symptoms that can last months or years. If patients at risk could be identified, they could receive early interventions such as brain cooling and anti-seizure medications. New MRI protocols that can measure free, non-directional diffusion of water, coupled with sophisticated analytics, are achieving unprecedented pictures of what happens inside the brain after injury. Full story »

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Google Glass worn by Alex Pelletier croppedAlexandra Pelletier is a manager in the Innovation Acceleration Programat Boston Children’s Hospital. She directs the FastTrack Innovation in Technology Program, a hospital initiative to accelerate, rapidly develop and deliver innovative clinical software solutions.

Do you know the feeling of opening a new box with technology in it? I’m not a tech geek, but when my Google Glass arrived, with its crisp and simple packaging, my visceral reaction was “this is really cool.” Nonetheless, I’m approaching Glass carefully, because even the best technologies still require humans to use them. That means that they must be easy to use, must connect with other systems seamlessly and must offer value that makes its adoption worthwhile.

Google Glass is gaining some real excitement in health care. Each day my Twitter feed lights up with a new report of a hospital or practice trying it out. Here at Boston Children’s, we too are investigating the use of this technology through the Google Glass Explorer Program (watch Vector for more to come). We see promising potential for Google’s head-mounted display technology to transform communication and access to real-time information. Full story »

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Stethoscope and keyboard health insurance claims dataThe only time most of us ever look at an insurance claim is after a hospital or doctor visit, when we get a claim summary from our carrier. And then as far as we know, it gets filed away, never again to see the light of day.

But there’s a lot to be learned from these claims data.

As with electronic medical records (EMRs), behind every claim an insurer receives is a detailed record about symptoms, tests, diagnosis and treatment. Properly compiled and analyzed, claims data can be an excellent resource for taking population-level snapshots of disease, helping to identify trends and reveal or probe associations.

That’s why claims data recently caught the eye of Kenneth Mandl, MD, MPH, and Mei-Sing Ong, PhD, two researchers in Boston Children’s Informatics Program (CHIP). Using claims records for roughly 2.5 million Americans, they turned their attention to two conditions—epilepsy and asthma—with interesting results. Full story »

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Ubiquitin RIG-I innate antiviral immunity Sun Hur

Ubiquitin (pink ovals) doesn't just tag proteins for recycling. It also may help keep our antiviral immune response in balance. (Image courtesy: Sun Hur)

If you follow cancer biology, then you’ve probably heard of ubiquitin before. Ubiquitin tags a cell’s damaged or used proteins and guides them to a cellular machine called the proteasome, which breaks them down and recycles their amino acids. Proteasome-blocking drugs like Velcade® that go after that recycling pathway in cancer cells have been very successful at treating two blood cancers—multiple myeloma and mantle cell lymphoma—and may hold promise for other cancers as well.

Less well known, however, is the fact that ubiquitin helps normal, healthy cells raise an alarm when viruses attack. Ubiquitin works with a protein called RIG-I, part of a complex signaling pathway that detects viral RNA and triggers an innate antiviral immune response.

Sun Hur, PhD, a structural biologist in Boston Children’s Hospital’s Program in Cellular and Molecular Medicine, has been studying RIG-I and other members of the innate cellular antiviral response for some time. And in a recent paper in Nature, she provided a structural rationale for how ubiquitin helps RIG-I do its job, and how that might help keep our immune system from getting out of hand.

Full story »

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cancer genomicsIn 2012, Boston Children’s Hospital held the international CLARITY Challenge—an invitation to interpret genomic sequence data from three children with rare diseases and provide a meaningful, actionable report for clinicians and families. (Click for more background on the children, findings and winners.)

The full proceedings, published March 25 in Genome Biology, concluded that while the technical approaches were markedly similar from center to center, the costs, efficiency and scalability were not. Most variable, and most in need of future work, was the quality of the clinical reporting and patient consenting process. The exercise also underscored the need for medical expertise to bring meaning to the genomic data.

That was CLARITY 1. CLARITY 2, focusing on cancer genomics in children, promises to be exponentially more complex. Full story »

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A clinician's-eye view of a patient with spinal muscular atrophy during a telemedicine visit.

A clinician's-eye view of a patient with spinal muscular atrophy during a telemedicine visit.

The jury is still out on telemedicine. Proponents and many patients appreciate its ability to deliver virtual patient care and to extend the reach of experts beyond the brick-and-mortar setting of a hospital. But the real question about telemedicine is: Does it make it difference? Does is it improve care and if so, in what circumstances?

TeleCAPE, a small pilot project at Boston Children’s Hospital, inches the dial toward “yes” for some patients—in particular, home-ventilated patients.

Home-ventilated patients require an inordinate amount of health care resources for even minor conditions. Costs for a simple urinary tract or viral respiratory infection that might be managed without hospitalization can reach up to $83,000 because the child’s complex medical needs require ICU admission. Full story »

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