A Queens College gymnasium served as an evacuation center after Hurricane Sandy.
Shannon Manzi, PharmD, chief pharmacist for the Massachusetts–1 Disaster Medical Assistance Team, directs the Clinical Pharmacogenomics Service at Boston Children’s Hospital and is team leader for Emergency and Combined Services in the hospital’s Department of Pharmacy. With MA-1 DMAT, she has deployed to Louisiana after Hurricane Katrina in 2005 and Hurricane Gustav in 2008 and to Haiti after the 2010 earthquake.
As I watch the Arizona-1 and Texas-3 Disaster Medical Assistance Teams (DMATs) respond to the tornado in Moore, Okla., I know they will serve with great skill and caring. But I wish the Massachusetts-1 DMAT was the team on call this month. Although we’re unlikely to be deployed for this disaster, our hearts are with the people of Moore and all our fellow responders.
Manzi
Thirteen years ago, I was asked to join the MA-1 DMAT as the pediatric pharmacist. It’s been one of the most grueling and difficult commitments of my life, but I’ve never looked back. I love it.
I have slept for weeks on the ground, not being able to shower or eat anything other than MREs (meals-ready-to-eat)—all while working 18- to 20-hour days. However, I hold no illusions that what we do is heroic. I can go home in two to three weeks to an intact house and family. This is not the case for the people we serve. Full story »
In the developing world, health care providers often don’t have access to diagnostic technologies like the automated lab tests taken for granted in the resource-rich United States. Specimens often have to be sent to a distant central lab, and it can be weeks before an answer wends its way back.
That’s a tough situation when you’re, say, trying to assess whether a patient is having liver toxicity from a drug, such as drugs used to treat tuberculosis (TB) and HIV. By the time the results come back and indicate you need to stop or switch medications, the patient may be long gone, unable to travel back to the clinic.
For the past four years, Nira Pollock, MD, PhD, associate medical director of the Infectious Diseases Diagnostics Lab at Boston Children’s Hospital, has been working with Diagnostics For All (DFA), a nonprofit organization based in Cambridge, Mass., to develop and test a low-cost diagnostic device that works on the spot, involving just a finger-stick and a square of paper. The technology is all in the paper square—using wax printing and microfluidics techniques Full story »
Engaging clinicians in change will require a cultural shift. (David Oliva/Wikimedia Commons)
Alyssa Bianca Velasco, ScB, is a clinical data specialist for the Standardized Clinical Assessment and Management Plans (SCAMPs) program at Boston Children’s Hospital.
Reducing health care costs doesn’t have to involve making sacrifices in patient safety or quality of care or holding clinicians to rigid guidelines. Over the past several years, Boston Children’s Hospital has rolled out a methodology known as Standardized Clinical Assessment and Management Plans (SCAMPs). Described in the May issue of Health Affairs, SCAMPs are based on the idea that clinicians should be able to diverge from established medical best practices, provided they document the reasons and track the results—in essence making continual data-driven modifications to practice.
The success of SCAMPs in reducing practice variability and costs and improving outcomes at Boston Children’s has led other institutions, one by one, to adopt them. In the next phase, we plan to expand SCAMPs much more broadly, creating a network of hospitals that will pool pertinent clinical data into a centralized non-profit institution, the Institute for Relevant Clinical Data Analytics (IRCDA).
I am part of a team that is providing training, analytics and IT support to help make that large-scale implementation happen. Full story »
Psyché et l’Amour, François Gérard, 1798
Maude Tessier, PhD, is assistant director of business development and strategic initiatives in the Technology and Innovation Development Office at Boston Children’s Hospital. Her role is to initiate, develop and realize alliances between Boston Children’s and industry partners. She tweets from @maude_tessier.)
I log on to the Web portal with excitement and set up my profile. I browse for potential matches, reading though all their interests to see if they match my own. I send out requests to meet face to face. I wait. Have I received favorable responses? Were my short email invite and profile enticing enough? Is my dance card getting full?
It’s not a dating website, but rather the prelude to a biotech business partnering conference. In my role as a leader of business development and marketing efforts at Boston Children’s Technology and Innovation Development Office, my objective is to quickly and effectively pitch our most promising work to industry contacts, in hopes of continuing conversations after the conference is over. Attending these conferences is a great way to “break the ice”—and it is key to my success in building relationships and developing partnerships and alliances with life sciences companies.
I liken it to speed and online dating combined. Full story »
Joshua Frase, who died from X-linked myotubular myopathy (MTM), with his father, Paul Frase, in 2006.
Part 2 of a two-part series. (Read part 1.)
Back in the 1990s, rheumatologist Richard Weisbart, MD, of University of California, Los Angeles (UCLA), was studying lupus in a mouse model and found that the mice were making an antibody that had the intriguing ability to get inside tissues and cells.
Weisbart shifted his work away from studying lupus to studying and refining the antibody, called 3E10, and he and others showed that proteins could be delivered into different tissues of the body simply by attaching them to a fragment of 3E10.
Dustin Armstrong, PhD, a postdoc at Novartis at the time, was trying to find molecules that could activate growth in weakened muscles—without activating possibly cancerous growth in other tissues. He saw Weisbart’s work and contacted UCLA. In 2008, he obtained seed money and founded a company around 3E10-based therapeutics for muscular diseases, now known as Valerion Therapeutics (formerly 4s3 Bioscience).
“There’s a huge need for therapies for genetic muscle diseases, and muscle was a tissue we could target well with our technology,” says Armstrong. Full story »
Will Ward at the NSTAR Walk for Boston Children’s Hospital in 2012—his family’s fifth year leading a team to raise funds for the Beggs Laboratory.
This two-part series examines two potential treatment approaches for myotubular myopathy, a genetic disorder that causes muscle weakness from birth.
Sixth-grader William Ward cruises the hallways at school with a thumb-driven power chair and participates in class with the help of a DynaVox speech device. Although born with a rare, muscle-weakening disease called X-linked myotubular myopathy, or MTM, leaving him virtually immobile, he hasn’t given up.
Neither has Alan Beggs, PhD, who directs the Manton Center for Orphan Disease Research at Boston Children’s Hospital, and who has known Will since he was a newborn in intensive care.
“From the very beginning, Alan connected with our family in a very human way,” says Will’s mother, Erin Ward. “In the scientific community, he’s been the bridge and the connector of researchers around the world. That makes him unique.”
Since the 1990s, Beggs has enrolled more than 500 patients with congenital myopathies from all over the world in genetic studies, seeking causes and potential treatments for congenital myopathies—rare, often fatal diseases that weaken children’s skeletal muscles from birth, often requiring them to breathe on a ventilator and to receive food through a gastrostomy tube. Full story »
Advances in medical care sometimes present challenges on the flipside. Case in point: Over the past three decades, progressive developments in pediatric cardiac care have allowed many babies born with congenital heart disease (CHD) to survive. And longevity continues to improve. This progress, however, has brought hospitals a burgeoning patient population with tremendously complex and varied disease states.
About 90 percent of children born with heart defects now survive to adulthood, thanks to diagnostic, interventional and critical care improvements. Specifically, one-year survival has improved from 67.4 percent from 1979 to 1993, to 82.5 percent from 1994 to 2005.
“The number of pediatric hospital admissions for congenital heart disease is increasing relatively slowly, but as the patients live longer and develop common adult medical issues, adult patient admissions are increasing much more rapidly,” says Alexander Opotowsky, MD, MPH, cardiologist at the Boston Adult Congenital Heart (BACH) and Pulmonary Hypertension services at Boston Children’s Hospital. Full story »
(Scott Foresman/Wikimedia Commons)
In the aftermath of the Boston Marathon bombings, first responders did whatever they could to help victims. For many of those injured, tourniquets proved to be the difference between saving and losing a limb—or a life.
“There’s no doubt that tourniquets played a key role in treating the bombing victims,” says Boston Children’s Hospital Trauma Center Director David Mooney, MD.
Two children who were later treated at Boston Children’s had tourniquets applied at the site of the tragedy. One arrived with extensive lacerations caused by one of the two detonated bombs. The other was in worse condition, having suffered blood vessel damage among other problems. Both children are doing better, although one will require further treatment.
Dating back to Roman times, a simple tourniquet, encircling a limb just above a wound, was the go-to method to stop bleeding. Since then, tourniquets have been used on the battlefield and in emergency rooms and operating rooms. However, had the bombings taken place 10 or 15 years ago, those wounded might not have been treated with tourniquets, Mooney believes. Full story »
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Lin28, a known player in cancer, is hard to suppress with drugs. But two related enzymes present highly druggable targets. (Emw/Wikimedia Commons)
Two fundamental processes in biology—stem cell generation and carcinogenesis—are turning out to be closely intertwined. The lab of
Richard Gregory, PhD, has been teasing out this relationship at the molecular level.
In 2008, Gregory and his colleagues showed how a factor called Lin28, which is associated with numerous cancers, makes a cell more prone to revert to a less specialized, stem-like state.
Lin28 acts by preventing maturation of Let-7—an ancient family of microRNAs found in creatures from humans to worms. Let-7 is the yin to Lin28’s yang: it causes stem cells to differentiate (embryonic stem cells, which are completely unspecialized, have very low levels of it). If a cell’s Let-7 can’t mature, it can’t differentiate; instead, it remains stem-like and can potentially become cancerous.
Suppressing Lin28 with RNA interference (RNAi) has been shown to suppress tumor growth. But Lin28 is difficult to target with drugs. Full story »
(Jerome Gerrior Racing/Flickr)
In the hours and days following the Boston Marathon bombings, the first concern for the victims was literally life and limb—stabilizing the survivors and treating wounds suffered in the blasts.
But as the survivors begin the road to recovery—a road that promises to be long and complicated—subtler effects of the blast may become apparent, including traumatic brain injuries (TBIs).
“The difference between traumatic brain injuries and the other injuries we’ve seen is that the extent of other injuries can be readily seen,” says Mark Proctor, MD, a neurosurgeon and director of Boston Children’s Brain Injury Center. “You can have a traumatic brain injury without any external signs.”
TBIs have been a major concern among soldiers serving in war zones like Iraq or Afghanistan who have experienced the concussive force of bomb or improvised explosive device (IED) explosions—not unlike the explosions on Marathon Monday. Full story »
