Illustration: David Roberson
This post originally appeared in longer form on Harvard Medical School’s news site. Try not to scratch when you read it.
There’s itch, and then there’s itch.
New research has revealed distinct sets of itch-generating neurons that explain why current itch therapies often fail. It also suggests new ways to selectively silence itch.
“We think this [research] has therapeutic implications,” says Clifford Woolf, PhD, director of the F.M. Kirby Neurobiology Center at Boston Children’s Hospital and professor of neurology at Harvard Medical School (HMS).
While itch is more aggravating than life-threatening, Woolf and HMS graduate student David Roberson hope their work might one day ease the torment itch can cause, particularly in children.
“If you go into the pediatric immunology wards, you see little kids with their hands in mittens or sometimes tied down because they scratch themselves to a point where they damage themselves,” says Woolf. Full story »
Could diabetes be treated without insulin shots? (Tess Watson/Flickr)
For decades, patients have managed their type 1 diabetes by injecting themselves with insulin to regulate the glucose in their blood. While this form of medical management addresses the immediate danger of low insulin levels, long-term complications associated with diabetes, like heart and kidney diseases, still threaten more than 215,000 children currently living with the disease in the United States.
“Insulin injections can manage hyperglycemia by reducing the patient’s glucose levels, but it is not the cure,” says Paolo Fiorina, MD, PhD, of the Nephrology Division at Boston Children’s Hospital.
Fiorina is currently involved in new research targeting a molecular pathway that triggers diabetes in the first place—potentially providing a permanent cure. It could potentially change the face of diabetes treatment in children. Full story »
Interior of a bladder (Gray's Anatomy of the Human Body)
Ask a group of 15-year-olds what’s on their minds, and you’ll likely hear something along the lines of video games, parties and maybe homework. Ask Adrian Haber, a sophomore at Boston Latin School, and you’ll hear something surprising: nanoparticles and bladder spasms.
Under the mentorship of urologist Hiep Nguyen, MD, at Boston Children’s Hospital, Haber has pioneered a new drug delivery system for bladder spasm medication. Nguyen believes Haber’s work has laid the groundwork for what may become a safer, more effective alternative to existing drug therapies.
The student and doctor began their partnership in November 2012; Haber, already a veteran of the Boston Regional Science Fair, was looking for a new project—one that would combine his interests in physics and biology. His mother Constance Houck, MD, an anesthesiologist at Boston Children’s, knew just the person to ask. And Nguyen had a problem that was long overdue for a solution. 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 »
Just like Goldilocks wouldn’t eat porridge that was too hot or too cold, blood vessels won't grow properly in tissues that are too stiff or too loose. (Project Gutenberg/Wikimedia Commons)
In the tale
Goldilocks and the Three Bears, Goldilocks tries all of the bears’ porridge, chairs and beds, finding that only the little bear’s things were just right. Everything else was a little off for her…too hot or too cold, too hard or too soft and so on.
Similarly, for everything to work as it should in the body, things need to be just right. Blood pressure shouldn’t be too high or too low; organs can’t be too big or too small, etc.
Donald Ingber, MD, PhD, and his lab in Boston Children’s Vascular Biology Program take this “just right” approach when thinking about how organs and tissues are structured. Recently, he and a member of his research staff, Akiko Mammoto, MD, PhD, discovered that by changing the stiffness of the surrounding tissues—not too loose and not too tight— they could keep blood vessels from leaking. Their finding could have real consequences for people with sepsis or other diseases featuring leaky vessels. Full story »
There are a couple of ways by which aspirin might affect cancer. (cpradi/Flickr)
Aspirin does a remarkable number of things in the body, enough that it’s said it would never win approval today from the Food and Drug Administration as an over-the-counter drug.
But among those functions are some that may explain something that doctors have recognized for some time: patients with cancer who have been taking aspirin tend to have better outcomes. Full story »
In mice, boosting amounts of a microRNA family called miR-17-92 led to dramatic enlargements of embryonic and postnatal hearts, with thicker ventricle walls.
Challenging accepted wisdom about the heart, Boston Children’s Hospital cardiologist
Bernhard Kühn, MD, recently showed that infants, children and adolescents
are capable of generating new heart muscle cells, or cardiomyocytes. That work raised the possibility that scientists could stimulate regeneration to repair injured hearts.
Now, we have a potential therapeutic target to accomplish this: a family of microRNAs called miR-17-92 that regulates cardiomyocyte proliferation. In Circulation Research earlier this month, a team led by Kühn’s research colleague Da-Zhi Wang, PhD, demonstrates its potential. Full story »
Leonard Zon (top) and Massachusetts Lt. Governor Timothy Murray in the Stem Cell Program's zebrafish facility. (Courtesy MLSC)
Ed. Note: Leonard Zon, MD, is founder and director of the Boston Children’s Hospital Stem Cell Program, which yesterday was awarded $4 million by the Massachusetts Life Sciences Center to build the Children’s Center for Cell Therapy.
As a hematologist, I see all too many children battling blood disorders that are essentially untreatable. Babies with immune deficiencies living life in a virtual bubble, hospitalized again and again for infections their bodies can’t fight. Children disabled by strokes caused by sickle cell disease, or suffering through sickle cell crises that drug treatments can’t completely prevent. Children whose only recourse is to risk a bone marrow transplant—if a suitably matched donor can even be found.
Over the past 20 years, my lab and that of George Daley, MD, PhD, at Boston Children’s Hospital have worked hard to give these children a one-time, potentially curative option—a treatment that begins with patients’ own cells and doesn’t require finding a match. Full story »
