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.
“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.”
Reports from parents and a growing number of studies over the past 10 to 15 years suggest that children with autism spectrum disorder (ASD), especially more severe ASD, are prone to gastrointestinal disorders. Researchers have attributed the association to altered GI microbiota, abnormal intestinal physiology, immune alterations and other mechanisms. Some speculate that the connection results from unusual eating patterns in children with ASD.
Looking at IBD (Crohn’s and colitis) sets the bar a little higher, since IBD is uncommon and also unlikely to be caused by dietary factors (though it can certainly be aggravated by them). In a new study in the journal Inflammatory Bowel Disease, Kohane and colleagues crunched three large databases to create what they believe is the largest ASD/IBD study to date.
Four children with life-threatening malformations of blood vessels in the brain appear to be the first to benefit from 3D printing of their anatomy before undergoing high-risk corrective procedures.
The children, ranging from 2 months to 16 years old, all posed particular treatment challenges: cerebrovascular disease often entails complex tangles of vessels in sensitive brain areas.
“These children had unique anatomy with deep vessels that were very tricky to operate on,” says Boston Children’s neurosurgeon Edward Smith, MD, senior author of the paper and co-director of the hospital’s Cerebrovascular Surgery and Interventions Center. “The 3D-printed models allowed us to rehearse the cases beforehand and reduce operative risk as much as we could. You can physically hold the 3D models, view them from different angles, practice the operation with real instruments and get tactile feedback.”
I think my daughter Esmé is extraordinarily unique—from her tiny pudgy feet that she likes to stuff in her mouth to her beautifully lashed blue eyes and outrageously untamed hair. It’s a mom thing. I guess it is a symptom of loving another person more than life itself.
But my daughter is also unusual in a more scientific way: in her genes.
It’s 1 a.m. on a Wednesday. A two-year-old boy involved in a rollover automobile accident is brought into the emergency department at Boston Children’s Hospital. A scan shows fluid in his abdomen. He is becoming progressively unstable, his blood pressure plummeting despite blood transfusions. A decision is made to bring him to the operating room (OR), where a surgical team performs an exploratory laparotomy for a liver laceration and massive bleeding.
On July 13, 14 and 15, the entire seventh floor of the nearby Longwood Center became a theater, rooms with walls of cardboard became the stage, and hospital staff members became the actors. It was just one of many simulations—complete with cardboard transfusion machines, heart-lung machines and more—intended to help architects design ORs, procedure rooms, recovery rooms and other clinical spaces. These spaces will eventually make up a new 11-story, 445,000-square-foot hospital building. During the week, similar exercises also took place for a planned facility in Waltham.
Growing up in the San Francisco area, Cigall Kadoch, PhD, had a passion for puzzles. The daughter of a Moroccan-born, Israeli-raised father and a mother from Michigan who together developed an interior design business, Kadoch excelled in school and pretty much everything else. Above all, she loved to solve brain-teasers.
In high school, however, Kadoch came up against a problem that defied solution. Breast cancer took the life of a beloved family caretaker who had nurtured her interests in science and nature. She knew little about cancer except that it took lives far too early.
“I was deeply saddened and very frustrated at my lack of understanding of what had happened,” recalls Kadoch. “I thought to myself, cancer is a puzzle that isn’t solved, let alone even well-defined, and I want to try. As naïve a statement as that was, it was a defining moment—one which I never could have predicted would actually shape my life’s efforts.”
Funding drives biomedical research, and research drives treatment innovation. Access to funds, particularly National Institute of Health (NIH) awards, is critical to move research forward. The 21st Century Cures Act, which passed the U.S. House on July 10, could give the NIH $8.75 billion more in new grants to disperse over the next five years, the largest increase since the Recovery Act of 2009.
How would those funds be used? Can research find a better way to treat patients? Prevent disease? Disseminate advances in medicine?
In 2014, Boston Children’s led the U.S. in NIH awards. Here’s a look at how a few research teams are leveraging NIH funding to improve care for both children and adults.
Bone marrow transplantation, a.k.a. stem cell transplantation, can offer a cure for certain cancers, blood disorders, immune deficiencies and even metabolic disorders. But it’s a highly toxic procedure, especially when a closely matched marrow donor can’t be found. Using stem cells from umbilical cord blood banked after childbirth could open up many more matching possibilities, making transplantation safer.
But what if the blood stem cells in those units could be supercharged to engraft more efficiently in the bone marrow and grow their numbers faster? That’s been the quest of the Zon lab for the past seven years, in partnership with a see-through zebrafish called Casper.
Hypoplastic left heart syndrome (HLHS) is a rare but serious form of congenital heart disease that leaves the left pumping chamber (ventricle) of the heart severely underdeveloped. Children born with HLHS can’t pump enough oxygenated blood from their heart to the rest of their body and need surgery as soon as possible to survive. Treatment ultimately involves three corrective surgeries throughout the infant and toddler years.
The first surgery, known as the Norwood procedure, is the riskiest of the three. Ideally performed within the first week of life, the procedure re-routes the heart’s plumbing to ensure enough oxygenated blood is circulated while the child grows big enough for the second surgery. A device called a graft is used to connect the fully-functional right ventricle to the aorta, bypassing the stunted left ventricle, for proper blood flow. However, with each ventricular contraction, the graft gets squeezed, which can cause it to shift or lose its shape over time. Repeat interventions to adjust the graft are often needed.
Hospitals are among the most hazardous workplaces in the U.S. In 2011, according to the Occupational Safety and Health Administration, 253,700 accidents were reported, an average of 6.8 work-related injuries for every 100 full-time employees. Rates of injuries reported to OSHA are decreasing in all industries except for hospitals, whose rates are double the average.
Could a set of digital apps help identify and reduce occupational and environmental risks in a quick and efficient manner? That is what Nick Kielbania, MS, CSP, CHMM, director of Environmental Health & Safety (EH&S) and Adrian Hudson, PhD, MCompSc, principal software architect at Boston Children’s Hospital, set out to create.
Their web-based solution, enabled for Apple and Android devices, is called the BCH Environmental Health and Safety Application Suite. Designed to aid hospital emergency response, safety and support services, the applications encompass fire, clinical, research, construction and environmental safety, with additional apps for on-call and administrative personnel.