What does 2016 have in store in the realm of science and clinical innovation? Vector asked clinical, digital and business leaders from around Boston Children’s Hospital to offer their forecasts. …
For some pediatric cancers, such as acute lymphoblastic leukemia, older forms of therapy — and older ways of defining who receives which therapy — have served well over the last few decades. But that approach is no longer sufficient. Revolutionary gains have been made in adult oncology using personalized genomic therapy — therapy based on matching treatments to the genetic makeup of a patient’s tumor. The time has come to take them to the pediatric space.
But how will pediatric oncology get there? A panel discussion at Boston Children’s Hospital’s Global Pediatric Innovation Summit about personalized cancer genomics — moderated by Bloomberg News’s John Lauerman and featuring Katherine Janeway, MD, clinical director of the Solid Tumor Center at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center — took on this question. Panelists raised four overarching concepts to consider: numbers, sharing, collaboration and incentives. …
As survival has improved dramatically for children with even the most serious forms of heart disease, neurodevelopmental disabilities have been increasingly recognized. These can affect not only school performance, but also future employment, quality of life and social relationships.
“We’ve known for a while that children with congenital heart disease (CHD) have a higher risk of developmental delays,” says Amy Roberts, MD, a genetic cardiologist at the Boston Children’s Hospital Heart Center. “There are multiple hypotheses as to why that might be, and they’re not mutually exclusive.”
The side effects of surgery, such as oxygen deprivation during bypass, are commonly thought to be to blame. Others suspect problems with the in utero environment. But these factors are not the whole story.
“Even in studies that have measured every known risk factor, only one third of neurodevelopmental disabilities in children with CHD can be explained by factors related to the child’s heart disease, medical history or family factors,” notes Jane Newburger, MD, MPH, director of the Cardiac Neurodevelopment Program at Boston Children’s.
Perhaps there is a genetic component?
In a recent study published in Science, a team of researchers from seven hospitals (Boston Children’s, Brigham and Women’s Hospital, Children’s Hospital of Philadelphia, Columbia, Mount Sinai, Yale and University of California Los Angeles), examined the whole genomes of 1,213 patients with complex CHD, looking for genetic indicators that a child will have developmental delays alongside his or her CHD. …
Children living outside industrialized nations have limited access to health care, and many children with severe kidney dysfunction do not have access to dialysis. Some developing countries have access to manual peritoneal dialysis, which requires the placement of a catheter into the abdominal cavity every one to two hours, 10 hours per day. But supplies are expensive, and many countries lack the infrastructure needed to get large quantities of dialysis fluid to children’s homes.
At the recent 2015 Boston Children’s Hospital Global Pediatric Innovation Summit + Awards, pediatric nephrologist Sara Jandeska, MD, of Rush Children’s Hospital in Chicago, pitched a portable, affordable solution: providing just the dialysis salts.
More than half of all heart murmur referrals to pediatric cardiologists are for a Still’s murmur — a benign murmur that naturally occurs in 50 to 90 percent of children and goes away by adolescence.
Every year, pediatric cardiologists in the United States see 1.3 million children with Still’s murmurs. That adds up to over $400 million in consultation fees alone.
The cardiologist, in turn, may still be unsure whether the murmur is benign after listening to the child’s heart with a stethoscope. He or she might order a follow-up echocardiogram to be certain. If this happens just 10 percent of the time, that’s an additional $200 million in unnecessary costs incurred per year. On top of the financial burden on the healthcare system, the referrals and testing cause unnecessary anxiety for patients and families.
A mobile app in development by Raj Shekhar, MD, of Children’s National Health System and his team has the potential to significantly alleviate these burdens. The app, called StethAid, allows pediatricians to identify a Still’s murmur, thus establishing the child’s murmur as benign and eliminating the need for cardiac referral.
Is there anything more fundamental to human life than the heartbeat? That thud, thud, thud — that reliable rhythm — is synonymous with being alive.
When a person undergoes open-heart surgery, however, the heartbeat must be interrupted to give surgeons access to that essential organ. The organic pulse is temporarily replaced by a machine that provides continuous blood flow to the body.
Doug Vincent, President and CEO at Design Mentor, Inc., has been studying the ways in which current continuous flow devices fail to provide optimal cardio-pulmonary support. Vincent has designed his own support mechanism device that simulates the natural pulsating rhythm of the heart, called VentriFlo.
Some 5 to 17 percent of all children have developmental dyslexia, or unexplained reading difficulty. When a parent has dyslexia, the odds jump to 50 percent. Typically, though, dyslexia isn’t diagnosed until the end of second grade or as late as third grade — when interventions are less effective and self-esteem has already suffered.
“It’s a diagnosis that requires failure,” says Nadine Gaab, PhD, an investigator in Boston Children’s Hospital’s Laboratories of Cognitive Neuroscience.
But a new study led by Gaab and lab members Nicolas Langer, PhD, and Barbara Peysakhovich finds that the writing is on the wall as early as infancy — if only there were a way to read it and intervene before the academic, social and emotional damage is done. …
Spinal muscular atrophy (SMA), a condition affecting one in every 6,000 to 10,000 children, is caused by a defect in a gene called SMN1 — which stands for “survival of motor neuron.” The defect leaves children with too little functioning SMN protein to maintain their motor neurons, which begin wasting away. Muscle strength declines and children eventually develop difficulties eating and breathing.
For Vivienne, whose name means “to live,” that meant being slow to reach motor milestones like crawling, cruising and walking as a toddler. For her parents, it meant hearing that her life expectancy would not be normal.
But a new back-door approach seems to be helping Vivienne, now in first grade, at least thus far.
As it happens, most of us carry a backup gene for SMN1 — namely SMN2. …
Hacking Pediatrics, now in its third year, continues to experiment with its format. 2015’s “Mashup” had a greater focus on partnerships, curation and delivering value to innovators at Boston Children’s Hospital. The brunt of the idea pitching and team formation took place in advance, allowing the event, on November 14, to be collapsed into one day.
The Hacking Pediatrics team (Kate Donovan, Mike Docktor, Meg McCabe, Cassandra Bannos and Leila Amerling) brokered collaborations with a dozen industry partners such as Microsoft, Cerner, Box, CVS Health and Boston Scientific. Over the course of a hectic 12-hour day, they worked with 17 teams of Boston Children’s innovators and experts from partner organizations who presented their final ideas to a panel of judges.
In another change for 2015, the Hacking Pediatrics team issued nine awards — but no immediate prizes. This was meant to incentivize teams to continue to work and meet milestones to earn real rewards, like a $10,000 design prize offered up by design firm Mad*Pow. …