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.
Sepsis is the most common cause of death in infants and children worldwide, and its incidence is increasing. Damage is caused not only by the bloodstream infection itself but by the systemic inflammatory cascade it triggers — which has been difficult to control without also causing long-lasting immune suppression. During a five-minute Ignite Talk at the 2015 Boston Children’s Hospital Global Pediatric Innovation Summit + Awards, Brian McAlvin, MD, a critical care intensivist at Boston Children’s Hospital, introduced the audience to a filtration technology that could cure systemic inflammatory response syndrome (SIRS).
SIRS, McAlvin noted, is the underlying mechanism for a variety of diseases, not just sepsis. His invention, the Antibody Modified Conduit, is essentially a small tube with antibodies painted on the inner surface that recognize and remove the inflammatory agents. “This technology allows us to choose the inflammatory molecules in the circulation,” says McAlvin, “and take them out of the blood as the condition evolves by changing the antibody that’s present.”
The talk won the pitch competition, earning McAlvin an Apple watch, a one-on-one mentoring session with an influential venture capitalist and a meet-and-greet with Boston Children’s innovation acceleration team, VCs and other stakeholders.
See more posts and videos from the Global Pediatric Innovation Summit.
The global market for pain medications is huge — some estimates predict it will hit $41.6 billion by 2017. However, the costs of pain medicine development are huge, too; it takes roughly $900 million to bring a new analgesic compound to market. In part, this is because some 80 percent of compounds that look promising in preclinical animal studies (largely in rodents) fail in late-stage clinical trials.
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.
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.
Tears of the knee’s anterior cruciate ligament — or ACL — are on the rise in middle school and high school athletes. The current treatment involves grafting in a piece of tendon from elsewhere in the body. It works very well, but requires six months to two years of post-op rehabilitation to regain strength in the knee and the place where the tendon was taken from (often the hamstring). Plus, up to 80 percent of patients develop arthritis within 15 years of the procedure.
Orthopedic surgeon Martha Murray, MD, wondered, “What if we could somehow stimulate the original ACL to heal back together?” …
“Precision medicine” looks to be heading down the same path as “big data” and “innovation”: The term is becoming so widely used that it threatens to detract from the real impact it is already having in patients’ lives.
But for children, who are still developing and have the most to gain, precision medicine is more than a bumper sticker. On the micro scale, early genetic testing—perhaps routinely, someday, in newborns—can help guide medical care, targeted therapies and preventive strategies based on a child’s genetic makeup. On a macro scale, big data from the larger population becomes a predictive tool, guiding medical decisions that could be life-altering in a still-malleable child.
“If you can make an early diagnosis, you can amplify the effects of what you do, rather than try to change the highways once they’re built,” said Wendy Chung, MD, PhD, of Columbia University Medical Center during a panel discussion last week at Boston Children’s Hospital’s Global Pediatric Innovation Summit + Awards (#PedInno15). …
What happens when you put a doctor who specializes in cystic fibrosis in the same room as two biotech executives, one of whom is a ‘dadvocate’ of a teenager with CF? View the highlights and reactions to a a dynamic panel discussion at the Boston Children’s Hospital Global Pediatric Innovation Summit + Awards with Gregory Sawicki, MD, MPH, director of the Boston Children’s Cystic Fibrosis Center; David Meeker, MD, Genzyme president and CEO; Bob Coughlin, Massachusetts Biotechnology Council president and CEO; and moderator Luke Timmerman, founder and editor of The Timmerman Report.