Studies pinpoint risk factors for complications of pediatric intestinal failure
Pediatric intestinal failure occurs when patients lack the amount of intestine necessary for growth and development. As advances in treatment have led to better survival rates for children with short bowel syndrome and other forms of intestinal failure, the team at Boston Children’s Center for Advanced Intestinal Rehabilitation (CAIR) is studying factors that affect quality ... Read More about Studies pinpoint risk factors for complications of pediatric intestinal failure
Tackling an aggressive, treatment-resistant lymphoma where it lives
Anaplastic large cell lymphoma, a form of non-Hodgkin lymphoma, is the most common aggressive lymphoma in children. Chemotherapy and radiation fail to cure about 30 percent of cases. When tumors are driven by the oncogene ALK — which is the case for the majority of children — kinase inhibitor drugs like crizotinib are very effective ... Read More about Tackling an aggressive, treatment-resistant lymphoma where it lives
One-time treatment could block a deadly form of graft-versus-host disease
Even when a bone marrow transplant cures leukemia or lymphoma, patients can still pass away from graft-versus-host disease (GVHD), in which T cells in the donor graft attack the recipient’s own tissues. Leslie Kean, MD, PhD, director of stem cell transplant at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, has long sought to prevent this ... Read More about One-time treatment could block a deadly form of graft-versus-host disease
From bench to bedside: A promising option for unremitting ulcerative colitis
Many existing treatments for inflammatory bowel disease, like Remicade® and Humira®, work by blocking inflammatory cytokines such as tumor necrosis factor (TNF) and interleukins IL-12 and IL-23, which are over-produced in autoimmune disease. But few patients with ulcerative colitis get complete relief from these drugs. A small but promising open-label clinical trial, published recently in ... Read More about From bench to bedside: A promising option for unremitting ulcerative colitis
Perfecting the craft of modeling disease in stem cells: Dosh Whye
Part of an ongoing series profiling researchers at Boston Children’s Hospital. Dosh Whye has always wanted to make peoples’ lives better, but he never imagined that tending to stem cells in a lab seven days a week would be the way he would do it. Now, as an assistant director of the Human Neuron Core ... Read More about Perfecting the craft of modeling disease in stem cells: Dosh Whye
Seeking a path forward for custom genetic treatments
In 2018, at age 7, Mila Makovec became the world’s first person to be treated with a drug made just for her. Languishing from Batten disease, a rare, fatal neurodegenerative disorder, she received an antisense oligonucleotide (ASO) drug designed to silence her genetic mutation, injected into the fluid around her spine through a lumbar puncture. ... Read More about Seeking a path forward for custom genetic treatments
Facial weakness: A dark matter detective story
Elizabeth Engle, MD, has devoted her career to finding genetic and developmental causes for disorders of eye, eyelid, and facial movement. From common conditions like strabismus to very rare disorders, these conditions can impact a person’s appearance and impair social communication, making it hard to shift one’s eyes up, down, or sideways or adjust facial expressions. Each ... Read More about Facial weakness: A dark matter detective story
Finding a way to help newborns who can’t immediately have heart treatment
Newborns with complex congenital heart defects (CHD) and pulmonary overcirculation often need treatment as soon as possible. Unfortunately, some of these children are not in good enough health to withstand surgery. To address this challenge, Boston Children’s heart specialists leaned into technological innovation, their experience, and a perseverance that would ultimately confirm their belief that even the ... Read More about Finding a way to help newborns who can’t immediately have heart treatment
Taming vaccine data: Joann Arce, PhD
Part of an ongoing series profiling researchers at Boston Children’s Hospital. Joann Arce, PhD, is a data tamer — corralling and wrangling vast quantities of data to extract insights on how our immune systems react to vaccines and infections. Her work is paving a path toward smarter, more potent vaccines. As lead of data management ... Read More about Taming vaccine data: Joann Arce, PhD
Female athletes and sports injuries: Psychology matters
If the goal of sports medicine is to promote sports participation, the state of an injured athlete’s musculoskeletal system is part of a larger puzzle. In fact, a growing body of research suggests that psychological factors also play a significant part in how athletes recover and if they return to sports. Consider anterior cruciate ligament ... Read More about Female athletes and sports injuries: Psychology matters