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
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
Another angle on Alzheimer’s: CSF, proteomics, and metabolic enzymes
Currently there are no objective, easily assessed diagnostic markers for Alzheimer’s disease, and no good therapeutic options. Taking an agnostic approach, proteomics expert Hanno Steen, PhD, and neurobiologist Judith Steen, PhD, who share a lab at Boston Children’s Hospital, teamed up to analyze proteomics data from the cerebrospinal fluid (CSF) that bathes the brain, combining ... Read More about Another angle on Alzheimer’s: CSF, proteomics, and metabolic enzymes
Could we make blood anywhere in the body?
Our bodies make blood in a specialized niche — a “nursery” within our bone marrow that nurtures blood stem cells so they can replicate and make different kinds of blood cells. The lab of Leonard Zon, MD, has even shown how blood stem cells, once they settle in the niche, are “cuddled” by nearby cells. ... Read More about Could we make blood anywhere in the body?
A new cancer mechanism: Failed cellular housekeeping
Cancer can stem from mutations in many different genes. New research from Boston Children’s Hospital and Dana-Farber Cancer Institute pinpoints a gene that, when mutated, causes cancer through a mechanism not before seen: Inability of cells to dispose of their trash, namely defective strands of RNA. This mechanism appears to cut across many different malignancies, ... Read More about A new cancer mechanism: Failed cellular housekeeping
An unexpected journey reveals a potent way to attack tumors
Research on the effects of prenatal exposure to the Zika virus has yielded an unexpected dividend: a potentially promising way to trigger natural killer (NK) cells to fight cancer. NK cells are first-responder immune cells. When enough of their activating receptors are triggered, they mobilize to kill infected, stressed, or cancerous cells at an early ... Read More about An unexpected journey reveals a potent way to attack tumors
Conquering a rare metabolic condition: A family, a pediatrician, and two labs join forces
As a newborn, Sam Hoffman never cried or made a sound. His mother, Carolyn, often had to wake him up to feed him. He missed many of his infant milestones. At one visit, his pediatrician tapped his leg and couldn’t get a reflex. A urine test found extremely high levels of 4-hydrobutyric acid or GHB ... Read More about Conquering a rare metabolic condition: A family, a pediatrician, and two labs join forces
Making ionocytes: A step toward cell or gene therapy for cystic fibrosis
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators were a breakthrough for cystic fibrosis, improving the movement of chloride and water and moistening mucus secretions. But these drugs are expensive, don’t work in all patients with cystic fibrosis, and have side effects and interactions with other drugs. People who do respond to CFTR modulators must take ... Read More about Making ionocytes: A step toward cell or gene therapy for cystic fibrosis
Study shows young adults don’t easily transition to self-care of diabetes
Diabetes is challenging to manage at any age, but even more so for young adults who are handling the condition on their own for the first time. They need to transition from pediatric to adult care as smoothly as possible — but a recent study finds this isn’t happening. Research led by Katharine Garvey, MD, MPH, ... Read More about Study shows young adults don’t easily transition to self-care of diabetes