Archive for Nancy Fliesler
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
Tagged: cancer, cellular and molecular medicine, lymphoma
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
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
Tagged: genetics and genomics, neurology, neuroscience, ophthalmology
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
Tagged: big data, coronavirus, immunology, research rising stars, vaccines
Playing the long game: An exciting discovery in telomere disease
Each time our cells divide, the protective caps that keep our chromosomes from fraying, called telomeres, lose a bit of their DNA. Telomeres shorten steadily as we age, but in certain medical conditions like dyskeratosis congenita, the process is accelerated. “Your telomeres determine your lifeline; how long they are determines how old your body is,” ... Read More about Playing the long game: An exciting discovery in telomere disease
Immune biomarkers predicted COVID-19 severity and could help in future pandemics
Why did some people fall critically ill from COVID-19 and others not? In May 2020, as COVID-19 swept the world, Boston Children’s Hospital helped launch a national, NIAID-funded study called IMPACC (IMmunoPhenotyping Assessment in a COVID-19 Cohort). Taking a “systems immunology” approach, the goal was to document the virus’s impact on the immune system in ... Read More about Immune biomarkers predicted COVID-19 severity and could help in future pandemics
Advancing mother-child health globally: Grace Chan MD, MPH, PhD
First in an ongoing series profiling researchers at Boston Children’s Hospital. Globally, five million children die annually before the age of 5. Forty percent of these deaths are in the first month — and many are preventable. Grace Chan, MD, MPH, PhD, finds these numbers unacceptable. They contrast starkly with U.S. medicine, including at Boston ... Read More about Advancing mother-child health globally: Grace Chan MD, MPH, PhD
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
Tagged: alzheimers disease, metabolism, neurology, neuroscience, proteomics