Weight loss: When scientific paradigms collide
David S. Ludwig, MD, PhD, co-directs the New Balance Foundation Obesity Prevention Center at Boston Children’s Hospital. Because conventional thinking usually rests on years (sometimes centuries) of research, scientists are naturally skeptical of radical new ideas. But occasionally, problems with normal science arise, such as a persistent failure to solve important puzzles and anomalies that can’t be ... Read More about Weight loss: When scientific paradigms collide
Making ready-made CAR T cells for cancer immunotherapy
In CAR T-cell immunotherapy, T cells from a patient’s own blood are engineered to carry so-called chimeric antigen receptors (CAR) that enable the T cells to attack and kill tumor cells. While CAR-T therapy is a powerful approach for certain leukemias and lymphomas, it’s not available for many patients who need it. It can be ... Read More about Making ready-made CAR T cells for cancer immunotherapy
A new lens on cardiac surgery could help prevent arrhythmia
Sometimes, a change in perspective can lead to a medical breakthrough. A type of microscopy typically used to detect cancer and other diseases has been adapted to reveal the location of unseen conduction tissue around the heart. The Boston Children’s clinician behind this innovation now aims to prove the safety and effectiveness of fiber-optic confocal ... Read More about A new lens on cardiac surgery could help prevent arrhythmia
A new vaccine formulation could finally protect babies against RSV
Though often mild, respiratory syncytial virus (RSV) infection can cause babies to be hospitalized with bronchiolitis or pneumonia. Globally, it is the leading cause of death in children under 5. Several vaccines against RSV are being tested in adults. But there has been no progress on an RSV vaccine for children since 1966 — the ... Read More about A new vaccine formulation could finally protect babies against RSV
Chasing Rett syndrome biomarkers: Could overnight EEG studies improve care?
In Rett syndrome, a genetic condition affecting girls almost exclusively, mutations in the MECP2 gene cause a regression of language and motor skills starting at 12 to 18 months of age. Children then usually stabilize, but how Rett will ultimately affect them is hard to predict. Some girls develop epilepsy; others have no seizures at ... Read More about Chasing Rett syndrome biomarkers: Could overnight EEG studies improve care?
A potential danger of CRISPR gene editing — and why base editing may be safer
Gene therapy using CRISPR/Cas9 gene editing is currently in clinical trials around the world for a variety of diseases, including various cancers, blood disorders, and metabolic disorders. It works by making cuts in DNA — both strands of the double helix — to insert or remove genes. But CRISPR carries a potential, previously undiscovered danger, ... Read More about A potential danger of CRISPR gene editing — and why base editing may be safer
New work transforms our knowledge of how blood is formed
The origins of our blood may not be quite what we thought. In groundbreaking research, scientists in the Stem Cell Program at Boston Children’s Hospital used cellular “barcoding” techniques in mice to track the development of blood in real time — and found that blood cells originate not from one type of mother cell, but ... Read More about New work transforms our knowledge of how blood is formed
Genomic ‘fingerprinting’ yields better treatments for pediatric solid cancers
Genomic profiling is increasingly used for solid tumors in adults and for pediatric brain tumors and blood cancers, allowing treatments to be matched to patients’ mutations. But for children with solid tumors, genomic fingerprinting has been elusive, because these cancers are so varied and individually so rare. Therapies therefore remain non-specific: chemotherapy, surgery, and/or radiation. ... Read More about Genomic ‘fingerprinting’ yields better treatments for pediatric solid cancers
Pregnant mothers who get COVID-19 vaccines are also protecting their babies
Recent studies have shown that COVID-19 vaccination is safe for expectant mothers and can protect them against infection, severe illness, and death from COVID-19. We also know that mothers vaccinated during pregnancy pass coronavirus antibodies to their babies. The latest research — drawing on 30 children’s hospitals in 22 states — now confirms that vaccinating ... Read More about Pregnant mothers who get COVID-19 vaccines are also protecting their babies
Exploiting a vulnerability in an aggressive leukemia
Survival has improved greatly in children with acute lymphocytic leukemia (ALL). But a certain form of ALL that occurs mostly in babies is still very lethal, with a survival rate below 50 percent: B-cell acute lymphoblastic leukemia with rearrangements of the mixed lineage leukemia gene, or MLL B-ALL. “Something about the biology of this type ... Read More about Exploiting a vulnerability in an aggressive leukemia