Archive for drug development
From our labs and clinics: The top 10 COVID-19 science stories of 2021
As COVID-19 waxed, waned, morphed, and waxed again this year, research was taking place throughout Boston Children’s Hospital. Ongoing national studies the hospital is leading or co-leading include Overcoming COVID-19, a CDC-funded study on COVID-19 and multisystem inflammatory syndrome in children (MIS-C); IMPACC, examining immune responses in people hospitalized with COVID-19; and MUSIC, studying MISC’s ... Read More
Emerging protein-based COVID-19 vaccines could be game-changing
Current messenger RNA vaccines appear to offer at least some protection against new SARS-CoV-2 variants, including Omicron, especially for people who have received boosters. But manufacturing costs and the need for ultra-cold refrigeration have limited availability of these vaccines in low-and middle-income countries. That’s where emerging protein-based COVID-19 vaccines — including two candidates developed at ... Read More
It takes a village: Creating best practices for personalized treatments
In 2019, doctors and scientists at Boston Children’s Hospital announced a medical landmark. In the space of one year, a team led by Timothy Yu, MD, PhD, pinpointed a one-of-a-kind genetic mutation in a girl named Mila with Batten disease, developed a custom drug called milasen to repair the mutation, and treated Mila with it. ... Read More
New research NETs a fresh angle for treating severe inflammation
As we’ve seen during the COVID-19 pandemic, serious infections sometimes trigger an excessive inflammatory reaction that does as much harm — or more — than the infection itself. New research at Boston Children’s Hospital and Brigham and Women’s Hospital suggests a potential way to block this hyperinflammation response by repurposing or modifying an existing drug. ... Read More
Finding new targets for acute myeloid leukemia in children
Acute myeloid leukemia (AML) is the second most common leukemia diagnosed in children. It is hard to treat and can be fatal in some cases. While there have been some recent successes with genetically targeted therapies for adults, AML has different genetic features in children, and care has been slow to advance. “The state of ... Read More
A promising new antiseizure drug tailored to newborns
Neonatal seizures can lead to serious consequences, including significant cognitive and motor disabilities, lifelong epilepsy, and death. They are often highly resistant to treatment, in part because seizures in newborns are fundamentally different from seizures in older children and adults. Yet they are treated in much the same way as older patients, with little change over the decades. Better treatment is ... Read More
A bio-inspired approach to delivering local anesthetics
Site 1 sodium channel blockers such as tetrodotoxin and saxitoxin are small-molecule drugs with powerful local anesthetic properties. They provide pain relief without toxic effects on local nerves and muscles, and are an attractive alternative to opioids. But injected by themselves, the anesthetics can easily float away, causing severe systemic toxicity. Encapsulating these drugs in ... Read More
RNA-modifying protein offers a possible lead for treating aggressive cancers
A protein that modifies RNAs, called METTL1, could be a target for treating some aggressive, difficult-to-treat cancers, suggests new research in Molecular Cell. The study provides evidence that blocking METTL1 curbs cancer cells’ ability to grow, selectively killing them, and the researchers believe it could be targeted with drugs. METTL1 and related proteins had previously ... Read More
Motor neurons made from patients’ cells reveal possible ALS drugs and targets
Amyotrophic lateral sclerosis (ALS) is a severe, fatal neurodegenerative disorder causing loss of motor neurons and voluntary muscle action. While mouse studies have identified potential treatments, these drugs have typically done very poorly in human trials. “One of the most difficult challenges in drug discovery is identifying a target that has a key role in ... Read More
Looking for cancer’s Achilles heel: The Pediatric Cancer Dependency Map
Thanks to developments in precision medicine, some adult cancers are now treated with designer drugs that target the genetic mutations that caused them. But most children with cancer have not reaped the same benefits. Unlike adult cancers, childhood cancers carry few genetic mutations. And the mutations these tumors do have are typically harder to make ... Read More