Archive for clinical trials
What’s the difference between the COVID-19 vaccines?
After a long, difficult year, the world had reason to celebrate in late 2020 when the U.S. Food and Drug Administration (FDA) authorized two COVID-19 vaccines for emergency use. Many more COVID-19 vaccines are in various stages of development. Several of these are in the final stage of clinical trials and could be submitted for ... Read More
It’s personal: How the Boston Children’s progeria research community brought new life to an old drug
In late November, the U.S. Food and Drug Administration (FDA) approved a repurposed cancer drug called lonafarnib to treat Hutchinson-Gilford progeria syndrome, better known as progeria — an ultra-rare incurable genetic disease associated with rapid aging. On average, children with progeria die before age 15, usually the result of rapidly accelerated atherosclerosis resulting in heart ... Read More
Poverty predicts worse cancer outcomes, even in children receiving top-tier care
A pair of recent studies suggests that even among patients receiving advanced cancer care, poverty is a predictor of worse outcomes. The disturbing findings raise many questions that follow-up studies are now exploring. Key takeaways Poverty was associated with 3.7-fold higher mortality among children receiving targeted immunotherapy for high-risk neuroblastoma. Household poverty was associated with ... Read More
The new COVID-19 vaccines: Will they be safe?
Just within the past week, pharmaceutical companies have announced encouraging news about two COVID-19 vaccines in the final development phase. As more vaccine developers announce similar reports in the coming weeks and months, it’s key to remember that vaccine safety is as important as effectiveness against the virus. Fortunately, scientists have learned lessons from the ... Read More
Remdesivir: What to know about the first drug approved to treat COVID-19
In late October, the U.S. Food and Drug Administration (FDA) officially approved the antiviral drug remdesivir (Veklury) for COVID-19. The approval has been controversial, with many scientists questioning the strength of the evidence and proposing that the drug undergo further clinical trials before receiving full FDA approval . What does this mean for children who ... Read More
A diabetes drug with a potential side benefit: Reduced COVID-19 mortality
In 2006, the Food and Drug Administration approved the drug sitagliptin to lower blood sugar in type 2 diabetes. Now, a multicenter observational study in Italy suggests the drug also cuts mortality in diabetic patients hospitalized with COVID-19 — by as much as half. Paolo Fiorina, MD, PhD, of Boston Children’s Hospital led the study, ... Read More
Global partnership leads to improved treatment for childhood diarrhea
Diarrheal diseases remain a major childhood health problem in many parts of the world. Each year, they lead to the deaths of more than 500,000 children under age 5 globally. A new large clinical trial based in Tanzania and India, led by investigators at Boston Children’s Hospital, local research universities, and the World Health Organization ... Read More
Two drugs join forces against COVID-19
Two drugs, including one developed by a researcher at Boston Children’s Hospital, inhibit the SARS-CoV-2 virus that causes COVID-19 in tests of human cells. Both drugs, vacuolin-1 and apilimod, originally developed years ago, target a large enzyme called PIKfyve kinase. Prior to this study, little was known about this enzyme’s role in COVID-19 infection. Details ... Read More
Trial tests dornase alfa, a cystic fibrosis drug, for severe COVID pneumonia
Boston Children’s Hospital and Brigham and Women’s Hospital will soon begin testing an existing drug, dornase alfa, in patients with severe COVID-19 pneumonia and respiratory failure. The randomized, controlled clinical trial aims to enroll 60 adults and children over age 3 who require mechanical ventilation. Key takeaways:· A randomized trial is testing whether a cystic ... Read More
Hydroxychloroquine and COVID-19: What we know so far
Despite the current hype, infectious disease experts and others are cautious about using chloroquine drugs in the current COVID-19 pandemic. While the urgent need for COVID-19 treatments justifies the rapid pace of trials, several concerns have been raised related to the limitations of studies published so far. Kristin Moffitt, MD The drugs chloroquine and hydroxychloroquine ... Read More