Archive for drug development
Botulism breakthrough? Taming botulinum toxin to deliver therapeutics
While rare, botulism can cause paralysis and is potentially fatal. It is caused by nerve-damaging toxins produced by Clostridium botulinum — the most potent toxins known. These toxins often lurk in contaminated food (home canning being a major culprit). Infants can also develop botulism from ingesting C. botulinum spores in honey, soil, or dust; the ... Read More about Botulism breakthrough? Taming botulinum toxin to deliver therapeutics
2020, the year COVID-19 tuned us into science: Part 2
Thanks — or no thanks — to COVID-19, 2020 has been a year in which science became a household topic of discussion. Our last post recounted what we collectively learned in the spring: what COVID-19 is, who is at risk, how to test for it. As the year unfolded, there were new lessons to be learned. ... Read More about 2020, the year COVID-19 tuned us into science: Part 2
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 about It’s personal: How the Boston Children’s progeria research community brought new life to an old drug
Precision chemo-immunotherapy for pancreatic cancer?
Pancreatic cancer is highly lethal and in great need of better treatments. Only about 10 percent of patients remain alive five years after diagnosis. In a new study, researchers in the lab of Marsha Moses, PhD, at Boston Children’s Hospital offer a glimmer of hope. Key takeaway An antibody-drug combination effectively targeted, penetrated, and shrank ... Read More about Precision chemo-immunotherapy for pancreatic cancer?
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 about A diabetes drug with a potential side benefit: Reduced COVID-19 mortality
Lung ‘organoids’ capture early-stage lung cancer; could help test treatments
Lung cancer, the leading cancer killer in the U.S., is often missed in its earlier stages. And while recent imaging advances offer earlier detection, early-stage lung cancers still have no targeted treatments. Key takeaways Mini “organoids” made from lung cells offer a rapid platform for tracking early-stage lung cancers and for testing possible treatments. RNA ... Read More about Lung ‘organoids’ capture early-stage lung cancer; could help test treatments
Gene therapy’s future may be all about the bases
Gene therapy offers the possibility of a cure for many genetic disorders, especially those involving a single gene. The first kind of gene therapy used a virus to carry a corrected copy of the gene into people’s cells. When the early viral vectors used in the 1990s were found to have off-target effects, sometimes even ... Read More about Gene therapy’s future may be all about the bases
A drug treatment for telomere diseases?
For years, Donna Martin carried a piece of scrap paper with the words “dyskeratosis congenita,” which she believed might explain her son Brad’s sudden, mysterious affliction. A routine blood test had revealed Brad’s bone marrow was failing, unable to keep up with his need for healthy blood cells. His condition, Donna knew, would worsen over ... Read More about A drug treatment for telomere diseases?
Targeted small-molecule agent shows early promise against a dangerous infant leukemia
Leukemias involving reshuffling or rearrangement of the mixed lineage leukemia (MLL) gene, known as MLL-rearranged or MLL-r leukemias, account for 70 to 80 percent of acute leukemias in infants under one year old. In these blood cancers, a subset of acute myeloid and acute lymphoid leukemias (AML and ALL), the MLL gene breaks and reattaches ... Read More about Targeted small-molecule agent shows early promise against a dangerous infant leukemia
Boosting host immune defenses to treat tuberculosis
Current treatment regimens for Mycobacterium tuberculosis (Mtb), the causative agent of tuberculosis, are long, complex, and hard for people to sustain. Moreover, the bacteria often develop drug resistance, and many people harbor multi-drug-resistant strains. In 2018 alone, nearly 1.5 million people died from tuberculosis worldwide. Now, a study in iScience suggests a new approach that ... Read More about Boosting host immune defenses to treat tuberculosis