Archive for drug development
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
Made-to-order therapies get a boost with new FDA guidelines
Ed. note: Mila passed away in February, 2021, at age 10. The Mila’s Miracle Foundation continues to work to pave a pathway for personalized treatments. Science-based treatments for rare genetic diseases have burgeoned in the past decade. That includes diseases so rare they affect just a handful of patients — or in some cases, just ... Read More
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
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
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
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
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