Repurposed drug thalidomide shows promise for hard-to-treat brain and spinal cord arteriovenous malformations
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Central nervous system arteriovenous malformations, or CNS-AVMs, are abnormal tangles of blood vessels in the brain or spinal cord. They form direct high-flow connections between arteries and veins and can cause hemorrhagic stroke, seizures, headache, neurological deficits, or progressive disability, especially in children and young adults. Current treatments, including microsurgery, embolization, and stereotactic radiosurgery, can be effective but are invasive and may carry substantial risks for patients with complex lesions.
New discovery uses salt to overcome major obstacle in gene therapy
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Researchers at the University of Houston’s College of Pharmacy have discovered an unexpected simple strategy to improve the performance of mRNA vaccines and gene therapeutics: adding salt. The findings, published in Small, address one of the biggest challenges facing modern gene medicine - getting fragile therapeutic material to the right place inside cells.
Common asthma drug shows promise for reversing fatty liver
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MUSC researchers are tackling MASH, or metabolic dysfunction-associated steatohepatitis, a liver disease affecting hundreds of millions worldwide. It is also a leading cause of liver transplantation, yet treatment options remain limited.
A new paper published in Nature partner journal (npj) Metabolic Health and Disease suggests that a widely used asthma medication, formoterol, could potentially offer a different therapeutic pathway altogether. Formoterol is a beta-2 adrenergic receptor agonist that has been prescribed for decades to open airways in conditions like asthma and chronic obstructive pulmonary disease.
Nanoparticles overcome drug-resistant cancer via sequential drug release and photothermal therapy
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Cancer cells frequently develop the ability to expel anticancer drugs before they can work - a phenomenon called multidrug resistance (MDR) - which is one of the leading reasons why chemotherapy fails in patients. This research addresses that problem with a fundamentally new strategy: instead of simply increasing drug doses or switching drugs, researchers engineered nanoparticles that first disable the cancer cell's drug-expulsion mechanism, and only then release the anticancer drug.
AI-assisted approach identifies IRS4 as a promising drug target in multiple solid tumors
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Published in Science Advances, St. Jude Children’s Research Hospital scientists used a mix of genetic cancer dependency data, artificial intelligence (AI) and naturally occurring mutations to prioritize safer cancer drug targets. They focused their efforts on targets most likely to be effective while limiting unwanted toxicity, identifying IRS4 as a potential dependency across multiple tumor types. The work provides a proof of principle for evaluating potential toxicity early in the search for novel therapeutics.
Researchers identify natural compound that disarms drug-resistant bacteria
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Every year, antibiotic-resistant Staphylococcus aureus, commonly known as “staph,” causes serious infections and outbreaks in hospitals and community settings, disproportionately affecting vulnerable populations, including the elderly and those with weakened immune systems. Methicillin-resistant strains, known as MRSA, are a leading contributor to deaths associated with antimicrobial resistance globally.
Scientists discover a new way to make drug-resistant cancer treatable again
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Cancer cells survive by repairing damage to their DNA - even damage that would normally be fatal. One of their most important defense systems is homologous recombination, a high-precision repair pathway that fixes broken DNA using key proteins such as RAD51 and CHK1. While therapies such as PARP inhibitors have successfully targeted this vulnerability, many tumors eventually regain their DNA repair ability and become resistant to treatment.
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