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Utrophin as a novel therapeutic approach for Duchenne muscular dystrophy
Upregulating utrophin using small molecules could be a new therapeutic approach for Duchenne muscular dystrophy (DMD).
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Small Molecules
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Method to make integrin receptor-targeting drugs safer in future presented in study
Research into the structure of the drug-integrin complex has enabled the creation of drugs which inhibit integrin as effectively as currently used compounds, without causing excessive bleeding.
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Small molecule targeting mRNA could slow the progression of Parkinson’s disease
Research identified a small molecule that targets the mRNA of α-synuclein and prevents the formation of the protein, an approach which could provide novel neurodegeneration therapies.
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Could Frizzled receptors be the target of new cancer therapeutics?
The G-protein coupled receptor Frizzled, implicated in diseases like cancer, can be targeted with small molecules which could provide the basis for anti-cancer therapeutics, according to researchers.
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Novel small molecule could treat Alzheimer’s disease and Dravet syndrome
A new experimental drug was shown by researchers to restore natural brain rhythms in mouse models of Alzheimer’s and promote learning and memory.
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Common prostate cancer mutation inspires path to new anti-cancer therapy
PPP2R2A gene allele deletion in prostate cancers promotes the uncontrolled division of cells, reinstatement of its protein causes cancer cell death, so could provide a new therapeutic option.
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Accurate model of organ scarring created using stem cells in a lab
Researchers have developed a 'scar in a dish' model derived from human stem cells which mimics the progressive scarring that occurs in human organs and has lead to a drug candidate being identified to stop the progression of fibrosis.
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Researchers find 150 possible inhibitors to fight Nipah virus
A group of scientists from India have used 3D imaging of the Nipah virus to discover potential inhibitors for the disease.
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Promising new therapy developed to treat leukaemia
VTP50469, a new targeted therapy for MLL-rearranged leukaemia, shows greater efficacy and fewer side-effects in mice, according to new research.
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New screening method identifies potential compounds to combat cancer
The new screening technique may enable development of novel combination-immunotherapy regimens against cancers and persistent infections.
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PJ34 triggers self-destruction of human pancreatic cancer cells
A study conducted on human pancreatic tumours transplanted in mice reveals promise for an effective therapy in the future, say researchers.
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Researchers must not rely on generic online searches for chemical probes, study finds
The ICR has revealed that during drug discovery, researchers should not use general search engines and vendor catalogue information to decide on their use of chemical probes.
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SEL120: a promising CDK8 kinase inhibitor
A novel CDK8 inhibitor has been developed by scientists at Ryvu Therapeutics. Dr Krzysztof Brzozka showcases this molecule and explains the R&D behind it.
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High-throughput screening used to discover new class of antibiotics
Researchers have used a high-throughput screening method to test over 125,000 molecules and identify a new class of antibiotics.
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Osteoporosis in mice reversed with new drug target
A pharmaceutical target has been identified by Duke University that, when activated, can reverse bone degradation caused by osteoporosis.