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New study finds cystic fibrosis drug allows patients to safely scale back lung therapies

A new multi-site study led by researchers at CU Anschutz shows that people with cystic fibrosis (CF) who start the triple-drug therapy elexacaftor/tezacaftor/ivacaftor (ETI) can safely reduce many of their daily lung treatments while maintaining good health for years.

The study was published in the Journal of Cystic Fibrosis.

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MIT chemists synthesize a fungal compound that holds promise for treating brain cancer

For the first time, MIT chemists have synthesized a fungal compound known as verticillin A, which was discovered more than 50 years ago and has shown potential as an anticancer agent.

The compound has a complex structure that made it more difficult to synthesize than related compounds, even though it differed by only a couple of atoms.

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World's first mitochondrial disease treatment 'MA-5' commences Phase II clinical trial

MA-5 is a therapeutic intervention for mitochondrial disease that has been demonstrated to ameliorate severe symptoms such as neurological disorders, renal dysfunction, hepatic dysfunction and motor disorders in animal disease models, which are induced by mitochondrial dysfunction. Crucially, it also improved survival rates.

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Researchers identify target to overcome treatment resistance in preclinical models of KRAS-mutant cancers

Researchers at The University of Texas MD Anderson Cancer Center have identified a specific protein, RASH3D19, that is responsible for activation of RAS signaling pathways involved in aggressive tumor growth and resistance to KRAS inhibitors in patients with KRAS-mutant cancers. Blocking RASH3D19 in combination with KRAS inhibitors improved outcomes in preclinical models, suggesting this combination as a potential therapeutic strategy for patients with KRAS-mutant cancers.

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Experimental drug repairs DNA damage caused by disease

Cedars-Sinai scientists have developed an experimental drug that repairs DNA and serves as a prototype for a new class of medications that fix tissue damage caused by heart attack, inflammatory disease or other conditions.

Investigators describe the workings of the drug, called TY1, in a paper published in Science Translational Medicine.

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Finnish study shows robust immune responses to H5N8 avian influenza vaccine

Finland was the first country to offer the zoonotic avian influenza A(H5N8) vaccine manufactured by Seqirus to at-risk occupational groups following the extensive clade 2.3.4.4b A(H5N1) outbreak affecting wild birds and fur farms in Finland in 2023.

A new study published in Nature Microbiology shows that the MF59-adjuvanted A(H5N8) vaccine induced strong immune responses, including both functional antibodies and memory T-cell responses,

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A starting point for the development of new pain and cancer drugs

The human P2X4 receptor plays an important role in chronic pain, inflammation and some types of cancer. Researchers at the University of Bonn and the University Hospital Bonn (UKB) have now discovered a mechanism that can inhibit this receptor. The results were recently published in the scientific journal Nature Communications and open up a pathway for the development of new drugs.

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How the nervous system activates repair after spinal cord injury

After a spinal cord injury, cells in the brain and spinal cord change to cope with stress and repair tissue. A new study from Karolinska Institutet, published in Nature Neuroscience, shows that this response is controlled by specific DNA sequences. This knowledge could help develop more targeted treatments.

When the central nervous system is damaged - for example, in a spinal cord injury - many cells become reactive.

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From inhibition to destruction - kinase drugs found to trigger protein degradation

Protein kinases are the molecular switches of the cell. They control growth, division, communication, and survival by attaching phosphate groups to other proteins. When these switches are stuck in the "on" position, they can drive cancer and other diseases. Not surprisingly, kinases have become one of the most important drug target families in modern medicine: today, more than 80 kinase inhibitors are FDA-approved, and nearly twice as many are in clinical development.

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FDA expands artificial intelligence capabilities with agentic AI deployment

The U.S. Food and Drug Administration today announced the deployment of agentic AI capabilities for all agency employees. Agentic AI capabilities will enable the creation of more complex AI workflows - harnessing various AI models - to assist with multi-step tasks.

Agentic AI refers to advanced artificial intelligence systems designed to achieve specific goals by planning, reasoning, and executing multi-step actions.

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