Breaking WORLD PHARMA NEWS

Shape-shifting cell channel reveals new target for precision drugs

From small ions to large molecules, cellular gates control what can pass in and out of cells. But how one such gate, called pannexin-1 (PANX1), can handle vastly different cargo sizes has remained a long-standing mystery.

In a new study, Northwestern University scientists uncovered the molecular trick behind PANX1’s versatility.

Read more …

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.

Read more …

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.

Read more …

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.

Read more …

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.

Read more …

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.

Read more …

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,

Read more …

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.

Read more …

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.

Read more …

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.

Read more …