Why metabolism matters in Fanconi anemia
Peer-Reviewed Publication
Updates every hour. Last Updated: 29-Nov-2025 16:11 ET (29-Nov-2025 21:11 GMT/UTC)
A discovery from Australian researchers could lead to better treatment for children with neuroblastoma, a cancer that currently claims 9 out of 10 young patients who experience recurrence. The team at the Garvan Institute of Medical Research in Sydney found a drug combination that can bypass the cellular defences these tumours develop that lead to relapse.
In preclinical studies, researchers found that priming the immune system with a Treg-expanding therapy before stem cell transplant boosted survival, protected vital organs, and promoted a balanced gut microbiome—offering hope for safer, more effective treatment of blood cancers. The study is highlighted on the cover of the Nov. 27, 2025, issue of the journal, Blood.
A new collaborative study from Pusan National University and Yonsei University suggests that the FOLFIRINOX regimen showed numerically improved survival outcomes compared to current standards such as FOLFOX, FOLFIRI, and nal-IRI/FL, while maintaining manageable toxicity levels. These findings suggest that FOLFIRINOX could serve as a promising second-line treatment option for patients with advanced BTC following first-line chemotherapy failure, warranting further validation through prospective clinical studies.
When gene transcription falls out of sync with other biological processes, that dysfunction can contribute to aging, cancer and other diseases. Researchers revealed how key regulatory proteins work in a precise hierarchy to meticulously adjust pacing during transcription. These regulatory proteins may now emerge as potential drug targets for a variety of disorders. The single-molecule platform that revealed these findings is a novel approach to studying similar processes that could have broad applications in biology.
For decades, kinase inhibitors have been a mainstay of cancer therapy, designed to switch off enzymes that fuel uncontrolled cell growth. But new research shows that these drugs often go further: they can also cause the very proteins they target to be dismantled by the cell, making them yet another tool for the emerging field of Targeted Protein Degradation (TPD). In a new study published in Nature, scientists at CeMM and AITHYRA in Vienna, and the IRB in Barcelona, with partners across Europe, the US and China, have now mapped this effect systematically, uncovering a widespread but overlooked phenomenon in pharmacology.