Researchers at Umeå University have contributed new insights into how cancer cells protect themselves from cell death. The study provides a deeper understanding of how key proteins interact within the cell and could, in the long term, support the development of new cancer therapies.

Neuroblastoma kills more children under one year of age than any other extracranial solid tumor, and high-risk cases have resisted meaningful improvement in survival for decades. A team at the Hebrew University of Jerusalem has now identified a molecular accomplice: neuronal nitric oxide synthase, or nNOS, which feeds the mTOR growth-signaling pathway through nitrosative stress. Blocking nNOS, either pharmacologically with the compound BA-101 or genetically with siRNA, silenced mTOR signaling and crippled malignant behavior in human neuroblastoma cells. In a xenograft mouse model, BA-101 shrank tumors dramatically (p < 0.001). The nNOS–mTOR axis emerges as a new and targetable vulnerability. NeuroNOS Ltd., which partly funded this work, has obtained a license for the patent applications of the BA-101 molecule filed by Yissum (The Hebrew University Technology Transfer Company). The authors, in collaboration with NeuroNOS, have also demonstrated the therapeutic efficacy of BA-101 in glioblastoma.

More than three-quarters of all cases of liver cancer worldwide are associated with chronic viral hepatitis but scientists have been limited in their ability to model how these viruses lead to cancer. In the new study, a Rockefeller team showed that mice infected with an engineered version of a rat virus develop liver inflammation, scarring, and ultimately cancer similar to that seen in humans with viral hepatitis-associated liver cancer. The new mouse model can be used to study how liver virus infection leads to cancer as well as to test treatments.

Salk scientists have created a platform to study mitochondrial DNA mutations that lead or contribute to human disease, and generated a library of 155 mitochondrial DNA mutant cells using the platform. The platform, library, and findings will accelerate therapeutic development for mitochondrial disorders, as well as help scientists treat mitochondrial dysfunction in other diseases and conditions like cancer or aging.