News of Note—A startup’s phase 2 drug slows liver cancer in mice; A new target in the fight against aging

A drug being developed by eFFECTOR was effective in mice with liver tumors driven by the genes MYC and KRAS. (Pixabay)

Phase 2 drug from eFFECTOR stops liver cancer in mice

Startup eFFECTOR has raised more than $110 million since it was founded in 2013 to study a small molecule designed to block two cancer-promoting enzymes. Now scientists at the University of California, San Francisco, have fresh evidence in mice that the treatment, eFT508, both slows the growth of liver cancer and prevents the cancer from evading an immune response. Davide Ruggero, Ph.D., a UCSF researcher and co-founder of eFFECTOR, discovered that in mice with liver tumors driven by the genes MYC and KRAS, the drug slashes levels of the immune-suppressing protein PD-L1 in half. The survival rate of the mice improved following the treatment with eFT508, the UCSF team reported in the journal Nature Medicine. The drug is currently in phase 2 trials to treat several cancer types. (Release)

A new way to fight age-related muscle diseases?

Swiss scientists have discovered that a protein called Pum2 promotes dysfunction in mitochondria—the energy producers inside of cells. A team at Ecole Polytechnique Fédérale de Lausanne screened cells from the muscles and brains of old animals and discovered elevated levels of Pum2, which corresponded with lower levels of another protein that normally promotes the clearance of dysfunctional mitochondria. The scientists used the gene editing technology CRISPR-Cas9 to inactivate the gene that makes Pum2 in old rodents. That significantly improved mitochondrial function in the animals, they reported in the journal Molecular Cell. They believe targeting Pum2 and other “RNA-binding proteins” could be effective in treating neuromuscular diseases. (Release)

Using bacteria to discover cancer promoters

When DNA becomes damaged, normal cells can acquire mutations that lead to cancer. But it’s not just external factors like smoking that cause DNA damage; cells that overproduce proteins can bring it upon themselves. Scientists at Baylor College of Medicine wanted to pinpoint which proteins cause cancer when they’re overproduced, so they took the bacterium E. coli, genetically modified it to glow red whenever its DNA was damaged, and then overexpressed each of its 4,000 genes. They identified 284 proteins that damage DNA. These proteins have also been linked to human cancers—a finding that may be helpful for developing new methods to identify people who face a high risk of developing the disease, the Baylor researchers believe. They published the finding in the journal Cell. (Release)

Virtual Clinical Trials Summit

Virtual Clinical Trials Summit: The Premier Educational Event Focused on Decentralized Clinical Trials

In this virtual environment, we will look at current and future trends for ongoing virtual trials, diving into the many ways companies can improve patient engagement and trial behavior to enhance retention with a focus on emerging technology and harmonized data access across the clinical trial system.