Cystic fibrosis (CF) is characterized by lack of hydration in the airways by impaired functioning of cystic fibrosis transmembrane conductance regulator (CFTR), leading to infection, inflammation and lung tissue damage. It is hypothesized that inhibiting the epithelial sodium channel (ENaC) in the airways in CF may enhance the mucociliary clearance (MCC) and provide clinical benefit, but numerous inhaled ENaC blockers have failed in clinical trials. Enterprise Therapeutics Ltd. is developing an inhaled ENaC blocker compound, ETD-001, for the treatment of CF.

Researchers from the National Cancer Institute presented data from a study that aimed to explore the role of the major nuclear exporter protein exportin 1 (XPO1) in pancreatic neuroendocrine tumor (pNET) subsistence and therapy resistance.

Activation of the stimulator of interferon genes (STING) pathway has recently proven effective as an antiproliferative strategy in glioma and melanoma.

Craniopharyngiomas are a rare brain cancer of the suprasellar region, and despite being benign tumors, their management can be challenging. Investigators from the Children’s Hospital of Philadelphia have aimed to identify transcriptional factors that may predict recurrence of craniopharyngiomas.

Scientists at the University of Copenhagen have demonstrated that the trigeminal nerve, a cranial nerve whose activation underlies migraine pain, has direct access to cerebrospinal fluid (CSF) transported by the glymph system. Furthermore, in the run-up to a migraine, levels of multiple proteins in the CSF changed. One of them was calcitonin gene-related peptide (CGRP), a driver of migraine pain and target of several approved drugs for both treatment and prevention of migraine.