Making ionocytes: A step toward cell or gene therapy for cystic fibrosis

Cystic fibrosis transmembrane conductance regulator (CFTR) modulators were a breakthrough for cystic fibrosis, improving the movement of chloride and water and moistening mucus secretions. But these drugs are expensive, don’t work in all patients with cystic fibrosis, and have side effects and interactions with other drugs. People who do respond to CFTR modulators must take them for a lifetime.

Bold new therapy delivery method shows initial promise as treatment for Duchenne muscular dystrophy

Doug Millay, Ph.D., a scientist with the Division of Molecular Cardiovascular Biology at Cincinnati Children’s has dedicated his career to revealing the most fundamental mechanisms of skeletal muscle development. He has been a leader in characterizing how two “fusogens” called Myomaker and Myomerger mediate the entry of stem cells into mature muscle cells to build the tissue that humans depend upon for movement, breathing, and survival.

Patients in clinical trial for prostate cancer report no decline in quality of life at one year post-treatment

The patient-reported outcomes (PROs) of the phase 3 NRG Oncology clinical trial RTOG 0815 comparing dose-escalated radiotherapy (RT) alone to dose-escalated RT combined with short-term (6 months) androgen deprivation therapy (STAD) indicate that the addition of STAD did not significantly impact urinary or bowel quality of life but did significantly decrease hormone and sexual quality of life. However, this decline in quality of life was temporary and there were no clinically meaningful differences in quality of life between treatment arms by one year after the initiation of treatment. These results of this study, led by Benjamin Movsas, were recently published in the Journal of Clinical Oncology.