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.

Action needed on breastfeeding data collection to gauge impact of medicines

A new systematic review from Swansea University in collaboration with ConcePTION, an Innovative Medicines Initiative (IMI), has called for action to monitor infants for any possible adverse drug reactions from exposure to medicines through breastmilk, even though problems may be rare; target additional support to breastfeeding patients whose medicines may affect breastfeeding; and provide long-term population-based data on any adverse events in infants exposed to medicines through breastmilk and any reduction in breastfeeding rates.

Study shows NIH investment in new drug approvals is comparable to investment by pharmaceutical industry

The National Institutes of Health (NIH) spent $187 billion for basic or applied research related to 354 of the 356 drugs approved by the FDA from 2010 to 2019, according to a new study from Bentley University’s Center for Integration of Science and Industry. The study, published in JAMA Health Forum, shows that the amount invested per approved drug by the NIH is comparable to that of reported investment by the biopharmaceutical industry.

Scientists identify antivirals that could combat emerging infectious diseases

A new study has identified potential broad-spectrum antiviral agents that can target multiple families of RNA viruses that continue to pose a significant threat for future pandemics. The study, led by Gustavo Garcia Jr. in the UCLA Department of Molecular and Medical Pharmacology, tested a library of innate immune agonists that work by targeting pathogen recognition receptors, and found several agents that showed promise, including one that exhibited potent antiviral activity against members of RNA viral families.