ResearchPUBMED2 days ago
Scientists are studying a new type of medicine made from circular RNA, which is RNA shaped like a circle instead of a line. Because of their circular shape, these medicines may stay in the body longer and work better than current RNA medicines. Researchers are testing these circular RNA medicines to treat cancer, immune system diseases, and rare diseases.
WHY IT MATTERSCircular RNA therapeutics could eventually offer longer-lasting treatments for rare diseases with fewer doses needed, but patients should know that claims about safety and effectiveness vary depending on how the medicine is made—there's no one-size-fits-all answer yet.
ResearchPUBMEDApr 14
Scientists have discovered that certain rare genetic diseases are caused by mutations in genes that help cells copy their DNA. These genes normally produce proteins that untangle special twisted DNA structures called G-quadruplexes that get in the way during copying. When these proteins don't work properly, cells can't copy their DNA correctly, leading to problems like weak immune systems, slow growth, birth defects, and increased cancer risk.
WHY IT MATTERSUnderstanding which genes cause these G-quadruplex problems could help doctors diagnose patients with unexplained immunodeficiency, growth delays, or birth defects, and may eventually lead to targeted treatments for these currently untreatable conditions.
ResearchPUBMEDApr 1
Researchers studied whether the amount of cancer in a patient's body before treatment affects how well two immunotherapy drugs work together. They looked at 722 patients with rare cancers who received nivolumab and ipilimumab (two drugs that help the immune system fight cancer). The study wanted to understand if patients with smaller tumors do better than those with larger tumors when treated with these combination drugs.
WHY IT MATTERSIf baseline tumor size predicts treatment response in rare cancers, doctors could better counsel patients on expected outcomes and identify which patients might benefit most from this dual immunotherapy approach before starting treatment.