Month: January 2025

Rare Disease Innovation Hub begins to take shape: An FDA Official said the Agency’s virtual Rare Disease Innovation Hub, which was announced in July 2024, will be a focal point of contact where the rare disease community can raise issues about drug development. Julie Tierney, the Center for Biologics Evaluation and Research’s (CBER) Deputy Director for Strategy, Policy and Legislation, told a meeting sponsored by the American Society of Gene and Cell Therapy that a new Director of Rare Disease Strategy is being hired for the hub and will “identify the sticky spots” and develop a Policy Agenda.

Animal research paved the way for uterus transplants: Uterus transplants provide a promising alternative to surrogacy and adoption for people with severe uterine conditions, requiring two (2) surgeries, and in vitro fertilization (IVF) to create embryos that are then transferred to the uterus, leading to successful pregnancies. Uterus transplantation began with successful research in animals, including with mice, sheep, pigs and non-human primates, which helped refine the technique and paved the way for human trials.

An October 2023 order tasked the Department of Health and Human Services (DHHS) with recording reports of unsafe healthcare practices involving AI and pushed the National Institute of Standards and Technology (NIST) to build a U.S. AI Safety Institute as a home for technical guidance. Among his first actions as president, Donald Trump has rescinded an Executive Order signed during the Biden Administration that directed the U.S. government to develop safeguards for the development of artificial intelligence programs, including generative AI models and their use in the healthcare industry.

Improving clinical trials inclusivity for people with disabilities: Despite recent FDA efforts to increase diversity in clinical trials through an updated diversity action plan, people with disabilities remain underrepresented because of procedural barriers and study designs that exclude them, writes Richie Kahn, Co-Founder of the Patient Engagement firm Canary Advisors. A report by The National Council on Disability shows that people with disabilities are often unfairly excluded from clinical trials and calls for better accommodations, inclusive eligibility criteria and enforcement of accessibility laws.

Scientists use Artificial Intelligence (AI) to prepare and prevent future pandemics: The National Science Foundation is funding the development of a machine learning tool that analyzes posts on social media site X to detect early signs of a pandemic, predict outbreaks and evaluate possible effects of specific public health policies. The Coalition for Epidemic Preparedness Innovations has funded development of a tool called EVEScape that predicts the behavior of SARS-CoV-2 and other viruses. Meanwhile, drugmakers such as AstraZeneca are using AI to screen antibody candidates, which could help them develop vaccines and antivirals in the event of a virus outbreak.

FDA to investigate tampon safety after testing finds heavy metals: The FDA is investigating possible effects of toxic metals in tampons after a study in the journal Environmental International found traces of lead, arsenic, mercury or other metals in 14 brands of tampons. “The FDA’s laboratory study will measure the amount of metals that come out of tampons under conditions that more closely mimic normal use,” the agency said.

Placebo effect relieves chronic pain in mice: A study published in Current Biology has demonstrated that the placebo effect could be used to relieve chronic pain in mice by conditioning them to associate certain environments with pain relief. This suggests that similar strategies could be used to manage human pain, such as by increasing the effectiveness of pain medications by combining them with the placebo effect.

Lost starfish arms could bolster regenerative medicine: Similar neurohormones in starfish and humans could help scientists better understand the molecular processes behind the animal’s ability to regenerate limbs and lead to advances in regenerative medicine, researchers write in a Current Biology paper based on study of the species Asterias rubens, the common sea star. In humans and starfish, cholecystokinin-type neurohormones set off changes in target cells by binding to specific receptor proteins on the cell surface, which causes gallbladder contractions in humans and muscle contractions in starfish that allow them to detach their arms, then close the wound.

Drug that shrinks children’s heart tumors might work for dogs: Propranolol is used to treat hypertension, atrial fibrillation and pediatric vascular tumors, and now researchers at Atlantic Veterinary College are running a study to see if the drug can shrink heart tumors in dogs. Canine heart tumors are not rare; however, many dogs do not show signs of illness until the tumors are advanced, so finding enough dogs for the study may be a challenge, says veterinarian Abigail Martin, the Co-Lead Investigator and a second-year cardiology resident.