The US Food and Drug Administration (FDA) announced it is unifying its various Adverse Reporting systems into a single system called the Adverse Event Monitoring System (AEMS). The Agency said the move would modernize adverse event reporting systems and provide more transparency.
Family funding accelerates research into frontotemporal dementia: A wealthy family’s investment through the Bluefield Project helped transform research on frontotemporal dementia caused by GRN (a gene that codes for a protein called progranulin ) mutations, driving discoveries about progranulin’s role in brain inflammation and cellular waste recycling and helping launch several clinical trials. Despite setbacks with some of these trials, Bluefield continues to advance biomarker research and patient recruitment, and its founders have also created a new nonprofit to explore alternative therapeutic targets.
Multi-cancer blood tests offer promise but face skepticism: Multi-cancer early detection blood tests that analyze tumor DNA in the bloodstream are being promoted as a way to detect dozens of cancers from a single sample; however, early trial results, including a large study of the Galleri test, have not yet shown a significant reduction in late-stage cancer diagnoses and have raised concerns about missed cancers and false positives. Experts caution that although advances are being made, MCEDs’ accuracy and ability to improve patient outcomes remain uncertain.
In the first reported case of its kind, surgeons in China connected a liver failure patient to a genetically modified pig liver that filtered his blood for nearly three (3) days while he awaited a human transplant. The procedure served as a temporary “bridge” therapy until a donor organ became available, highlighting the potential of xenotransplantation to support patients with severe organ failure.
The core mission of the US Food and Drug Administration’s (FDA) inspections program, as well as how it prioritizes inspections, has remains unchanged despite recent leadership changes at the agency, said Ivy Sweeney, Deputy Director of the Office of Human and Animal Drug Inspectorate (OHADI).
A study published in Cell Stem Cell found that a single dose of mesenchymal stem cells can improve endurance in older adults with frailty. The highest dose resulted in a 20% improvement in a six-minute walk test. The stem cells, derived from donated bone marrow, do not require immunosuppressive medications.
Vaccine pioneer warns of resurgence in vaccine-preventable diseases: Stanley Plotkin, a pioneering vaccine scientist often called the “godfather of vaccines,” reflects on his career developing the rubella and rotavirus vaccines and warns that declining vaccination rates could allow previously controlled childhood diseases to return. “In the absence of disease, people are less worried, and they ask themselves: Why should I vaccinate the child because there’s no disease around?” Plotkin said.
Spinal cord organoids show promise for paralysis therapy: Research with mice previously showed that a single injection of a peptide gel helped animals regain walking after severe spinal cord injury, and new studies with human stem cell-derived spinal cord organoids found the same treatment reduced scar tissue and encouraged nerve fibers to grow after injury. The gel forms a soft scaffold that actively interacts with nerve cells to promote regrowth, targeting the scar barriers that normally prevent recovery after paralysis.
Decades-old drug may help prevent Alzheimer’s disease: A study published in Science Translational Medicine found that the decades-old anti-seizure drug levetiracetam reduced production of amyloid-beta 42 in research with engineered mouse models and human neurons. The Researchers found that levetiracetam works by maintaining amyloid precursor protein on the cell surface, favoring a non-amyloidogenic pathway.