An unknown number of probationary staff at the US Food and Drug Administration (FDA) were fired this past weekend as part of the Trump Administration’s effort to shrink the size of the federal government. While the administration has not provided any specifics on the scope of the reductions, the round of firings, which affected staff across several federal health agencies, targeted workers with fewer employment protections.
First new schizophrenia drug approved in decades: KarXT, the first new schizophrenia medication granted approval in decades, introduces a novel mechanism that activates brain muscarinic receptors to slow the release of dopamine, demonstrating antipsychotic and cognitive benefits without many of the side effects of older drugs. While the drug’s high price and twice-daily dosing regimen present challenges for patients, researchers are working to create alternatives with easier dosing schedules and investigating selective activation of muscarinic receptors to deliver either cognitive or antipsychotic benefits.
Artificial Intelligence (AI) tool goes beyond conventional tools for repurposing drugs: An AI tool called TxGNN identified potential treatments for over 17,000 rare and undiagnosed diseases from about 8,000 medications and predicted side effects and contraindications, according to a study in Nature Medicine. The new tool surpassed the predictive capabilities of conventional models by training on shared genomic features across multiple diseases, improving its ability to extrapolate effective treatments from well- or poorly understood conditions to accelerate the repurposing of drugs.
Doctor aims to support people with cancer after his own diagnosis: Physician Sachin Kulkarni did not expect to be diagnosed with stage 4 colon cancer after deciding it was time for a checkup when he turned 40; however, he approached treatment methodically, thinking about who he was fighting for and recognizing he had to take it one step at a time. Now, with no evidence of disease, Kulkarni is giving back to the community that supported him, advocating and supporting those with cancer and survivors.
Woman with T1D (Type-1 Diabetes) produces insulin after stem cell treatment: A 25-year-old woman with Type 1 Diabetes produced her own insulin after undergoing a transplant using stem cells derived from her body, which were reprogrammed into a pluripotent state, differentiated into 3D islet clusters and then injected into her abdominal muscles for improved monitoring and potential removal. The study, published in Cell, reports that the woman has been maintaining stable blood glucose levels and producing sufficient insulin for over a year without the need for additional injections, although some experts would like to see her producing insulin for five (5) years before considering her cured.
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.