Using AI to repurpose existing drugs for treatment of rare diseases:  Identifies possible therapies for thousands of diseases, including ones with no current treatments

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, 42, diagnosed with Stage 4 colon cancer after dismissing first symptom for almost a year:  Dr. Sachin Kulkarni’s life changed thanks to a decision he made once he turned 40

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.

Stem cells reverse woman’s diabetes — a world first:  She is the first person with type 1 diabetes to receive this kind of transplant

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.

FDA official discusses Agency’s new Rare Disease Innovation Hub

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.

Dozens of Babies Have Been Born from Uterus Transplants:  Uterus transplants are becoming more common, opening up the possibility of pregnancy and parenthood to people with certain health conditions

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.

Trump cancels Biden executive order on AI safety

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.

Why Are We Still Excluding Persons With Disabilities (PWDs) In Clinical Trials?

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.

How much will AI help in the next pandemic?

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 Safety of Toxic Metals in Tampons

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.

New clues to how placebo effect works could lead to drug-free treatments for chronic pain:  Scientists “reverse engineer” placebo effect in mice by stimulating brain areas involved in pain-relief response

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.