NATIONAL INSTITUTES OF HEALTH

A clinical trial led by Keith McCrae, M.D., at Cleveland Clinic demonstrated that pomalidomide, a cancer drug, is safe and effective for treating hereditary hemorrhagic telangiectasia (HHT), a rare bleeding disorder. The trial, supported by the National Institutes of Health, was stopped early due to successful findings and published in the New England Journal of Medicine. Pomalidomide significantly reduced nosebleed severity, required fewer blood transfusions, and improved quality of life for HHT patients.

Lack of funding and understanding of long COVID's mechanisms hinder drug development, frustrating patients and researchers. Despite initial positive results with Ampligen, Aim ImmunoTech faced funding challenges. The NIH's RECOVER initiative focuses on observational studies rather than new compounds, and private investors avoid long COVID due to perceived risk. Small biotech companies struggle to sustain research efforts, with some shifting focus or shutting down. Public-private partnerships and increased federal funding are suggested as solutions to advance long COVID treatment research.

Thomas A. Russo identified genetic elements transforming classical Klebsiella pneumoniae into hypervirulent strains, which can infect healthy people and become drug-resistant. Research published in eBioMedicine highlights pVir plasmid as the primary genetic determinant of hypervirulence, with potential therapeutic targets for countermeasures.

80% of advanced-phase CML patients achieved bone marrow remission with decitabine, venetoclax, and ponatinib in a Phase II trial, according to MD Anderson researchers. The trial included patients with accelerated or myeloid blast phases of CML and Philadelphia chromosome-positive AML.

USC's Keck School of Medicine launches a $2.7 million NIH-funded, multi-site study to understand how type 1 diabetes affects brain development in children, aiming to refine clinical guidelines and therapeutic approaches.

NIH funding supports UMD studies on treating HIV, substance use, and depression comorbidities, exploring long-acting HIV treatments and behavioral activation therapy delivered by peers.

FDA approves Merck's Keytruda for malignant pleural mesothelioma in combination with chemotherapy, improving survival rates in a Phase 3 trial.

NIH-led studies identify RNF114 protein reversing cataracts in 13-lined ground squirrels and rats, suggesting a potential surgery-free treatment for cataracts, published in the Journal of Clinical Investigation.

The article profiles various professionals in health and statistics, including John R. Lumpkin, M.D., M.P.H., Mollyann Brodie, Ph.D., Kennon R. Copeland, Ph.D., Tara Das, Ph.D., M.P.H., M.L.I.S., Scott H. Holan, Ph.D., V. Joseph Hotz, Ph.D., Bradley Malin, Ph.D., Sally C. Morton, Ph.D., Lucila Ohno-Machado, M.D., M.B.A., Ph.D., Andy Peytchev, Ph.D., C. Matthew Snipp, Ph.D., Kelly Hoover Thompson, J.D., and David R. Williams, Ph.D., detailing their roles, achievements, and contributions to their respective fields.