NATIONAL INSTITUTES OF HEALTH

Nicholas Douville, M.D., Ph.D., and team aim to use precision medicine for better surgical care, exploring genetic factors in perioperative outcomes. Despite initial lack of strong genetic associations in acute kidney injury, they turn to the diverse All of Us Research Program dataset, proposing tools to map surgical procedures and study emergent health conditions, emphasizing genetics as one factor in precision medicine.

Dr. Gary Gibson hypothesizes that thiamine deficiency alters glucose metabolism in brain cells, leading to Alzheimer's. His research led to a nationwide clinical trial evaluating benfotiamine, an oral drug boosting thiamine levels, as a potential treatment.

2024 saw breakthroughs in gene therapies for sickle cell disease, at-home flu vaccines, mind-controlled prosthetics, non-opioid pain medicine, AI medical scribes, fruit fly brain mapping, clinical-grade hearing aids, new schizophrenia drugs, brain-computer interfaces, HIV prevention meds, cervical cancer screening alternatives, maternal health apps, AI protein structure prediction, VR for mental health, screen time reduction tools, gene therapy for deafness, women's health research, self-serve vision tests, blood tests for colorectal cancer, and potential lupus cures.

Vagus nerve stimulation therapy showed significant improvement in depressive symptoms, quality of life, and daily functioning for patients with treatment-resistant depression, according to a national clinical trial led by WashU Medicine. The study involved nearly 500 participants, with activated devices showing more time with improved symptoms and better quality of life. The therapy, if covered by CMS, could become more accessible.

In 2024, KU Medical Center strengthened partnerships, increased nursing workforce, and welcomed new leadership. Key research included precision medicine, muscular dystrophy drug trials, and Alzheimer's prevention. Historic milestones were celebrated, and efforts to improve health disparities continued. Construction began on the Wichita Biomedical Campus, and faculty and students engaged in global health initiatives. Academic programs were nationally recognized.

Tiziana Life Sciences announced dosing the first patient with intranasal foralumab for moderate Alzheimer’s disease, targeting brain inflammation. Foralumab, a fully human anti-CD3 monoclonal antibody, stimulates T regulatory cells to reduce neuroinflammation. This approach differs from beta amyloid or tau protein reduction strategies and represents a novel treatment avenue for Alzheimer’s disease.

Martin A. Makary, Trump's nominee for FDA commissioner, argues for reforming the Orphan Drug Act, criticizing drug companies for exploiting it with mainstream drugs. He believes orphan drugs should have single uses in rare diseases, not generate significant profit, and not be used for over 200,000 patients. Makary also opposes government-provided monopolies, suggesting they should incentivize innovation without excessive market control. His proposals include taxing orphan drug revenues and reducing exclusivity when revenues exceed $1 billion, though these require legislative action.

New research from the University of Chicago uses advanced imaging to study the structure of adhesion G protein-coupled receptors (aGPCRs), revealing how their extracellular regions interact with transmembrane regions, potentially opening new avenues for drug development targeting these complex receptors involved in diseases like cancer and brain disorders.

The 3D Cell Culture Market, valued at USD 1.4 billion in 2023, is projected to reach USD 4.0 billion by 2032, growing at a 12.4% CAGR. 3D cell cultures offer a more in vivo-like environment, crucial for drug discovery, personalized medicine, and oncology. Government investments and rising cancer rates drive demand, with North America leading and Asia-Pacific emerging as a fast-growing region.