Inside Precision Medicine

New Method Shows Promise to Deliver Therapeutics Across the Blood-Brain Barrier Researchers at the Icahn School of Medicine at Mount Sinai say they have developed a novel platform to overcome one of the biggest challenges in brain disease treatment: getting macromolecule therapeutics to cross the blood-brain barrier (BBB). Their study, published today online in Nature Biotechnology, describes a new method that safely and effectively delivers large therapeutic molecules into the brain—a discovery that could pave the way for treating a range of neurological and psychiatric disorders, including amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, and various forms of addiction. https://hubs.li/Q02Zmf4C0

BridgeBio’s Attruby Approved for Rare Heart Disease Last week, the FDA approved BridgeBio’s Attruby (acoramidis) for adults with Transthyretin amyloid cardiomyopathy (ATTR-C)—a rare disease that can cause serious heart problems and death. Attruby is the first and only approved treatment with a label specifying near-complete stabilization of Transthyretin (TTR). The company also said Attruby demonstrated the most rapid benefit seen in any Phase III study of ATTR-CM to date. https://hubs.li/Q02ZlRSK0

Spatial Transcriptomics Offer New Insights into Alzheimer’s Disease Researchers at the University of California (UC) Irvine have conducted a transcriptomics analysis of Alzheimer’s disease, looking at the spatial differences in gene expression found across the brains of patients with early-stage and late-stage Alzheimer’s, as well as Alzheimer’s disease in Down syndrome. Their results, published in Nature Genetics, uncovered new clues about disease processes that are intimately tied to specific cell types and brain regions. The data could be used to prioritize gene targets with higher translational potential in clinical development. https://hubs.li/Q02ZhdNX0

AMP Expo Chatter: Can AI Algorithms Be LDTs? Conversations at the conference raised awareness of a growing issue in molecular pathology and diagnostics: the use of AI in lab-developed tests. https://hubs.li/Q02ZhdNT0

New Approach Revives Exhausted T Cells to Continue Cancer Fight The study, from the University of Pittsburgh and UPMC Hillman Cancer Center shows that blocking the protein responsible for transporting lactic acid into T cells not only improves the function of T cells cells but also improved tumor control in mouse models of cancer.

Cure51 Launches Major Study of Cancer “Super-Responders” Researchers are “seeking to unlock the biological mechanisms responsible for exceptional cancer survivors.” The study will involve Cure51 partnering with nine leading U.K. hospitals: Cambridge University Hospitals NHS Foundation Trust, including: The Royal Marsden NHS Foundation Trust, The Christie NHS Foundation Trust, University Hospital Southampton NHS FT, The Beatson West of Scotland Cancer Centre, Velindre University NHS Trust (NHS Wales), Barts Health NHS Trust, University Hospitals Birmingham NHS Foundation Trust, and Leeds Teaching Hospitals NHS Trust.

Skin Tone Bias Reduces Accuracy in Photoacoustic Imaging for Breast Cancer Detection A new study from The Johns Hopkins University highlights how skin tone can affect the accuracy of photoacoustic imaging, a technique used in breast cancer diagnostics.

EZH2 Enzyme Linked to Drug Resistance and Tumor Growth in Prostate Cancer Researchers at Weill Cornell Medicine have uncovered a surprising role for the enzyme EZH2 in driving aggressive tumor growth in prostate cancers resistant to treatment.

Cancer-Like Genetic Mutations Found in Healthy Breast Tissue Cells The findings suggest that cancer-like mutations appear silently in breast cells that appear normal, potentially offering insights into the origins of the disease. The researchers are from The University of British Columbia, BC Cancer, Harvard Medical School, and Memorial Sloan Kettering Cancer Center.

Novel AI Tool “Sees” Gene Expression in Tumor Biopsy Images A tool created by Stanford University School of Medicine researchers using artificial intelligence can detect gene expression levels within tumor cells using just standard microscopy images of the tumor biopsy, and can predict a patient's risk for breast cancer recurrence.