Inside Precision Medicine

Protein Degradation Method Paves a Way for New Cancer Therapies Targeted protein degradation is a method that selectively eliminates disease-causing proteins. Scientists at the University of California, Riverside are now using this method to pave a way for new cancer therapies.

Uncovering the Mysteries of the Gut–Brain Connection Links between the gut and the brain were discovered many years ago, but what role does the gut microbiome play in this relationship? Recent research suggests that disruption of or abnormal activity in the gut microbiome may increase our risk for neurological disease, but whether this knowledge can be harnessed to treat, diagnose, or monitor such conditions remains to be seen. https://hubs.li/Q02ZKj-60

Young Age Increases Risk of Distant Recurrence in ER-Positive, ERBB2-Negative Breast Cancers A group of Korean researchers has determined that women under 45 who get hormone-receptor negative breast cancer are at higher risk of distant recurrence even several years after treatment.

Comprehensive Genomic Profiling at Diagnosis Extends Survival in Patients with Advanced Cancer A new study from Providence presents a strong case for integrating complete gomic testing into the early stages of cancer diagnosis. The study demonstrates that implementing genomic profiling as part of the initial diagnostic workup can streamline treatment and increase access to precision therapies that substantially extend survival.

Behind the Breakthroughs: How to Turn $1,000,000 CAR Ts into “Real” Medicines In this episode of Behind the Breakthroughs, Helen Sabzevari, PhD, president and CEO of Precigen, discusses the barriers to implementing cell and gene therapies that impede the accessibility and implementation of precision medicine. Sabzevari also explains how key cell and gene therapy platform tools can provide safety measures, such as guardrails to cellular toxicity, and address cost by offering “off-the-shelf” efficient gene delivery vectors that can be used on-site, cutting out centralized manufacturing. https://hubs.li/Q02ZKhpF0

Novel Vaccine Shows Promise for Triple-Negative Breast Cancer Washington University School of Medicine in St. Louis researchers have developed a neoantigen DNA vaccine that has shown promising results in a phase I trial among patients with triple-negative breast cancer.

Early Research Suggests Congenital Heart Defects Linked to Placenta Research in mice suggests that genetic mutations in the SLC25A1 gene, which plays a role in placental development, could be behind some congenital heart defects. This work is early stage but provides interesting insights into the potential causes of some congenital heart defects and suggests that developing some kind of early treatment could be possible. https://hubs.li/Q02ZJBvm0

Scribe Therapeutics Verifies In Vivo CRISPR-Based Cardiometabolic Therapies For over a decade, Benjamin Oakes has been on a mission to develop a suite of genome and epigenome editing tools built for unique molecular advantages in activity, specificity, and deliverability that translate into safer and more effective genetic therapies, which led him to co-found Scribe Therapeutics in 2018 with Doudna, Savage, and Brett Staahl, PhD. This week at the American Heart Association (AHA) Scientific Sessions 2024, Scribe Therapeutics revealed that their genome editing and epigenetic modifying technologies are knocking on the door of clinical trials for major cardiometabolic diseases affecting millions of patients. https://hubs.li/Q02ZJyL10

Men with Cardiovascular Disease Risk Show Brain Health Decline a Decade Earlier than Women A long-term study by researchers at the Imperial College London and Cardiff University / Prifysgol Caerdydd suggests that men with cardiovascular disease (CVD) risk factors, including obesity, face cognitive decline as early as a decade before similarly affected women. The research, which analyzed data from 34,425 UK Biobank participants, found that men begin to experience significant brain health deterioration from their mid-50s to mid-70s, while women are most vulnerable from their mid-60s to mid-70s. https://hubs.li/Q02ZJbRx0

GLP-1 Obesity Drugs Also Provide Substantial Kidney Protection The biggest and most comprehensive analysis of GLP-1 receptor agonists on kidney and cardiovascular disease shows these drugs have significant benefits in people with and without diabetes. In both types of patients, the combined reduction in the risk of kidney failure, worsening kidney function, and death due to kidney disease was 19%. The study also confirmed that GLP-1 receptor agonists protect cardiovascular health. https://hubs.li/Q02ZJ8vz0