UNIVERSITY OF UTAH

University of Utah researchers identified a gene variant linked to high-altitude adaptation in Andean populations, which could serve as a biomarker for predicting blood cancer severity and treatment responses. The NFKB1 gene variant, found in 90% of Aymara people and 30% of Europeans, Hispanics, and Asians, shows lower inflammation and better treatment outcomes in blood cancer patients.

The University of Utah's Wesley Sundquist lab laid the groundwork for lenacapavir, a long-lasting HIV prophylactic named Breakthrough of the Year by Science. Lenacapavir, developed by Gilead Sciences, provides six months of protection and has shown exceptional efficacy in clinical trials, potentially diminishing HIV/AIDS as a global health crisis.

Wesley Sundquist's research on HIV's molecular structure led to the development of lenacapavir, a long-lasting HIV prophylactic. Lenacapavir, effective for six months, has shown 100% efficacy in preventing HIV transmission in clinical trials.

The market for targeted alpha therapies (TATs) has expanded significantly, with Radium-223 dichloride (Xofigo) as a key milestone. TATs are being explored for various cancers and neurodegenerative diseases, with RYZ101 in Phase 3 trials for neuroendocrine tumors and Actimab-A for leukemia. The TAT market is growing due to clinical advancements, investor interest, and regulatory support, positioning TAT as a key component of precision medicine.

ViaLase Inc. partners with John A. Moran Eye Center to study aqueous outflow dynamics using iPerfusion technology, aiming to advance incision-free glaucoma management. The research, led by Ike Ahmed, Fiona McDonnell, and Ian Pitha, explores the effects of multiple channels on intraocular pressure reduction via ViaLase’s femtosecond laser procedure.

Gene therapy targeting cBIN1 protein reversed heart failure in pig trials, improving heart function by 30% and stabilizing or shrinking the heart, a significant advancement in heart failure research.

C22.1: KS1 iPSCs showed lower functional KMT2D expression, H3K4me1/2 levels, and differential gene expression related to KS1 phenotypes. C22.2: Single-cell genomics identified cell-type variation in expression and chromatin, regulatory elements, and eQTLs, facilitating precision medicine for neuropsychiatric disorders. C22.3: XDP-TE in TAF1 gene linked to XDP; ZNF91 establishes H3K9me3 and DNA methylation, crucial for TAF1 regulation. C22.4: MORC2 mutations cause diverse neurological disorders; multi-omics analysis revealed DNA methylation and transcriptome signatures, linking epigenetic silencing to phenotypic manifestation. C22.5: SIRT6 knockdown in Drosophila neurons reduced movement; in neuroblasts, it was lethal, highlighting its role in neurodevelopment. C22.6: Long-read genome sequencing improved diagnostic sensitivity, identified ZFHX3 GGC-repeat expansion as SCA4 cause, and demonstrated abnormal autophagy in affected cells.

Dr. Neeraj Agarwal presented the MEVPRO-1 trial at the 2024 SUO annual meeting, evaluating mevrometostat plus enzalutamide in metastatic castration-resistant prostate cancer (mCRPC) patients previously treated with abiraterone. The trial aims to assess if adding mevrometostat to enzalutamide can reverse anti-androgen resistance, extending clinical benefit. Approximately 600 patients will be randomized 1:1 to receive the combination or enzalutamide/docetaxel, with radiographic progression-free survival as the primary endpoint.

NIH Pragmatic Trials Collaboratory adds LungSMART, a trial addressing lung cancer screening disparities in community health centers via telehealth interventions, supported by a National Cancer Institute grant.

The U.S. Supreme Court will review FDA's denial of flavored e-cigarette sales by Triton and Vapetasia, citing health risks to youths. The FDA appeals a lower court ruling that the agency did not follow proper legal procedures under the Administrative Procedure Act. The case could impact the FDA's regulatory power and the market availability of e-cigarettes.