ASCI

@FumihikoUrano @washumedicine Wolfram syndrome and a phase II trial of sodium phenylbutyrate and taurursodiol: Post: jci.org/posts/733 Video: youtu.be/eNXiqYD3uC8

Phase 2 trial of sodium phenylbutyrate and taurursodiol in Wolfram syndrome, a rare and progressive degenerative disease: doi.org/10.1172/JCI198… ASCI member Fumihiko Urano @FumihikoUrano & team @washumedicine show treatment was associated with improved or stabilized pancreatic function, vision, and overall symptom burden. #WolframSyndrome

GLP-1-based therapeutics have revolutionized anti-diabetic and anti-obesity treatments. doi.org/10.1172/jci.in… Here, Wolfram Ruf & team identify the intestinal protease TMPRSS2 as a critical regulator of #GLP1 in an obesity-induced diabetes preclinical model. The image shows colocalization of PAR2 and TMPRSS2 in the mouse small intestine. #Metabolism

ASCI member Kristopher T. Kahle @MassGeneralNews and colleagues provide a Review! 👇

How does caloric restriction reduce energy expenditure while preserving energy storage? doi.org/10.1172/jci.in… Alan R. Saltiel @LabSaltiel & team @UCSanDiego show TBK1 acts as a brake on fat burning during fasting—and the combination of a TBK1 inhibitor and an AMPK activator reverses obesity and improves #MetabolicHealth in mice. The figure: Combined TBK1 inhibition and AMPK activation improve metabolic health in obese mice.

@HopkinsMedicine Severity-dependent atrial remodeling and atrial fibrillation vulnerability in a clinically relevant aortic regurgitation mouse model: doi.org/10.1172/jci.in…

~10% of individuals with cystic fibrosis are unresponsive to CFTR modulators. doi.org/10.1172/jci.in… Here, ASCI member Garry R. Cutting & team @HopkinsMedicine report on gene editing for the correction of a common disease-causing variant in primary CF airway cells. #CysticFibrosis #CF

Spotlighting ASCI member @aadel_chaudhuri 👏

Nature research paper: Non-invasive profiling of the tumour microenvironment with spatial ecotypes go.nature.com/4wf1BFl

This Research Article has high translational potential for #ProstateCancer therapy: doi.org/10.1172/jci.in… Jindan Yu @yu_jindan & team @EmoryMedicine find the new-generation AURKA inhibitor VIC-1911 suppresses prostate cancer growth as a single agent and further increases sensitivity to PARP inhibitors. The figure: VIC-1911 significantly inhibits PC tumor growth either as a single agent or in combination with PARPi. w/@bilenma

This Research Article advances our understanding of how metabolism shapes neuronal development: doi.org/10.1172/JCI197… Ralph J. DeBerardinis & team @UTSWMedCenter generated neural progenitor cells from patients with L2HGDH deficiency, finding L-2HG accumulation epigenetically activates MYC to impair neural differentiation—and the effect is reversible by gene editing or pharmacologically. The figure shows MAP2 (red) and β-III-tubulin (green) in corrected neurons; DAPI (blue). #Neurodevelopment

A landmark in cardiac metabolism: doi.org/10.1172/JCI202… Jay D. Horton & team @UTSWMedCenter find unrestrained fat oxidation in heart cells deplete cardiolipins that are needed for mitochondrial energy, triggering heart failure in mice and revealing a potential metabolic target. The figure shows decreased heart function in cardiac-specific ACC1/ACC2 double KO (dHKO) mice. #CardiacMetabolism #HeartFailure

Deeply grateful to be elected into the American Society for Clinical Investigation (ASCI) — a reflection of the extraordinary group of trainees and colleagues @SmithBIDMC and beyond. @the_asci

Congratulations @rkwadhera on your induction into @the_asci, in recognition of his exceptional contributions to cardiovascular and health policy research! Read more here: smithcenter.bidmc.org/news/dr-rishi-…

Join us in celebrating #WashUMedCRTC alumni Suzanne Schindler, MD, PhD; Christopher Smyser, MD; and Sidharth (Sid) Puram, MD, PhD on their recent election to @the_asci! Learn more about this distinguished honor: bit.ly/4ubrSD2 @WashUMedENT @WashUNeurology

✅Clear association with disease severity ✅Mechanistic insight ✅Robust multi-platform design doi.org/10.1172/JCI194… In this Research Article, Deepak A. Rao @Deepakarao & team @BrighamWomens implicate CD57+ T effector memory cells as a driver of lung injury in systemic sclerosis-interstitial lung disease (SSc-ILD) and potentially other forms of ILD. The figure shows CD57+, CD8+, and CD45RA+ staining of SSc-ILD lung tissue.

#StanDOM's @Joseph_C_Wu receives @the_asci's 2026 Stanley J. Korsmeyer Award, which recognizes physician-scientists for scientific excellence, innovation & mentorship. Wu delivered the Korsmeyer Lecture, "Genomics, AI & NAMs for Drug Discovery" at ASCI's annual meeting.

“Being inducted into @the_asci is a tremendous honor and a meaningful acknowledgement of the importance of our work to understand the pain experience and its underlying mechanisms in individuals with #autoimmune #RheumaticDiseases. I am deeply grateful to the mentors, collaborators, trainees, and the patients whose partnership and insight inspire the questions we pursue,” Yvonne Lee, MD, MMSc, said. news.feinberg.northwestern.edu/2026/04/15/fei…

Congratulations to Drs. @SoleimanpourLab and @adina_turcu, who were recently inducted into The @the_asci! Learn more about the society and their research at beta.the-asci.org.

“It’s a tremendous honor to be part of such a historic and prestigious organization. @the_asci has long been home to physician-scientists who have fundamentally changed the way we understand and treat disease, and I’m deeply grateful to join their ranks,” said Seth M. Pollack, MD (@immunosarc), who is also a co-leader of the #CancerImmunology and #Immunotherapy Initiative at @LurieCancer. Read more: news.feinberg.northwestern.edu/2026/04/15/fei… #SarcomaImmunobiology