UNIVERSITY OF MIAMI

Dr. Maria “Ken” Figueroa, Sylvester's associate director for translational research, connects researchers to bring scientific findings closer to the clinic. She facilitates collaborations through meetings and site visits, and her lab studies cancer and epigenetics. Dr. Figueroa highlights her work on how aging corrupts the epigenome and its contribution to cancer, and her efforts to predict therapeutic outcomes. She emphasizes the circular nature of translational research, involving lab, patient, community, and legislation.

A digital intervention combining text messages and a web-based dashboard, added to health behavior counseling, improved weight-for-length trajectories and reduced obesity incidence at 24 months in a diverse population, compared to counseling alone.

Cindy Medina Pabon discusses the FDA approval of zolbetuximab-clzb (Vyloy) for HER2-negative, Claudin 18.2-positive gastric or gastroesophageal junction adenocarcinoma. Zolbetuximab, combined with chemotherapy, was approved based on SPOTLIGHT and GLOW trials, showing survival benefits. This marks a significant advancement in targeted therapy for gastric cancer.

A Virginia Commonwealth University hepatologist, Dr. Sayed Aseem, received a $445,526 grant to contribute to the WIND-PSC project, aiming to study primary sclerosing cholangitis (PSC) and develop treatments. The project, led by PSC Partners, seeks to enroll 2,000 patients globally to create a comprehensive database for PSC research. PSC, a rare liver disease, currently has no treatments and often leads to liver failure or cancer.

OCU400 gene modifier therapy showed 90% of RP patients stabilized or improved BCVA, and was safe in LCA patients, with no SAEs. OCU400 targets NHR gene NR2E3 for IRDs, with 16 RP patients (81%) experiencing stabilization or improvement in BCVA, LLVA, and MLMT. No SAEs related to OCU400 were reported in LCA patients.

The University of Miami will invest $30 million to enhance research in basic science, neuroscience, and aging, creating a new computational biology program at the Miller School of Medicine and fostering university-wide collaborations.

Barry Farber, a UM alumnus, bequeathed $5.4 million to Sylvester Comprehensive Cancer Center to support the construction of the Kenneth C. Griffin Cancer Research Building, set to open in 2025. His estate also included over $500,000 in immediate-use research funding for head and neck cancers, sarcomas, prostate cancer, and the Firefighter Cancer Initiative.

Alzheimer's disease research saw a decade of stagnation until FDA approved aducanumab in 2021. Since then, therapies like donanemab and lecanemab have shown promise. NKGen's SNK01, an NK cell therapy, and Longeveron's Lomecel-B, an MSC therapy, are in phase 2 trials, demonstrating efficacy and safety. Other novel therapies, including Biogen's BIIB080 and AAV2-BDNF gene therapy, are also in development.

A multidisciplinary team reversed type 1 diabetes in a 25-year-old Chinese patient using reprogrammed stem cell transplant, with the patient achieving insulin independence 75 days post-transplant. This marks a milestone in diabetes treatment, with similar success seen in a type 2 diabetic patient. The procedure, based on a modified stem cell reprogramming technique, aims to avoid immune-suppressing drugs, but more clinical studies are needed to confirm long-term efficacy.

Two UCF College of Medicine cancer researchers, Alicja Copik and Debbie Altomare, received $100,000 each from the Florida Breast Cancer Foundation to support their innovative breast cancer projects. Copik focuses on enhancing natural killer (NK) cells to fight cancer, while Altomare studies cellular pathways influencing cancer growth and immunity. This marks the first time two College of Medicine researchers have received the state cancer support in the same year.