BAYLOR COLLEGE OF MEDICINE

Dr. Seth Lerner received the Huggins Medal at the 2024 SUO annual meeting, reflecting on Dr. Charles Huggins' contributions and team science. Lerner highlighted his experiences in team-based research, including the Bladder Cancer Research Consortium and the TCGA MIBC project, emphasizing the importance of collaboration and validation studies. His work with SWOG and NCTN led to numerous clinical trials and insights into bladder cancer molecular subtypes and treatment responses. Lerner also discussed his involvement with the FDA, AUA, and SUO in establishing guidelines for single-arm trials in BCG-unresponsive NMIBC.

FDA grants Orphan Drug Designation to Capsida Biotherapeutics for CAP-002, a gene therapy for STXBP1 disorder, developed with Dr. Mingshan Xue at Texas Children’s Duncan Neurological Research Institute. The therapy aims to treat a rare developmental and epileptic encephalopathy affecting up to one in 26,000 children globally, with plans to file an Investigational New Drug application in 2025.

Baylor College of Medicine received $3.5 million from the National Human Genome Research Institute for the MAGNET program to improve genetic diagnoses in NICUs across underserved Texas regions using low-cost whole genome and RNA sequencing. The program aims to adapt Baylor’s Consultagene platform for in-patient use, offering virtual genetic counseling and provider consultation, and will train on-site providers in genomic medicine to integrate personalized treatments. The MAGNET program seeks to develop best practices for implementing cost-effective genomic platforms in under-resourced settings.

Researchers at Baylor College of Medicine and Rice University received a $2.3 million Breakthrough Award to develop a NanoGel antibody therapy targeting ER+ breast cancer metastasized to bone. The NanoGel releases antibodies in the acidic bone environment to target E-cadherin proteins, aiming to prevent further metastasis.

Bioethicists from the NIH Pragmatic Trials Collaboratory recommend sharing aggregate results from pragmatic clinical trials with participants, emphasizing early planning and healthcare system partnerships for effective disclosure.

A new open-source image analysis platform, SPACe, developed by researchers at Baylor College of Medicine, Texas A&M University, and the University of Houston, provides a computational resource-efficient tool to analyze large datasets from cell painting assays, capturing diverse single cell responses and revealing new avenues for drug development.

FDA approves Imkeldi, a liquid imatinib formulation for leukemia and cancers, aiding dosing accuracy and adherence. A breast cancer vaccine targeting triple-negative breast cancer showed positive immune responses in a phase 1 trial, with phase 2 planned for 2025. Brexucabtagene autoleucel demonstrated high CNS remission rates in relapsed/refractory B-cell acute lymphoblastic leukemia. The FDA accepted darolutamide's supplemental application for metastatic hormone-sensitive prostate cancer, based on ARANOTE trial data. Ribociclib combined with NSAI improved outcomes for high-risk, node-negative, early-stage breast cancer patients in the NATALEE trial.

Sittig and Singh emphasize the need for safe AI implementation in healthcare, advocating for robust governance, rigorous testing, multidisciplinary oversight, clinician training, and transparency to mitigate risks and build trust.

First-in-human phase 1 clinical trials of GPC3-specific CAR T cells co-expressing IL-15 for solid tumors showed 33% objective antitumor response and 66% stable disease, with increased CAR T cell expansion and higher cytokine release syndrome incidence.

Greenwich LifeSciences partners with Italy's Gruppo Italiano Mammella (GIM) for Phase III FLAMINGO-01 trial evaluating GLSI-100 immunotherapy to prevent breast cancer recurrences. Nine top GIM institutions join the trial, with Italy being the first country to have all participating sites personally visited and trained by the company.