VANDERBILT UNIVERSITY MEDICAL CENTER

Sunil Patel and Max Kates present a phase II trial of sequential gemcitabine and docetaxel therapy in BCG-naive non-muscle-invasive bladder cancer patients, achieving 100% complete response at three months and 92% at twelve months. The therapy involves weekly induction followed by monthly maintenance, addressing BCG shortages and offering an alternative to cystectomy. Challenges include coordination with pharmacy and nursing teams, chair time, and treatment protocols, but the combination's effectiveness is attributed to different mechanisms of action and potential immunotherapeutic effects.

Vanderbilt University receives up to $46 million from ARPA-H to develop a vaccine against all alphaviruses, led by Jens Meiler and a multidisciplinary team. The project aims to use advanced technologies like computational modeling and AI for long-lasting protection, addressing a critical gap in global health.

This week's highlights include FDA actions, orphan drug designation, interim data from a CAR-T trial, trends in cell and gene therapy, and biomarker data indicating potential for treating autoimmune diseases.

Poseida Therapeutics' P-BCMA-ALLO1, an allogeneic BCMA-directed CAR-T therapy, showed 91% ORR in a phase 1/1b trial for r/r MM, with no graft versus host disease and low CRS risk. Enhanced lymphodepletion improved in vivo expansion and persistence, addressing unmet needs for patients failing other therapies.

Medtronic and Tempus launch ALERT study to address undertreatment of aortic stenosis and mitral regurgitation using AI, aiming to reduce disparities in TAVR access.

EQBMED publishes Site Maturity Assessment Model in 'Trials' to support clinical trial site growth and diversity, developed over two years with diverse partners and led by Drs. Leslie Curry and Marcella Nunez-Smith.

Poseida Therapeutics’ P-BCMA-ALLO1, an allogeneic BCMA-directed CAR-T therapy, is being evaluated in a phase 1/1b clinical trial for relapsed/refractory multiple myeloma. The therapy, which does not require leukapheresis or manufacturing wait times, demonstrated a 91% overall response rate and no graft versus host disease in the enhanced lymphodepletion arm, as presented at the 21st International Myeloma Society Annual Meeting.

Dietary zinc deficiency exacerbates Acinetobacter baumannii lung infection, linked to increased IL-13 production, potentially treatable with anti-IL-13 antibodies.

A novel drug combination of pazopanib and bevacizumab showed a 54.9% objective response rate and 23.3 months median progression-free survival in metastatic kidney cancer patients, with 64 months median overall survival. Belzutifan, a HIF-2α inhibitor, had manageable safety profile with predominant severe toxicities of anemia and hypoxia. Sitravatinib could not be safely combined with the immunotherapy doublet of nivolumab and ipilimumab at standard doses due to immune-related adverse events.

Day 2 of the 2024 Huntington Study Group Conference featured talks on palliative care, clinical trial challenges, and updates from various drug development companies, emphasizing the importance of early intervention and the use of biomarkers in HD treatment.