Clinical Trial News

Stanford scientists developed an AI algorithm to predict brain cancer outcomes, aiding in tumor understanding and targeting. Neurooncologist Reena Thomas received a $12M CIRM award for a clinical trial on CAR-T cell immunotherapy for brain cancer.

CAP-1002, an allogeneic cell therapy from donor heart tissue, aims to reduce inflammation, muscle degeneration, and enhance muscle regeneration, potentially prolonging muscle function. A Phase 3 study, Hope-3, is recruiting, sponsored by Capricor Therapeutics.

The CENTAUR trial evaluated sodium phenylbutyrate and taurursodiol (PB and TURSO) for ALS, showing a 4.8-month longer median survival and 36% lower death risk compared to placebo. A post hoc analysis using PRO-ACT database external controls found a 10.39-month longer survival for PB and TURSO, suggesting a greater survival benefit than ITT analysis indicated.

Paradigm collaborates with Bristol Myers Squibb to create a new clinical trial model, aiming to improve patient access, speed up results, and reduce provider burden. Starting with oncology, the initiative focuses on equitable enrollment and efficient trial operations, leveraging Paradigm's network and software to enhance clinical research accessibility and diversity.

FDA’s ODAC voted 14-6, finding sufficient evidence for eflornithine's EFS benefit in reducing relapse risk in pediatric high-risk neuroblastoma patients. Supported by Study 3b and ANBL0032 trial data, eflornithine showed improved EFS and overall survival, despite concerns over non-randomized trial limitations and safety warnings.

Semaglutide, a GLP-1 receptor agonist, improves blood sugar control and promotes weight loss in type 2 diabetes patients over three years. FDA-approved Wegovy for obesity, it's also used off-label for weight loss. Effective in real-world settings, it requires long-term use and professional monitoring, with potential side effects.

Phase III trial for Parkinson's disease terminated, reducing DNL-151's LoA to 14%. LIB Therapeutics' lerodalcibep shows promise in HeFH, increasing LoA to 65%. AstraZeneca's MEDI-7352 completes Phase IIb for osteoarthritis pain, PTSR rises to 45%. Incyte's itacitinib completes Phase II for CRS, PTSR increases to 60%.

Diffuse midline glioma (DMG), a fatal pediatric CNS cancer, resists surgical removal and sees temporary benefits from radiotherapy, with a median survival of 9–11 months. Its 'cold' tumor immune microenvironment, characterized by immunosuppressive myeloid cells and low lymphocyte infiltration, necessitates novel therapies like CAR T cell treatments, currently under clinical assessment for DMG.

NXC-201, a novel anti-BCMA CAR T-cell therapy, showed safety and efficacy in relapsed/refractory AL amyloidosis patients, with a 100% hematologic ORR. However, cardiac disease-related deaths were frequent. CRS occurred in 7 patients, manageable with tocilizumab. The therapy also demonstrated promise in multiple myeloma patients.

FDA revoked authorization for Evusheld due to its ineffectiveness against recent SARS-CoV-2 variants like XBB.1.5, now responsible for most infections. Other treatments remain effective.