MASSACHUSETTS GENERAL HOSPITAL

Metformin, a drug used to treat Type 2 diabetes, shows potential to slow aging in monkeys, improving cognitive abilities, reducing brain shrinkage, and restoring youthful cellular states. The drug also reduces chronic inflammation and age-related issues in multiple organs. The study bridges rodent and primate research, suggesting metformin could inform future clinical trials aimed at extending health span.

Despite challenges, the HEALEY ALS Platform Trial continues with optimism, as Clene Nanomedicine and Prilenia Therapeutics progress their ALS treatments based on positive secondary endpoint data. The trial, initiated by a $40 million donation from Sean Healey and AMG, aims to efficiently test multiple therapies simultaneously. While primary endpoints have not been met, the trial's design allows for quick identification of non-viable treatments, and positive outcomes in subgroups have supported further development for some candidates.

Harvard experts warn of predatory industries exploiting anti-obesity medication popularity, leading to unregulated supplements and laxatives marketed for weight loss, despite health risks. The high cost of medications drives patients to unapproved alternatives, exacerbating the obesity epidemic and societal weight bias.

BridgeBio Pharma's BBP-812, an AAV9 vector-based gene therapy for Canavan disease, received FDA's RMAT designation based on 12 months of safety and efficacy data from the CANaspire trial. All treated patients showed improvements in functional outcomes and a decrease in N-acetylaspartate levels, with the therapy being well-tolerated. The designation offers hope for children with Canavan disease and their families.

Lipophilic statins reduce HCC risk; hydrophilic statins, aspirin, and metformin do not. A clinical trial (NCT05028829) is ongoing to confirm benefits of atorvastatin.

Trimodal therapy shows potential as an alternative to radical cystectomy for recurrent high-grade T1 bladder cancer, with 88% of patients avoiding cystectomy at 3 years post-treatment. The therapy, involving radiation with radiosensitizing chemotherapy following repeated transurethral resection, reported overall survival rates of 69% at 3 years and 56% at 5 years, with 12% and 19% distant metastasis rates, respectively. Despite limitations in cohort size and participant heterogeneity, the study suggests this approach could be considered in place of cystectomy.

A study by Krishna Reddy and Nancy Rigotti found that immediate availability of cytisine, a tobacco cessation medication, could lead to 71,000 more people quitting smoking and gaining over 500,000 life-years. Each year of FDA delay reduces these gains by 10,000 life-years, emphasizing the need for timely review and approval.

AG Mednet expands leadership with Catherine Tyner as Head of Clinical Strategy and Jamie Russo as Head of Product to meet growing demand for Judi platform, which is used in over 2,000 clinical trials globally.

Intravenous glibenclamide (CIRARA) combined with endovascular thrombectomy (EVT) significantly improves independent ambulation in patients with large hemispheric infarction (LHI), according to phase 3 CHARM trial data. Patients with lesion volumes under 125 mL showed an odds ratio of 4.1 for achieving modified Rankin Scale (mRS) scores of 0-3 (P < .01), and an overall OR of 18.5 for better functional outcomes (P = .03). Despite early termination, CIRARA demonstrates potential as a breakthrough treatment in stroke recovery.

Alzamend Neuro partners with Massachusetts General Hospital for a Phase 2 trial of AL001, an oral therapy for Alzheimer’s-related dementia. The trial aims to determine the minimum effective dose and safety profile of AL001, a novel lithium-delivery system, compared to marketed lithium. Preclinical studies show AL001 prevents cognitive impairment and improves learning and memory in mouse models of Alzheimer’s. A completed Phase 1/2 trial suggests AL001 is well-tolerated and achieves similar blood lithium levels to marketed lithium at lower doses.