Clinical Trial News

Bracha et al. engineered Toxoplasma gondii to deliver large proteins to mouse brains, observing efficient delivery of various therapeutic proteins. They also demonstrated simultaneous delivery and characterized the system using MeCP2, showing functional protein delivery in brain organoids and mice, with highest levels in the cortex.

A patient under Dr. Scott Roberts' care tested positive for SARS-CoV-2 for over 500 days due to severe immunocompromise. The FDA authorized pemivibart for preexposure prophylaxis in immunocompromised individuals, and IDSA updated its guidelines to recommend its use. Despite concerns about efficacy against new variants, pemivibart offers a new preventive option for high-risk patients.

Lenvatinib plus pembrolizumab showed 88.4% 5-month PFS in platinum-refractory advanced B3-thymoma and thymic carcinoma, with median PFS of 14.9 months. The combination outperformed monotherapies, suggesting it could be a new standard treatment. Subgroup analyses indicated benefits regardless of age, gender, or histologic subtype, but significant benefits were seen in patients without liver metastases and those without lenvatinib dose reduction. Grade 3 or higher AEs occurred in 46.5% of patients, emphasizing the need for close monitoring.

A randomized clinical trial involving 757 participants found that the complete multicomponent Symptom Care at Home intervention, including automated self-management coaching and clinician follow-up for moderate and severe symptoms, achieved the greatest reduction in symptom burden compared to individual components alone. This suggests that multicomponent digital approaches to cancer symptom management offer optimal symptom burden reduction during chemotherapy treatment.

Fixed-duration treatments differ from continuous therapy in CLL. Venetoclax-based regimens are approved for 12 cycles with obinutuzumab in frontline and 24 cycles with rituximab in relapsed/refractory settings.

Cleveland Clinic study on GLP-1 receptor agonist medications like semaglutide and liraglutide finds semaglutide more effective for long-term weight loss, especially for obesity treatment. Higher doses and persistence with medication significantly impact weight reduction. Women and those with higher initial BMI more likely to achieve 10% weight loss.

VO659, an allele-preferential antisense oligonucleotide therapy, showed a 28% reduction in mutant huntingtin protein in cerebrospinal fluid among Huntington disease patients in a phase 1/2 trial, with no neuroaxonal damage observed. The therapy is safe, well-tolerated, and has a long half-life, suggesting infrequent dosing potential.

Ozempic and Wegovy (semaglutide) outperformed Saxenda (liraglutide) in weight loss, with 61% of obese and 23% of diabetic patients losing 10%+ body weight after a year. Saxenda achieved similar results in only 29% of obese and 12% of diabetic patients. Semaglutide users were more likely to lose 10%+ weight if treated for obesity, adhered to the drug schedule, had a higher BMI, and were female. Higher doses produced more weight loss.

AstraZeneca Korea launched Truqap, the first AKT inhibitor for HR+/HER2- breast cancer, targeting PIK3CA, AKT1, or PTEN mutations. Truqap, approved in April, offers new hope for patients progressing after endocrine therapy. The CAPItello-291 study showed Truqap plus fulvestrant doubled median progression-free survival to 7.3 months. Despite financial concerns over NGS costs, global guidelines recommend mutation testing for effective treatment.

Nebulized colistimethate sodium showed mixed results in bronchiectasis and Pseudomonas aeruginosa infections, with PROMIS-I showing reduced exacerbation rates (RR 0.61) and PROMIS-II showing no difference (RR 1.00). COVID-19 disruptions in PROMIS-II were identified as the cause for the differing outcomes. A meta-analysis supported colistimethate sodium's efficacy in reducing exacerbation rates (RR 0.65). Despite inconsistent trial results, inhaled antibiotics for bronchiectasis have received conditional endorsement in guidelines.