MASSACHUSETTS GENERAL HOSPITAL

Harvard's Joel F. Habener wins 2024 Lasker-DeBakey Award for discovering GLP-1 hormone, key to diabetes drugs like Ozempic. Habener, Svetlana Mojsov, and Lotte Bjerre Knudsen's work led to treatments revolutionizing obesity and type 2 diabetes. GLP-1 receptor antagonists offer benefits beyond blood sugar control and weight loss, potentially treating heart disease, inflammatory disorders, addictions, and depression.

Steritas LLC's pGTI will be used in the STAR-JIA trial to compare efficacy, safety, and cost-effectiveness of intravenous vs. oral glucocorticoids for JIA in children. The trial aims to assess steroid toxicity and is funded by the National Institute for Health and Care Research.

ARS Pharmaceuticals announces the U.S. availability of neffy® (epinephrine nasal spray) for treating Type I Allergic Reactions, including anaphylaxis, in adults and children weighing ≥30 kg. neffy is accessible through various programs, including a $25 co-pay for insured patients and a $199 cash price for uninsured patients. ARS also plans to expand access to EURneffy® in the EU by Q4 2024 and has submitted an FDA application for pediatric use.

Jason Efstathiou discusses the PARTIQoL trial, comparing proton beam therapy to IMRT for localized prostate cancer, focusing on patient-reported outcomes and bowel function at 24 months. The trial, involving 450 patients from 29 centers, employs innovative strategies to overcome accrual challenges, emphasizing rigorous data generation to assess proton therapy's benefits. Despite proton therapy's potential advantages, its limited availability and high costs in the U.S. highlight the need for comparative studies like PARTIQoL.

Troriluzole met primary endpoint in SCA study, showing significant f-SARA improvements. Biohaven plans to submit an NDA to FDA for all SCA genotypes. Troriluzole demonstrated 50-70% slower disease progression, delaying decline by 1.5-2.2 years. Treatment showed consistent efficacy across 3 years, with a 58% reduction in fall risk.

Troriluzole treatment in SCA patients shows 50-70% slower disease progression, delaying progression by 1.5-2.2 years over 3 years. A responder sensitivity analysis revealed an odds ratio of 4.1 (95% CI: 2.1, 8.1) for untreated vs. troriluzole-treated patients (p < 0.0001). Biohaven plans to submit an NDA to the FDA in Q4 2024 for troriluzole in SCA.

One in three former NFL players believe they have CTE, reporting more brain function issues, depression, and suicidal thoughts, compared to those without such beliefs.

AI in cancer care may aid early diagnosis, reduce radiologist workload, assist in clinical trial recruitment, manage data, and determine imaging responses. However, limitations include privacy, reliability, and cost. AI's broad applications span clinical trial design, treatment response, triaging, imaging, pathology, genomics, and more. AI models like Sybil can predict lung cancer risk from a single low-dose CT scan. Despite potential, human oversight remains crucial due to AI's lack of pathophysiological understanding.

Habener and Mojsov, honored with the Lasker Award, pioneered GLP-1 research, leading to diabetes and obesity drugs like Ozempic. Their work began with studying glucagon and pivoted to fish research, revealing GLP-1's potential. Mojsov's predictions and synthesis methods identified GLP-1's therapeutic effects, later developed by Knudsen into long-lasting treatments. GLP-1 drugs now show broader health benefits, including potential for dementia protection.