University of Iowa

Research highlights PGK1 enzyme's role in Parkinson’s disease (PD) energy deficits, suggesting it as a therapeutic target. Studies show PGK1 overexpression restores neuron function and protects against PD-like symptoms in models. However, PGK1 enhancement is ineffective for PD patients with DJ-1 mutations, revealing a functional link between PGK1 and DJ-1.

GE HealthCare's SIGNA™ MAGNUS, a 3.0T high-performance head-only MRI scanner, received FDA 510(k) clearance, offering advanced neuroimaging capabilities for detecting neurological, oncological, and psychiatric conditions. The system features an asymmetrical gradient coil design, HyperG technology, and aims to support advanced imaging and biomarker research.

GE HealthCare's SIGNA MAGNUS 3.0T MRI scanner received FDA 510(k) clearance, offering high-resolution imaging for neuroscience and clinical use, with upgrade options from SIGNA Premier systems.

Perspective Therapeutics reported Q3 2024 financial results, with $267.8 million in cash and investments, expected to fund operations into mid-2026. Key developments include advancing [212Pb]VMT01 trials in melanoma patients and [212Pb]VMT-α-NET in neuroendocrine tumors. The company started shipping from a second manufacturing facility in Somerset, New Jersey, and purchased additional facilities in Houston, Chicago, and Los Angeles. Q3 2024 showed a net loss of $15.1 million ($0.21 per share), with research and development expenses at $12.0 million and general administrative expenses at $7.0 million.

Wave Life Sciences announced financial results for Q3 2024, including first-ever RNA editing in humans with WVE-006 in AATD, and progress in three GalNAc-AIMer preclinical programs for cardiometabolic diseases. WVE-007 for obesity and WVE-N531 for DMD are advancing towards key milestones. FDA feedback on WVE-003 for HD supports potential accelerated approval. Cash and cash equivalents totaled $310.9 million as of September 30, 2024, with runway expected into 2027.

The FDA's approval of Trodelvy for bladder cancer treatment has been withdrawn by Gilead Sciences due to TROPICS-04 study's failure to meet overall survival primary endpoint. This leaves fewer systemic therapy options for urothelial cancer patients, though Trodelvy remains approved for other cancer types. Patients currently on Trodelvy should consult their providers about continuing treatment.

Agendia's FLEX trial, enrolling over 17,000 diverse breast cancer patients, aims to study full genome profiling and clinical data for stages I-III. The study, designed for patient convenience and clinician access, includes racial and ethnic diversity, offering deeper genetic insights and fostering investigator-initiated sub-studies. Key learnings emphasize the importance of diverse patient recruitment, patient-centric design, and advanced data management for clinical trials.

The era of disease-modifying treatments for Alzheimer disease has arrived with FDA approval of 3 medications for mild cognitive impairment or mild Alzheimer disease. Aducanumab, lecanemab, and donanemab target beta-amyloid to slow cognitive decline, with earlier treatment being more effective. These treatments, though promising, come with risks like amyloid-related imaging abnormalities (ARIA) and require careful monitoring and coordination among healthcare providers.

Science cafés, popular since their inception in France and the UK over 25 years ago, aim to make science accessible through relaxed public interactions. These events, often free, feature brief expert talks followed by Q&A sessions, with topics chosen based on community feedback. NIEHS-funded centers at the University of Arizona, Cincinnati, and Iowa have created such cafés to engage communities, share research, and address local environmental health concerns.