NATIONAL INSTITUTES OF HEALTH

Countries worldwide are advancing genomic medicine, with the UK leading through the 100,000 Genomes Project, the US investing heavily in research via NIH, France implementing the French Plan for Genomic Medicine 2025, Germany launching GenomDE, and the UAE and Australia also making significant strides. These initiatives aim to improve patient outcomes through personalised treatments, requiring adequate funding, clear regulatory pathways, and ethical safeguards.

CVI visuospatial deficits involve differential recruitment of dorsal and ventral visual streams, with ventral stream showing greater activation in CVI patients, suggesting a complex clinical profile.

Neurologist Kelly Gwathmey launched the VCU Health Rapid Access ALS Program to reduce up-to-15-month ALS diagnosis waits. The program offers immediate expert access and same-day testing, aiming to improve patient outcomes through early diagnosis and multidisciplinary care. Gwathmey highlights racial disparities in ALS diagnosis delays, with Black patients waiting 65% longer. The program also educates providers on ALS symptoms and promotes early referral to the clinic.

The PAN Foundation launched the Opening Doors to Clinical Trials initiative to improve equitable access, offering education, a trial finder tool, and live assistance. The initiative aims to address historical underrepresentation in trials, particularly among Black, Hispanic, and LGBTQIA+ communities, by providing resources and personalized support.

A clinical trial led by UVA Health's David Schiff, MD, found that combining temozolomide with radiation significantly improves 10-year survival rates for adults with grade 2 gliomas, from 47% to 70%. This discovery changes treatment protocols, as previous trials lacked evidence supporting temozolomide's benefit in these tumors.

UCSD researchers demonstrated GPT-4's effectiveness in automating functional genomics, achieving 73% accuracy in gene set enrichment analysis, and highlighting AI's potential in precision medicine.

Achieve Life Sciences announces successful End-of-Phase 2 FDA meeting for cytisinicline as a potential first-in-class vaping cessation therapy. The FDA agreed that one Phase 3 trial (ORCA-V2) along with the completed Phase 2 ORCA-V1 trial would suffice. The planned Phase 3 study will evaluate 3 mg cytisinicline three times daily versus placebo in approximately 800 adults who use e-cigarettes, targeting initiation in Q3 2025.

Aptose Biosciences Inc. and the National Cancer Institute collaborate on tuspetinib clinical development for AML and MDS in myeloMATCH trials, aiming to expedite tailored drug combinations.

Aptose Biosciences signs CRADA with NCI to develop tuspetinib for AML and MDS in myeloMATCH trials, aiming for tailored drug combinations. Separately, Aptose plans Phase 1/2 TUSCANY study for triple drug combo (tuspetinib, venetoclax, azacitidine) in AML patients unfit for chemotherapy, set to begin Q4.

Researchers at the University of Michigan are exploring the role of pets in the lives of LGBTQ cancer patients, aiming to understand how pets contribute to wellbeing, connectedness, and care during treatment and recovery. The study involves a multidisciplinary team and includes pilot data collection, with plans for focus groups to delve deeper into the impact of pet ownership on social isolation and health outcomes.