NATIONAL INSTITUTES OF HEALTH

FDA grants Breakthrough Therapy Designation to Allay Therapeutics' ATX101 for post-surgical pain management in adults following total knee arthroplasty (TKA), based on Phase II trial results showing sustained pain relief, reduced opioid use, and enhanced patient satisfaction. Allay plans to start a Phase IIB registrational trial early next year.

Aptose Biosciences Inc. and the National Cancer Institute (NCI) have entered a Cooperative Research and Development Agreement (CRADA) to collaborate on the clinical development of Aptose’s lead compound tuspetinib (TUS) in NCI’s myeloMATCH trials for acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). Tuspetinib, an inhibitor of key signaling kinases, will be part of precision medicine trials aiming to develop tailored drug combinations. Aptose is also developing tuspetinib in a triple drug combination (TUS+VEN+AZA) for newly diagnosed AML patients unfit for chemotherapy.

New research suggests fructose, common in high-fructose corn syrup, fuels cancer cell growth by converting to LPCs, which tumors thrive on, potentially accelerating tumor progression.

Refoxy Pharma secures $9.58 million to develop IPF drug RP-01 targeting FOXO3. Boehringer Ingelheim plans NDA submission for nerandomilast. Insilico's ISM001-055 and Endeavor's ENV-101 show disease improvement. Vicore's buloxibutid and BMS' BMS-986278 also chart progress. Pliant's bexotegrast mitigates chronic cough in IPF. IPOs and partnerships fuel IPF therapeutic field.

Treovir, Inc. opens Phase 2 trial testing G207, an oncolytic HSV immunotherapy, in pediatric brain tumor patients with high grade gliomas at first recurrence. The trial aims to evaluate efficacy and confirm safety, with enrollment currently at Memorial Sloan Kettering Cancer Center and The University of Texas MD Anderson Cancer Center. Treovir expects to expand on positive Phase 1 data, anticipating Phase 2 results to support a BLA filing for G207 market approval.

Dr. Marty Makary, Trump's FDA nominee, questions conventional medical wisdom in his book 'Blind Spots,' advocating alternative treatments and criticizing medical establishment 'groupthink.' His potential leadership could shift FDA focus to food regulation, but his book's selective use of evidence raises concerns about undermining FDA's drug approval standards and public trust. Makary's contrarian views, often based on observational studies, contrast with FDA's traditional reliance on rigorous clinical trials.

Tiziana Life Sciences focuses on immunotherapy with nasal drug delivery, potentially improving efficacy, safety, and tolerability. Intranasal foralumab, a fully human anti-CD3 monoclonal antibody, is under Phase 2a trial for non-active Secondary Progressive Multiple Sclerosis (na-SPMS). The company secured $4 million from NIH for Alzheimer's research and $3.4 million for na-SPMS trials. Ivor Elrifi was appointed CEO, and the FDA expanded the Expanded Access program to 30 patients, with 80% showing reduced microglial activity after six months.

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.