Three New Glioblastoma Research Advances Target Diagnostics and Immunotherapy

Aptorum Group Limited and DiamiR Biosciences Corp. announced on March 10, 2026 the publication of a paper by Giliberto, et. al. titled "Development of microRNA-Based Glioblastoma Biomarkers Using Blood Plasma Specimens" in Diagnostics, a peer-reviewed journal focused on clinical diagnostics and biomarker research. The study, conducted in collaboration with clinical researchers at the Perelman School of Medicine at the University of Pennsylvania, demonstrates the feasibility of detecting glioblastoma through a minimally invasive blood plasma test. The research identifies specific microRNA signatures in blood plasma that show significant promise as non-invasive biomarkers for glioblastoma diagnosis and monitoring.

GBM is among the deadliest of all human cancers, with a median survival of fewer than 15 months following diagnosis. Current diagnostic approaches rely on invasive surgical biopsies or costly imaging procedures. DiamiR's microRNA platform represents a potential paradigm shift, one that the company believes will enable earlier detection and improve patient monitoring through a simple blood draw.

The senior author on the paper stated that these results are exciting and set the stage for larger clinical studies, which the researchers hope will prove the clinical utility of plasma microRNA signatures for diagnosis and monitoring of brain tumors. Patients desperately need noninvasive tests to help inform their care, and this study is a positive step in that direction.

INOVIO and Akeso have announced a clinical collaboration to evaluate a novel combination immunotherapy regimen for glioblastoma that involves integrating targeted DNA-encoded tumor antigen priming with dual immune checkpoint inhibition. The partnership will investigate INO-5412 (composed of INOVIO's DNA immunotherapy candidates INO-5401 and INO-9012) in combination with Akeso's bispecific antibody, cadonilimab, which targets both PD-1 and CTLA-4 pathways. This combination will be studied within the Phase II adaptive INSIGhT trial sponsored by Dana-Farber Cancer Institute. Initial dosing is expected in the second half of 2026.

The collaborative strategy aims to prime tumor-specific immune responses while countering multiple immunosuppressive mechanisms within the tumor microenvironment. This approach has the potential to overcome the limitations seen with checkpoint inhibition alone. Previous exploratory studies combining INO-5401 and immune checkpoint inhibitors have suggested meaningful immune activation, which has motivated the addition of dual checkpoint blockade in this next phase of investigation.

The use of an adaptive platform trial design like INSIGhT highlights a broader trend toward more efficient screening of experimental therapies in difficult-to-treat cancers by enabling multiple investigational arms to be assessed against a shared control.

Separately, research published in Nature shows that MOV10, an RNA helicase, is overexpressed in glioblastoma and strongly negatively correlated with overall survival. Analysis of datasets from TCGA, GEO, and CGGA showed that MOV10 expression is elevated in GBM. Cox regression confirmed MOV10 as an independent prognostic risk factor for GBM.

Functional enrichment analysis revealed that MOV10 is involved in immune regulation and tumor progression pathways. MOV10 expression is closely linked to immune infiltration, immune checkpoint expression, and responses to immunotherapy. Immunofluorescence and Transwell assays confirmed that MOV10 knockdown reduced M2 macrophage migration and invasion in GBM cells. Clinical analysis further validated MOV10 overexpression in GBM tissues.

In vitro, MOV10 silencing suppressed GBM cell proliferation, inhibited EMT-like processes, and promoted apoptosis through autophagy modulation. The findings suggest that MOV10 plays a crucial role in GBM progression and could be a promising molecular target for therapy.

Aptorum Group Limited is a clinical stage biopharmaceutical company dedicated to the discovery, development and commercialization of therapeutic assets to treat diseases with unmet medical needs, particularly in oncology and infectious diseases. DiamiR Biosciences is a private molecular diagnostics company focused on developing and commercializing minimally invasive tests for early detection and monitoring of brain health conditions and other diseases. DiamiR's proprietary platform technology is protected by over 50 issued patents worldwide and is based on quantitative analysis of organ-enriched, including brain-enriched and inflammation-associated, microRNA signatures in plasma. As was previously announced on July 16, 2025, Aptorum Group and DiamiR entered into a definitive agreement for an all-stock merger transaction, which remains subject to stockholder approval of both companies and other customary closing conditions.