P1/2, N=20, Recruiting, National Institute of Allergy and Infectious Diseases (NIAID) | Trial completion date: Apr 2026 --> Dec 2026 | Trial primary completion date: Dec 2025 --> Dec 2026

P1/2, N=12, Not yet recruiting, Hospital Israelita Albert Einstein

We propose that a CXCR4 inhibitor (AMD3100) could decrease cancer stemness and improve the efficacy of the anticancer drug gemcitabine (GEM) in targeting hypoxic pancreatic ductal adenocarcinoma (PDAC) cells. The combined treatment significantly elevated pro-apoptotic BAX mRNA levels and reduced antiapoptotic BCL2 mRNA levels. Additionally, the combination therapy decreased the size of cancer cell spheroids from PANC1 and patient-derived cells.

G-CSF drives CC progression by enhancing H3K27ac-dependent upregulation of KLF5, which transactivates CXCR4 to promote EMT, proliferation and metastasis. Targeting the G-CSF/KLF5/CXCR4 axis may represent a potential therapeutic strategy for advanced CC.

Despite particularly low pre-collection peripheral blood CD34 counts, successful autologous stem cell collection in MM patients is feasible by routinely adding plerixafor to granulocyte-colony stimulating factor on day 4 of mobilization. There is limited analysis demonstrating that sufficient stem cells for one or more transplants can be collected using this method. This practical and novel approach may benefit the high number of MM patients who face limited resources, finances, long travel times, and social support. These results are highly relevant to physicians treating similar patients.

These nanocapsules are stabilized by PAMD-C, which is a cholesterol-modified polymeric analog of the FDA-approved CXCR4 antagonist AMD3100 (plerixafor). Treatment with therapeutic miR-34a mimic suppresses metastatic outgrowth, potentiates anti-tumor immunity, and doubles median survival relative to control paclitaxel chemotherapy. By combining unique PFC disposition features with RNA versatility, the delivery platform overcomes main biological barriers for inhalable RNA medicines and opens a translatable path for treating diverse pulmonary diseases.

An algorithm-driven, G-CSF-only mobilization with pre-emptive Plerixafor use based on CD34 counts resulted in 100 % successful collections, early engraftment, minimal plerixafor use, and no mobilization failures. This approach avoids chemotherapy toxicity, optimizes Plerixafor use, and prevents transplant delays.

P3, N=60, Recruiting, Guangzhou Gloria Biosciences Co., Ltd. | Not yet recruiting --> Recruiting | Trial completion date: Dec 2026 --> May 2027 | Trial primary completion date: Aug 2026 --> Dec 2026

A proof-of-principle drug screen identified PF 573228 (FAK inhibitor) and motixafortide (CXCR4 inhibitor) as potent suppressors of GBM20 and GBM1 infiltration, respectively. Bulk RNA sequencing revealed gene expression profiles correlating with invasive behaviour and drug sensitivity. This platform offers a valuable model for studying glioblastoma infiltration along axons and provides proof-of-principle that migration can serve as a measurable and actionable phenotype to screen therapeutic vulnerabilities in glioblastoma.

Imaging of CXCR4 with specific PET and the selection of patients on CXCR4 biomarker criteria offer the possibility of further improving precision medicine approaches so that CXCR4-targeted therapies will only be given to the most CXCR4-responsive patients. The role of CXCR4 in lung cancer pathogenesis and development is critically reviewed, the most recent results on plerixafor inhibition of CXCR4 are summarized, and new, potential strategies for combination treatment of CXCR4 with other inhibitors are explored.

This study provide new insights into the molecular basis of BC and support the idea of targeting TGFβ-1 and CXCR4 together for therapy. The above findings lay the foundation for future in vitro and in vivo experiments aimed at demonstrating that inhibition of both factors would be a viable strategy to improve the therapeutic outcome in BC.

P2, N=100, Recruiting, Thomas Jefferson University | Not yet recruiting --> Recruiting