Plerixafor was effective and well tolerated in pediatric patients with solid tumors, regardless of disease status (newly diagnosed or relapsed). These findings support its potential as a valuable stem cell mobilization option in pediatric transplant settings in Japan.

We developed a new C-X-C chemokine receptor 4 (CXCR4)-targeting radiolabeled peptide, [68Ga]Ga/[177Lu]Lu-BL34, using a novel and potent cyclic peptide based on structure-activity relationship studies of LY2510924... We report a new CXCR4-targeting pharmacophore that can be used as a radiotheranostic. [68Ga]Ga-BL34 and [177Lu]Lu-BL34 showed excellent imaging and therapeutic properties in preclinical studies and are promising candidates for clinical translation.

In conclusion, CTX + PEG-rhG-CSF + on-demand PXF is a highly effective salvage mobilization strategy for patients with initial HSC mobilization failure, yielding higher CD34+ cell counts with fewer aphereses and acceptable safety. With balanced advantages in efficacy, safety, and cost-effectiveness, the CTX + PEG-rhG-CSF + on-demand PXF regimen can be a preferred salvage option for ASCT candidates with prior mobilization failure.

Combining a small-molecule drug screen and RNAseq analysis, we show that cxcr4b expression is increased in nf1 mutants and that pharmacological inhibition of Cxcr4 with AMD3100 (Plerixafor) improves habituation learning...CXCR4 was previously identified as a potential therapeutic target for neurofibromin-deficient tumorigenesis. Our results suggest that Cxcr4 signaling also regulates neurofibromin-dependent cognitive function.

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