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ASH 2025 | Phase I study of motixafortide +/- natalizumab to mobilize HSCs for gene therapy in SCD
Zachary Crees, MD, Washington University School of Medicine, St. Louis, MO, discusses a Phase I study (NCT05618301) demonstrating that the use of motixafortide, a CXCR4 inhibitor, both alone and in combination with natalizumab, a VLA-4 inhibitor, significantly improved the mobilization of hematopoietic stem cells (HSCs) for gene therapy in sickle cell disease (SCD), without increasing the risk of toxicities. This interview took place at the 67th ASH Annual Meeting and Exposition, held in Orlando, FL.
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Transcript
The study that we conducted in sickle cell patients to improve mobilization involved the use of motixafortide as a single agent alone, and then subsequently in the same patients, a second mobilization with motixifortide plus natalizumab, which is a VLA-4 inhibitor, looking to see if we could improve the ability to collect stem cells for sickle cell patients going on to gene therapy, which is a significant unmet need in the field right now – we have FDA-approved products, and a significant proportion of patients can’t collect stem cells to be able to receive those curative therapies...
The study that we conducted in sickle cell patients to improve mobilization involved the use of motixafortide as a single agent alone, and then subsequently in the same patients, a second mobilization with motixifortide plus natalizumab, which is a VLA-4 inhibitor, looking to see if we could improve the ability to collect stem cells for sickle cell patients going on to gene therapy, which is a significant unmet need in the field right now – we have FDA-approved products, and a significant proportion of patients can’t collect stem cells to be able to receive those curative therapies. And so what we observed in this study was that motixafortide alone and in combination with VLA-4 inhibitors improved significantly our ability to mobilize hematopoietic stem cells, and those levels that we were able to mobilize corresponded with a predicted ability to collect enough stem cells for a gene therapy within just one collection cycle, which is significantly better than what we would have predicted or what we would have observed in the pivotal trials using single-agent plerixafor, which is currently the standard of care. And importantly, that we were able to use those mobilizing regimens that improve mobilization without any significant increased risk of toxicities.
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