This investigational drug enhances stem cell mobilization in multiple myeloma transplants

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This investigational drug enhances stem cell mobilization in multiple myeloma transplants

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Nature Medicine: This investigational drug enhances stem cell mobilization in multiple myeloma transplants

Harvesting a multiple myeloma patient’s own healthy stem cells, storing them and reinfusing them after high-dose chemotherapy improves overall survival compared with traditional chemotherapy-only approaches.

This approach, called autologous stem cell transplantation (ASCT) , depends on the ability to collect enough hematopoietic stem/progenitor cells (HSPCs) , usually derived from blood.

G-CSF (a protein that aids in the production of white blood cells and stem cells in the bone marrow) is the standard agent for mobilizing hematopoietic stem/progenitor cells (HSPCs) into the blood for collection and storage, but despite multiple days of treatment, 40–50 % of multiple myeloma patients still cannot generate optimal numbers of stem cells for autologous transplantation.

Motixafortide , a selective inhibitor of the chemokine receptor CXCR4, has been shown in phase 1 clinical trials in the past to increase the abundance of HSPCs in the circulating blood of healthy individuals.

On April 17, 2023, researchers from Washington University School of Medicine in St. Louis published a research paper entitled: Motixafortide and G-CSF to mobilize hematopoietic stem cells for autologous transplantation in multiple myeloma: a randomized phase 3 trial in Nature Medicine .

The Phase 3 clinical trial reported that in 90% of members of 80 multiple myeloma patients, a single injection of Motixafortide safely mobilized optimal numbers of stem cells for collection and transplantation. This approach may improve outcomes for patients with multiple myeloma.

As part of a multicenter phase 3 study, the research team compared the safety of the following in 122 patients with multiple myeloma undergoing hematopoietic stem/progenitor cell (HSPC) mobilization prior to autologous stem cell transplantation And Effect: Combining Motixafortide and G-CSF, vs Combining Placebo and G-CSF.

The results showed that Motixafortide + G-CSF was safe and well tolerated, with most treatment-emergent adverse events lasting only a short time.

A single Motixafortide + G-CSF treatment mobilized an optimal number of HSPCs for autologous stem cell transplantation in 93% of multiple myeloma patients (80 total) compared to placebo + G-CSF 26% of patients (a total of 42 people) .

In addition, the authors of the paper believe that Motixafortide+G-CSF well mobilizes more primary HSPCs, which are associated with enhanced self-renewal and regeneration.

The authors conclude that these findings suggest a new protocol for HSPC mobilization that is rapid, safe, and well tolerated, and has the potential to greatly improve the ability to harvest HSPCs for stem cell transplantation, as well as other HSPC-based gene therapies.

Paper link :
https://www.nature.com/articles/s41591-023-02273-z

This investigational drug enhances stem cell mobilization in multiple myeloma transplants

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