26 April 2023

A new medication appears to help multiple myeloma patients produce extra stem cells before autologous transplantation, researchers have reported.

The drug, motixafortide, is an inhibitor of the CXCR4 chemokine receptor. It was tested in GENESIS, a trial of 122 myeloma patients undergoing autologous haematopoietic stem cell transplantation.

Patients came from 18 sites in five countries. They were given the standard therapy for mobilising stem cells, G-CSF, and randomised to receive either motixafortide or a placebo.

The primary endpoint was to collect ≥6 × 106 CD34+ cells per kg within two apheresis procedures. Those given motixafortide were successful in producing the target number of stem cells in 92.5% of cases, compared with 26.2% of cases in the placebo group.

In addition, 88.8% of the motixafortide group were able to produce this goal in just one apheresis procedure, compared to 9.5% of cases in the placebo group.

In Nature Medicine on Monday, Dr John DiPersio of the Washington University School of Medicine, USA, and colleagues wrote that the drug “was safe and well tolerated, with the most common treatment-emergent adverse events observed being transient injection site reactions”.

They conclude: “Motixafortide plus granulocyte colony-stimulating factor mobilised significantly greater CD34+ hematopoietic stem and progenitor cell numbers, while preferentially mobilising increased numbers of immunophenotypically and transcriptionally primitive stem cells.”

Dr DiPersio commented: “This study suggests motixafortide works extremely well in combination with the standard drug, granulocyte colony stimulating factor (G-CSF), in mobilising stem cells in patients with multiple myeloma.

“We are hopeful that this investigational drug, if approved, will expand the number of patients who can receive an effective stem cell transplant for multiple myeloma.”

The drug is also being investigated by the same researchers as a stem cell mobiliser to support the genetic correction of inherited sickle cell anaemia.


Crees ZD, Rettig MP, Jayasinghe RG, Stockerl-Goldstein K, Larson SM, Arpad I, Milone GA, Martino M, Stiff P, Sborov D, Pereira D, Micallef I, Moreno-Jiménez G, Mikala G, Coronel MLP, Holtick U, Hiemenz J, Qazilbash MH, Hardy N, Latif T, García-Cadenas I, Vainstein-Haras A, Sorani E, Gliko-Kabir I, Goldstein I, Ickowicz D, Shemesh-Darvish L, Kadosh S, Gao F, Schroeder MA, Vij R, DiPersio JF. (2023) “Motixafortide and G-CSF to mobilize hematopoietic stem cells for autologous transplantation in multiple myeloma: a randomized phase 3 trial.” Nature Medicine, doi: 10.1038/s41591-023-02273-z

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

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