Durable response of glioblastoma to adjuvant therapy consisting of temozolomide and a weekly dose of AMD3100 (plerixafor), a CXCR4 inhibitor, together with lapatinib, metformin and niacinamide

Adan Rios,1 Sigmund H. Hsu,2 Angel Blanco,3 Jamie Buryanek, 4 Arhur L. Day, 2 Mary F. McGuire, 5 Robert E. Brown4

1 Division of Oncology at UTHealth McGovern Medical School, Houston, TX, USA

2 Department of Neurosurgery at UTHealth McGovern Medical School, Houston, TX, USA

3 Memorial Hermann Hospital, Texas Medical Center, Houston, TX, USA

4 Department of Pathology and Laboratory Medicine at UTHealth McGovern Medical School, Houston, TX, USA

5 Adjunct Faculty, Mathematics & Computer Science at University of St. Thomas-Houston, Houston, TX, USA


Adan Rios, email:

Keywords: glioblastoma, morphoproteomics, biomedical analytics, plerixafor, preventative and targeted therapy

Received: April 23, 2016 Accepted: June 03, 2016 Published: June 11, 2016


Glioblastoma multiforme (GBM) is a CNS (central nervous system) malignancy with a low cure rate. Median time to progression after standard treatment is 7 months and median overall survival is 15 months [1]. Post-treatment vasculogenesis promoted by recruitment of bone marrow derived cells (BMDCs, CD11b+ myelomonocytes) is one of main mechanisms of GBM resistance to initial chemoradiotherapy treatment [2]. Local secretion of SDF-1, cognate ligand of BMDCs CXCR4 receptors attracts BMDCs to the post-radiation tumor site [3]. This SDF-1 hypoxia-dependent effect can be blocked by AMD3100 (plerixafor) [4]. We report a GBM case treated after chemoradiotherapy with plerixafor and a combination of an mTOR, a Sirt1 and an EGFRvIII inhibitor. After one year temozolomide and the EGFRvIII inhibitor were stopped. Plerixafor, and the MTOR and Sirt-1 inhibitors were continued. He is in clinical and radiologic remission 30 months from the initiation of his adjuvant treatment. To our knowledge, this is the first report of a patient treated for over two years with a CXCR4 inhibitor (plerixafor), as part of his adjuvant treatment. We believe there is sufficient experimental evidence to consider AMD3100 (plerixafor) part of the adjuvant treatment of GBM. Significance: The adjuvant inhibition of GBM vasculogenesis(a process different from local angiogenesis) by specifically blocking the migration of BMDCs to the primary tumor site with inhibitors of the CXCR4/SDF-1 axis represents a potential novel therapeutic approach to GBM. There is significant pre-clinical evidence and validation for its use as demonstrated in a patient derived tumor xenograft model of GBM. Together with other specific anti-tumoral therapies, the active inhibition of vasculogenesis in the adjuvant treatment of GBM is deserving of further exploration.

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