Cytomedix, Inc., a regenerative therapies company, has initiated phase I clinical study with ALD-451 in brain cancer patients. This study is started in collaboration with Duke University Medical Centre.

The open-label study will enroll up to 12 patients and is intended to demonstrate the feasibility and safety of ALD-451 when administered intravenously in World Health Organization (WHO) grade IV malignant glioma patients following surgery, radiation therapy and treatment with temozolomide.  The trial also will obtain an initial description of the effects of ALD-451 on neurocognition. The clinical study is open for enrollment having received Investigational New Drug clearance from the US Food and Drug Administration and Investigational Review Board clearance from Duke University Medical Centre (ClinicalTrials.gov Identifier: NCT01639612).

Dr Annick Desjardins, Assistant Professor of Medicine at The Preston Robert Tisch Brain Tumour Centre at Duke University Medical Centre and the study's principle investigator. Co-investigators are Dr Henry S Friedman, Deputy Director, The Preston Robert Tisch Brain Tumour Centre and Dr Joanne Kurtzberg, chief scientific officer and medical director, Robertson Clinical & Translational Cell Therapy Programme. Cytomedix will be responsible for manufacturing ALD-451 for the clinical trial. Duke University Medical Centre, through the Robertson Clinical & Translational Cell Therapy Programme, will fund the trial and be responsible for all other aspects of the study.

"We are excited to initiate patient recruitment in this study and to explore the use of this cellular therapy to treat the neuro-cognitive side effects of treating these devastating cancers,” said Dr Desjardins.

Martin P Rosendale, chief executive officer of Cytomedix, stated, “We are  delighted to be working on this important trial with leading clinicians at Duke University Medical Centre, one of the world's leading brain cancer centres for both treatment and research. Malignant glioma patients who undergo surgery, radiation therapy and temozolomide treatment oftentimes experience deterioration of neuro-cognition and have poor patient-reported outcomes.   Earlier studies suggest that ALDH bright cells may repair neural brain damage.  We expect this study to corroborate those results and look forward to advancing the development of this very promising product candidate.”

Primary central nervous system (CNS) tumours represent about 1.35 per cent of all cancers and 2.2 per cent of all cancer-related deaths. Glial neoplasms represent about 40 per cent of all primary CNS tumours and about 75 per cent are malignant. Malignant gliomas include WHO grade III: anaplastic astrocytoma, anaplastic oligodendroglioma and anaplastic oligoastrocytoma, and WHO grade IV: glioblastoma and gliosarcoma. Because of their extensive infiltrative and invasive nature, malignant gliomas present unique challenges.   This  infiltrative nature, combined with their proximity to critical intracranial structures as well as operative difficulty distinguishing between normal and neoplastic cells, significantly reduces the efficacy of surgical resection.  Radiation therapy and systemic chemotherapy are necessary adjuncts to treatment.  Children and adults who receive radiation therapy involving the brain frequently experience a progressive cognitive decline, significantly affecting their quality of life.

ALD-451 is the population of autologous pluri-potent ALDH br stem cells isolated from the patients bone marrow using Cytomedix proprietary technology. These adult stem cells express high levels of the enzyme ALDH, an indicator of biological activity in heterogenous early stage stem cells. Preclinical research with ALD-451 bright cells suggests that they may promote the repair of tissue damage. Recently, preliminary data presented at the 2012 International Society of Cell Therapy showed that ALDH br bright cells reduced severity of intracranial inflammation after brain irradiation in an animal model. Investigators have also completed preclinical research that showed improvements in motor function, improvements in the slowing of decrease in brain volume, the reversal of decline in stroke-induced cell viability and improved blood flow, or perfusion, in the brain.

Cytomedix, Inc. is an autologous regenerative therapies company commercializing innovative platelet technologies for orthopedics and wound care with a pipeline of adult stem cell therapies for tissue repair.