Preclinical Testing: Regenerative Medicine Stem Cell Therapy for Disabled Stroke Patients

Preclinical Testing: Regenerative Medicine Stem Cell Therapy for Disabled Stroke Patients

McGowan Institute for Regenerative Medicine affiliated faculty member Michel Modo, PhD, associate professor in the Department of Radiology at the University of Pittsburgh, while at the Institute of Psychiatry, King’s College London, conducted a pivotal pre-clinical study using ReNeuron’s lead CTX neural stem cell line in a stroke model. The first clinical application for the CTX cell line is the company’s ReN001 stem cell therapy for disabled stroke patients, currently in Phase I clinical development. Study results were recently published in Stem Cells.

The newly-published data show significantly improved outcomes in the key behavioral tests in the CTX cell-treated group in preclinical tests. A gradual improvement in function was observed between 4 and 12 weeks post-implantation of the CTX cells. Where stroke damage was confined to the striatal region of the brain, up to 83% recovery of sensorimotor function was recorded in the cell-treated group. Up to 20% of the CTX cells were seen to differentiate into astrocytes in the cell-treated group, a neuronal cell type associated with behavioral improvements in the chronic phase of stroke. Furthermore, implantation of the CTX cells restored Collagen IV expression to almost control levels, indicating restoration of damaged blood vessels and the formation of new blood vessels in the damaged striatal region of the brain, via the process of angiogenesis.

These results build on the findings of previous efficacy and mode-of-action studies with the CTX cells, confirming their efficacy in models of stable state stroke damage and providing further evidence of the ways in which the cells may promote recovery of function in these models. This study also demonstrates that the size and location of the stroke lesion is an important determinant of the degree of functional recovery seen, as is the site of implantation of the cells. Taken together, these findings therefore provide valuable information to assist in the clinical development of ReNeuron’s ReN001 stem cell therapy for disabled stroke patients, both in terms of the size and location of stroke lesion targeted and the optimum route of administration of the CTX cells.