For Alzheimer’s Disease Patients, Company’s Cell Line Shows Treatment Promise

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Alzheimer’s disease mouse study

Neuralstem Inc. researchers have released the preclinical results of a human stem cell line with the potential to treat Alzheimer’s disease patients. The research paper, “Human Cortical Neural Stem Cells Expressing Insulin-Like Growth Factor-I: A Novel Cellular Therapy for Alzheimer’s Disease,” was published in the journal Stem Cells Translational Medicine.

Neuralstem’s patented technology consists of the generation of neural stem cells from the human hippocampus, which led to the discovery of molecules that the company believes may be able to stimulate the brain into forming new neurons, potentially reversing many neurodegenerative diseases resulting from the central nervous system’s (CNS) loss of nerve tissue and neurons.

The potential of this cellular therapy has been shown in amyotrophic lateral sclerosis (ALS), where scientists demonstrated that transplanted spinal neural stem cells (NSCs) were able to integrate in the spinal cord and form synapses, reducing the pathology and inflammation associated with the disease.

The company’s stem cell product candidate (NSI-566) is the first from Neuralstem, and is currently under development, with two completed clinical studies.  The company is also developing products to treat chronic spinal cord injury, paralysis from ischemic stroke, and major depressive disorder (MDD).

Researchers characterized the cell line HK532-IGF-I, a novel human cortex-derived NSC line modified to express insulin-like growth factor-I (IGF-I), a known promoter of neuron formation (neurogenesis) and synapse formation (synaptogenesis) in living organisms.

The team reports these cells differentiated into GABA-ergic neurons, a subtype of neurons that are dysregulated in Alzheimer’s disease, increased vascular endothelial growth factors (VEGF) in vitro and increased neuroprotection without compromising normal cellular function. The cells survived transplantation in a mouse model of AD and showed long-term survival in targeted brain areas. Moreover, the cells improved cognition and reduced amyloid beta aggregation in AD mice, according to an animal study presented at the 2015 annual meeting of the American Neurological Association.

Study author Karl Johe, Ph.D., chief scientific officer of Neuralstem, said in a press release, “IGF-1 is a potent neuroprotective molecule that is naturally produced in our brain and body. In aging or lesioned brains, extra addition of IGF-1 may be therapeutic.  Combining neural stem cell therapy with a protein therapy, in a single and permanent delivery, is a powerful new therapeutic modality.  HK532-IGF-1 is the first of such second-generation stem cell therapies we are developing in which the stem cells are used as stable and safe delivery vehicle of therapeutic proteins.”