Neuralstem Cell Therapy:
Repairing and Replacing Damaged Cells
Different regions of the brain and spinal cord house different, specialized cells. Neuralstem's technology enables the isolation and expansion of human neural stem cells from each of these regions of the developing central nervous system (CNS) in virtually unlimited numbers from a single donated tissue.
The goal of cell therapy is to replace and/or repair dead or diseased cells. Unlike other stem cell technologies, Neuralstem is growing regionally specific cells that are already suited to the task prescribed to them once transplanted into the CNS. In spinal cord indications, for instance, the company will be using human NSI-566 spinal cord stem cells only. Additionally, once inside the body, Neuralstem cells also do not become any cell other than that to which they are fated.
There are two primary ways that these cells can provide therapeutic effects.
Create: The transplanted cells may help create new circuitry
Express: The transplanted cells may express factors that protect existing cells
We believe that Neuralstem's cells do both.
In preclinical work conducted at major research centers across the U.S., Neuralstem cells integrated and made synaptic contact with the host. The cells also expressed one or more growth factors. These are special chemicals that the CNS uses to operate and thrive. Many of these growth factors are protective of cells. View published papers here: 1, 2, 3.
Neuralstem’s transplanted cells survive in patients and integrate into the host tissue, creating new circuitry and expressing growth factors. This dual function is important. In spinal cord injury, for instance, the company hopes to create circuitry that will help signals from the brain get to where they need to go. In many indications, the goal is to slow down or halt the degeneration of cells caused by disease, or by injury, by expressing neuroprotective growth factors into the system.
Delivering the Cells into the Spinal Cord and Brain Safely and Effectively
A vital component to the Neuralstem cell therapy platform is the delivery of the cells directly into the gray matter of the spinal cord, where they can protect and integrate with the patient's spinal cord neurons.
Neuralstem's proprietary Spinal Cord Delivery Platform and Floating Cannula were designed specifically by Neuralstem's ALS trial neurosurgeon, Nicholas M. Boulis, MD, for the world's first intraspinal delivery of stem cells. The safety of the device was first reported in data presented at the American Association of Neurologists' 2011 Annual Meeting, and its safety has since been repeatedly validated in the company’s completed two ALS clinical studies, in a total of thirty patients, which met primary safety endpoints. In addition to ALS, NSI-566 is also in a Phase I trial in chronic spinal cord injury at UC San Diego School of Medicine. You can view this breakthrough medical device in surgery here.
The Spinal Cord Delivery Platform and Floating Cannula will be utilized to deliver Neuralstem cells in the spinal cord safely and effectively for myriad diseases and injuries. Expected to be the standard in the industry and research community for intraspinal procedures, Neuralstem is licensing the breakthrough cell therapy device to industry and academia.
Delivery of neural stem cells into the brain will be accomplished using well-established stereotactic injection procedures. NSI-566 is in clinical development to treat ischemic stroke utilizing one-time treatments of these intracerebral injections to safely transplant cells near the stroke lesions of ischemic stroke patients.