Neuralstem announced that it had received approval from the FDA to begin Phase I safety trials of NSI-566. NSI-566 is a cell therapy candidate; the main goal of the study is to determine the safety and toxicity of human spinal stem cell transplantation for the treatment of paralysis and related symptoms due to chronic spinal cord injury.
Neuralstem is focused on producing technology that allows for the production of neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells constitutively into mature, physiologically relevant human neurons and glia. In August of 2012, Neuralstem completed dosing of the last patient in an FDA-approved Phase I safety clinical trial for amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease.
The secondary objectives of the Phase I testing are to determine graft survival in the transplant site by MRI, as well as the effectiveness of transient immunosuppression. Each patient in the study will receive six injections: the first four patients will receive 100,000 cells per injection, the second four patients will receive 200,000 cells per injection. Each patient will receive physical therapy post-surgery, as well as immunosuppressive therapy. The trial will end six months post-surgery for each patient.
Karl Johe, Ph.D., Neuralstem chairman of the board and chief scientific officer, said, “The company is both pleased and excited to begin this long-awaited trial to treat spinal cord injury patients with our spinal cord cells, NSI-566. In August, in the peer-reviewed journal ‘Cell,’ compelling evidence was presented that the cells can ‘bridge the gap’ in a severed spinal cord model and return functionality.
This will be the first stem cell trial to attack the problem that way, as well as the first to treat chronic spinal cord injury patients. In addition to the pre-clinical animal data, we have conducted 18 successful surgeries using the same cells and surgical device in our ALS trial. That trial has demonstrated that the surgical route of administration and the cells are safe and, well-tolerated and that the cells survive long-term in the patients. The successes of our human clinical experience, combined with the compelling data from the preclinical spinal cord injury animal studies gives us confidence that we are prepared to move into this additional indication for NSI-566.”
Richard Garr, Neuralstem President and CEO, added, “This is another critical milestone for the company. We believe the FDA approval of a trial for a second indication for our NSI-566 cells demonstrates an increasing level of comfort at the FDA with our technology and acknowledges the positive strides being made in the ALS trial. Our goal for this SCI trial is to transplant and collect the 6 month data for all patients within a one-year timeframe. The approval of this trial is also a demonstration of our product development strategy. While we can create cell products from various regions of the CNS that might be applicable to all manner of indications, we have chosen to address multiple indications where the same cell product is applicable. This allows us to leverage the product manufacturing and safety data created in each trial with the next. Along those lines, we will begin dosing patients with NSI-566 to treat paralysis from stroke in the next month, in China, and we are preparing for a trial with NSI-566 to treat acute spinal cord injury patients in Korea this summer. We are also currently in discussions with the FDA over the design and scope of our Phase II trial for ALS with NSI-566. As we begin to create proof-of-principle data in multiple indications, we are also creating additional NSI-556 safety data across indications and borders.”
For more information, visit www.neuralstem.com
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