Research - Seneca NSI-566
NSI-566 is an investigational neural stem cell therapy that uses human spinal cord stem cells (HSSCs). Stem cells are unique in that they have the capacity to differentiate into many types of specialized cells during development.
When injected into the spinal cord, NSI-566 differentiates into types of neurons found in adults. They surround and support motor neurons damaged by ALS, by integrating into the neural network and forming connections (synapses) with the patient’s own neurons. They also secrete certain molecules that promote motor neuron growth and survival.
Seneca Bio received orphan designation by the FDA for NSI-566 in ALS. NSI-566 was evaluated in Phase 1 and Phase 2 safety studies in 30 patients. The data showed that the intraspinal transplantation of the cells was safe and well tolerated. Subjects from both the Phase 1 and Phase 2 studies continue to be monitored for long-term follow-up evaluations.
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When injected into the spinal cord, these fetal stem cells differentiate into the kind of neurons that exist in the adult body. They surround and support the impaired motor neurons in ALS by integrating into the neural network and forming connections (synapses) with the patient’s own neurons
Seneca Biopharma announced that work is underway to advance NSI-566, its leading stem cell treatment candidate, into a Phase 3 study in people with amyotrophic lateral sclerosis (ALS).
The decision followed a meeting with the US Food and Drug Administration (FDA), and is supported by data collected from a Phase 1 (NCT01348451) and Phase 2 (NCT01730716) clinical trial.
Post-hoc comparison of combined cohorts from Phase 1 and 2 trials vs. historical controls revealed statistically significant difference in scores in functional status and in a composite statistic combining survival and functional status in subjects receiving human spinal cord-derived neural stem cells.
Study provides validation for additional trials to evaluate the benefit of neural stem cells in ALS patients