The latest news and information about what's going on with SCI science and research.
Neuralstem announced today
that it has been approved by the FDA to begin a Phase I safety trial of its neural stem cell line NSI-566 in patients with chronic spinal cord injury. The trial is open-label (no placebos or blinding of treatment) and will enroll up to eight ASIA A patients (complete -- no motor or sensory function, T2-T12), between one and two years after injury.
NSI-566 is derived from stem cells native to the brain and spinal cord. The company has found a way to produce them in large quantity for direct injection to the spinal cord. Previously, Neuralstem enrolled 18 people in a trial of this cell line targeting ALS. The company says a Phase II trial is coming, paid for in large part by the National Institutes of Health.
The SCI trial has been pending since August 2010 but no doubt benefited from research from Paul Lu and Mark Tuszynski at the University of California, San Diego. They published results
in September indicating that the NSI-566 line supported functional recovery in an animal model: “…early-stage neurons can overcome the inhibitory milieu of the injured adult spinal cord to mount remarkable axonal growth, resulting in formation of new relay circuits that significantly improve function. These therapeutic properties extend across stem cell sources and species."
Here’s more on that paper, reported in this blog
and in the media
, and this from Neuralstem:
In a September study published in the journal CELL
paralyzed rats transplanted with NSI-566 stem cells recovered significant locomotor function, regaining movement in all lower extremity joints. Additionally, the transplanted neural stem cells turned into neurons which grew multiple axons. These axons extended over 17 spinal segments above and below the point of severance, where made reciprocal synaptic connectivity with the host spinal cord neurons, improving electrophysiological and functional outcome.
No details yet on exactly when or where the SCI trial sites will take place. Concurrently, Neuralstem is also working with partners in the Czech Republic and South Korea to set up SCI trials for the 566 line.
The ALS trial was done at Emory University in Atlanta. The company reports that trial has gone very well: no adverse effects, some early indication of disease reduction. In December, Neuralstem announced that researchers showed the long-term survival of transplanted 566 cells in six patients who died (five from ALS progression) through a technology called DNA fingerprinting. The company has also presented data to suggest a trial for stroke may be forthcoming.