University of California approves stem cell trial for spinal cord injury

April 16, 2014 — Neuralstem, Inc. announced that the Institutional Review Board of the University of California, San Diego, School of Medicine has approved the Phase I safety trial to treat chronic spinal cord injury (cSCI) with its NSI-566 stem cells.

The NSI-566/cSCI Phase I trial will enroll patients with thoracic spinal
cord injuries (T2-T12) who have an American Spinal Injury Association
(AIS) A level of impairment, between one and two years after injury. AIS
A impairment, which is complete paralysis, refers to a patient with no
motor or sensory function in the relevant segments at and below the
injury.

The trial, which already has FDA approval, and has a
one-year completion goal, will be under the direction of principal
investigator (PI) Joseph Ciacci, MD, UC San Diego School of Medicine and
neurosurgeon at UC San Diego Health System. Much of the pre-clinical
work with the NSI-566 cells in spinal cord injury was conducted at UC
San Diego School of Medicine by Martin Marsala, MD, professor in the
Department of Anesthesiology, who will be another study investigator.

"Moving
our spinal cord cells into human trials for a second indication in the
U.S. is a major step forward for the company," said Karl Johe PhD,
Neuralstem's chairman of the board and chief scientific officer. "We
have demonstrated that the surgical route of administration is safe and
robust; that the cells survive transplantation and are biologically
active in the patients, and that both the cells and the surgery are
well-tolerated and safe. In animals, we have shown compelling
proof-of-principle of return of significant function. With 30 successful
spinal surgeries completed in our ALS trials, we feel we are ready to
tackle spinal cord injury and are excited to begin this ground-breaking
study. We wish to thank and acknowledge the support of our collaborators
at UCSD, without whom this trial would not be possible."

In a
peer-reviewed study, published in the journal, Stem Cell Research and
Therapy, in May 2013, rats transplanted with NSI-566 stem cells three
days after a spinal cord injury at L3 (lumbar 3), showed improvement
along several measures of motor function and a reduction of spasticity.
The study demonstrated that intraspinal grafting of NSI-566 cells during
the acute phase of a spinal cord injury could represent a safe and
effective treatment that ameliorates post-injury motor and sensory
deficits.

In a separate peer-reviewed study, published in the
journal, Cell, in August 2012, rats with surgically transected spinal
cords transplanted with Neuralstem's NSI-566 stem cells recovered
significant locomotor function, regaining movement in all lower
extremity joints. Furthermore, the cells turned into neurons which grew a
"remarkable" number of axons that extended for "very long distances,"
bridging above and below the point of severance. These neurons also
appeared to make reciprocal synaptic connectivity with the host rat
spinal cord neurons in the gray matter for several segments below the
injury.

There are more than 10,000 new spinal cord injuries (SCI)
in the U.S. each year. At the time of a recent survey done by the
Christopher & Dana Reeve Foundation, there were approximately
840,000 people living with chronic SCI, which refers to patients in whom
paralysis persists and becomes permanent. According to the National
Spinal Cord Injury Association, 85 per cent of SCI patients who survive
the first 24 hours are still alive 10 years later.

Neuralstem's
patented technology enables the production of neural stem cells of the
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 glial cells. Neuralstem's
NSI-566 spinal cord-derived stem cell therapy is in Phase II clinical
trials for amyotrophic lateral sclerosis (ALS), often referred to as Lou
Gehrig's disease. Neuralstem has been awarded orphan status designation
by the U.S. FDA for its ALS cell therapy.

In addition to ALS,
the company is also targeting major central nervous system conditions
with its NSI-566 cell therapy platform, including spinal cord injury and
ischemic stroke. The company has received FDA approval to commence a
Phase I safety trial in chronic spinal cord injury.