October 5 is Stem Cell Awareness Day. Seems like a good time to update recent stem cell news, including new clinical trials.
. This is the much-watched first embryonic stem cell trial
approved by the FDA, based on preclinical data from Hans Keirstead at the University of California, Irvine. This is looking first at safety. The target is acute spinal cord injury (within a week or so of injury); the first patient was dosed about a year ago. They just enrolled patient #4, this one in California, where the company is based; the latest procedure, by a Stanford surgeon at Santa Clara Valley Medical Center, was funded, in part, with public money from the California Institute for Regenerative Medicine (CIRM, the folks running the state’s $3 billion stem cell bill). No adverse effects have been reported for any of the earlier patients.
Stem Cells, Inc.
, another California biotech, finally enrolled its first patient (the trial
was approved last December), using adult stem cells (derived from aborted fetuses) in what they call early chronic spinal cord injury (three to 12 months post injury). This is also a safety trial. The preclinical work was also done at UC Irvine (in the labs of Aileen Anderson, who runs a core lab
there for the Reeve Foundation, and Brian Cummings).
From a company press release:
Most recently, StemCells scientists and UCI researchers achieved a significant breakthrough by demonstrating for the first time that human neural stem cell transplantation can restore lost motor function even when administered at time points beyond the acute phase of trauma. When transplanted into mice with chronic spinal cord injury, our human neural stem cells were shown to promote a significant and persistent recovery in walking ability in two separate tests of motor function when compared to control groups
The StemCells clinical trial is taking place in Switzerland, where Swissmedic, the equivalent of the FDA there, will oversee its safety. The stem cells were transplanted at Balgrist University Hospital of the University of Zurich to a 23-year old German who was spinal cord injured in an auto accident in April.
One of the lead surgeons at the Zurich hospital was Raphael Guzman, a Stanford colleague of Gary Steinberg, who transplanted the cells in Santa Clara Geron trial.
Advanced Cell Technology
(ACT), based in Santa Monica, CA announced last week that it had been given approval to begin the first embryonic stem cell trial in Europe. The trial
, featuring retinal pigment epithelial (RPE) cells derived from human embryonic stem cells, will take place at Moorfields Eye Hospital in London.
The company will test the cells for safety in people with Stargardt's disease, which causes pediatric blindness, and for macular degeneration, the leading cause of blindness in adults. ACT began its trials last July in the U.S., at the Jules Stein Eye Institute, UCLA School of Medicine and at the Oregon Health and Sciences University in Portland.
ACT sees a big market. From a company press release:
Degenerative diseases of the retina are among the most common causes of untreatable blindness in the world. As many as 30 million people in the United States and Europe suffer from macular degeneration, which represents a $25-30 billion worldwide market that has yet to be effectively addressed. Approximately 10% of people ages 66 to 74 will have symptoms of macular degeneration, the vast majority suffering from the "dry" form of AMD - which is currently untreatable. The prevalence increases to 30% in patients 75 to 85 years of age.
, a British biotech, has begun a clinical trial
to inject adult neural stem cells (from fetuses) into the brains of people disabled by stroke. The independent Data Safety Monitoring Board there has reviewed safety data so far (four patients) and has recommended the trial advance to a higher dose. Says the company: "Data from the laboratory safety tests, neurological examinations and neurofunctional tests conducted thus far indicate that the [ReN001] treatment is safe and well-tolerated at the initial dose."
, a Rockville, MD biotech, has already dosed 12 people with ALS using adult spinal cord stem cells. The procedures all took place in Atlanta at Emory University. While the trial
is looking at safety, there have been anecdotal reports of recovery.
In ALS, motor neurons die, leading to paralysis. In preclinical animal work, Neuralstem says its cells “made synaptic contact with the host motor neurons and expressed neurotrophic growth factors, which are protective of cells.”
Neuralstem has other cell therapy indications in mind, including brain cancer, stroke and spinal cord injury. In August 2010 the company filed an Investigational New Drug (IND) application to the FDA to begin a safety trial of its human spinal cord stem cells for chronic SCI – one to two years post. Trial sites include Miami, Philadelphia, Milwaukee and Atlanta.
China SCI Network
. Rutgers scientist Wise Young reports that several patients have been enrolled in an acute spinal cord injury trial
in China to test the safety of cells derived from umbilical cord blood. Dr. Young hopes to see additional patients enrolled in Austin, TX as soon as regulatory agencies are all aboard. Get the latest here
Read more about Stem Cell Awareness Day.