More stem cell firsts -- spinal cord injury and blindness

Tuesday was a very proud day here at CIRM headquarters. We were able to announce the enrollment of the first patient in a clinical trial funded, in part, by the agency. It was also a bit of a somber day as Paul Knoepfler pointed out in his blog yesterday. It meant the patient had recently undergone a life-changing tragedy that landed them in need of the hope the follow-on trials to this safety study might provide.

This morning's news feed brought a similar double-edged milestone. Stem Cells Inc, the Newark California-based biotech firm, announced that the day before their research team had injected adult neural stem cells into a spinal cord injury patient for the first time. The stem cells were administered at Balgrist University Hospital of the University of Zurich to a 23-year old German man who had suffered a spinal cord injury in an auto accident in April and was completely paralyzed below the waist.

A press release from the company posted on Yahoo has a quote from the patient that vividly points out the double-edged nature of this milestone to him:

"This terrible injury crossed out almost all my life plans, and has led me to an unexpected path. Participating in this clinical trial not only gives me a sense of hope, but it also helps move this important research forward."

There was another strong linkage between these two milestones. One of the lead surgeons at the Zurich hospital was Raphael Guzman who has a visiting appointment there and is on faculty at Stanford, where he is a colleague of Gary Steinberg who transplanted the cells in the CIRM-funded trial at Santa Clara Valley Medical Center.

Today's news feed brought a third milestone to our field. Advanced Cell Technology (ACT) announced that they had been given approval to begin the first clinical trial of cells derived from embryonic stem cells in Europe. They will be testing cells for a form blindness that strikes children called Stargardt's disease. Clearance to begin the trial came from the United Kingdom Medicines and Healthcare Products Regulatory Agency.

ACT launched a similar trial in the US in November 2010 and a second trial in January, this one for macular degeneration, the leading cause of blindness in the elderly. This summer the company announced the first patients were enrolled in each trial at UCLA in July.

Robert Lanza, chief scientific officer at ACT told a news organization in the UK in this morning's coverage:

"We're very pleased with the results so far. We're in the process of scheduling the next two patients for each of the two (US) trials."

We are keeping our fingers crossed that this early track record for safety holds.

DG