Major Step Forward on hESC Stem Cell Treatment for Spinal Cord Injury

The “first-in-man” clinical trial of a human embryonic stem cell therapy has cleared its first safety hurdle, Asterias Biotherapeutics, Inc., reported yesterday.

The Menlo Park, Ca., firm said the spinal cord injury treatment caused “no serious adverse” events in its phase one safety trial. The therapy was originally developed by Geron Corp., which abandoned the trial in 2011 and sold its stem cell business to Asterias, which is a subsidiary of Biotime, Inc., of Alameda, Ca.

Asterias also said in a press release, 

“In four of the five subjects, serial MRI scans performed throughout the 2-3 year follow-up period indicate that reduced spinal cord cavitation may have occurred and that AST-OPC1 may have had some positive effects in reducing spinal cord tissue deterioration. This effect was seen in the animal model testing of AST-OPC1.”

Jane Lebkowski
Asterias photo

Jane Lebkowski, president of research and development at Asterias, said,

“The safety demonstrated in this trial positions Asterias to start a new phase 1/2a clinical trial in 2014, subject to clearance from the FDA .”

The California stem cell agency appears ready to award $14.3 million to Asterias to assist in that new clinical trial, as reported yesterday by the California Stem Cell Report. The agency also had loaned Geron $25 million for its clinical trial. The money has since been repaid.

(Asked to clarify the genealogy of the Asterias clinical trial, Katy Spink, vice president of Asterias, said in an email,
"AST-OPC1 is the former GRNOPC1.  The results presented by Jane Lebkowski at ASGCT and referred to in (the) press release are from the clinical trial that was started by Geron in 2010.  Slides from that presentation are on our website.  We are unfortunately not at liberty to comment on the question of potential CIRM funding, or on details of our future development plans beyond what is described on our website and in our SEC filings.")

Stephen McKenna, director of the Rehabilitation Trauma Center at the Santa Clara Valley Medical Center, said,

“Spinal cord injury represents a tremendous unmet medical need that not only results in severe disability, but can also significantly shorten the projected lifespan of affected individuals. There are no approved therapies that can repair spinal cord injuries.”

Asterias said 12,000 persons suffer a spinal cord injury each year in the United States and about 1.3 million Americans are estimated to be living with a spinal cord injury.

(An earlier version of this item incorrectly said that Asterias announced the results in a press release today. The press release was dated yesterday. An earlier version also carried an incorrect first name for Jane Lebkowski. The material about the genealogy of the treatment was added shortly after the original version of this item was posted.)