California Biomedical Industry - California Healthcare Institute

Researcher profileHans Keirstead, Ph.D.:Leading the charge into thefuture of stem cellsHans S. Keirstead, Ph.D., co-directorof the Sue and Bill Gross Stem CellResearch Center, is an assistantprofessor of the Reeve-IrvineResearch Center of the University ofCalifornia, Irvine.Whereas other thought leaders in thestem cell research arena cite businessmodels and financing and regulatorypathways as potential challenges along thepath to commercializing stem-cell basedtherapeutics, Hans Keirstead, Ph.D., hasalready cleared those potential hurdles.Twice. And he has opened the doors if notpaved the path for those to follow.Keirstead is co-director of the Sue andBill Gross Stem Cell Research Centerand an assistant professor of the Reeve-Irvine Research Center, both at UCIrvine. There he directs a 20-personresearch team investigating the cellularbiology and treatment of spinal cordtrauma. He and his team developed theworld’s first embryonic stem-cell-basedtreatment, which proved be effective inmaking paralyzed rats walk again.To envision Keirstead’s approach, think ofthe spinal cord as a strand of electricalwire. The nerves are the wire inside thecord and they transmit signals betweenthe brain and all the other points ofthe body. Like an electrical cord, thespinal cord is insulated — the humantissue is made up of glial cells. When thespinal cord is damaged, the glial cells nolonger protect the signaling nerves. Thetrauma results in loss of such functionsas locomotion, sensation or bowel/bladder control.Keirstead and his team developed theworld’s first method of using embryonicstem cells to produce large quantities ofhigh-purity tissue to repair the nerves andreplace the glial cells. With new tissue, theconnection can heal, enabling signals topass between the brain and body again.Geron Corp. of Menlo Park licensedthe technology and, with Keirstead’sparticipation, “ran the long, expensivepath” to begin the world’s first clinicaltrial of an embryonic stem cell-basedtherapeutic. “It took $45 million andnine years,” Keirstead said. He addedthat because the field is brand new,so are the regulatory procedures.“Sometimes the FDA changed theirmind, sometimes we changed ourminds. It is an iterative process and ittook us awhile to figure it out.”On October 11, 2010, Geron’s first spinalcord injury patient was administeredGRNOPC.“The significance of the Geron trial ishuge,” Keirstead said. “The gates arenow open.”Indeed, in December 2010, CaliforniaStem Cell, Inc. filed its IND for asecond of Keirstead’s stem-cell basedspinal therapeutics — this one for thetreatment of spinal muscular atrophy.The development path to CaliforniaStem Cell’s IND, Keirstead said, took $3million over three years, and the FDAreview was three months, as is standardfor conventional drug developers’ INDsubmittals.“Every subsequent story owes a debtof gratitude” to Geron, Keirstead said.Then he added, “We are going to startseeing a lot of stem cell INDs. We are at areal turning point.”While agreeing that the biomedical worldwill be watching the Geron trial closely,Keirstead said that the idea that stemcell research would be set back if thetreatment does not work is dangerous.Geron’s product is a stem-cell derivative.Keirstead said that if the product doesnot work, it would likely be the case thatthe material is not the correct substanceand/or is not able to heal the damage— not that the technology is harmful. Incontrast to gene therapy, which sufferedsome failures a decade ago, a stem celltherapy failure in one condition or tissuewould not foreshadow performance oneway or the other in different applications.However, he added, “The question pointsto the dangers of public perception.”As a high-profile stem cell researcher,Keirstead has firsthand knowledge ofthe weight that public opinion carries.He said that one day he showed up at hislab and his researchers were standingoutside, ashen faced. They directed him tothe UCI Student Center, where picketershad hung a banner. On it was a five-foothighphoto of Keirstead and 12 smallerpictures of aborted late-term fetuses. Hetook the time to talk with the protesters,most of whom were relieved to learn howembryonic stem cells are obtained.“There’s so much misinformation outthere,” Keirstead said. But informationdoes not have to be true to influencepolicy making or political decisions. “Itbehooves us all to educate the public,”Keirstead said.California Biomedical Industry 2011 Report | 97