Ga. in stem cell focus

But the choice of an Atlanta institution to play a part in the landmark study was no accident. A confluence of factors has turned Georgia into a top-tier state for biotech research and development. There are learning centers such as Georgia Tech, the University of Georgia and Emory University, a pro-business climate that includes top-notch hospitals and the utility of Hartsfield-Jackson International Airport, among others.

The state’s growing stature in the field is further evidenced by another historic first set to occur at Emory University this week — the injection of fetal stem cells into the spine of an ambulatory ALS patient.

These trials, and the medical leaps they could produce, however, take place against a backdrop of divided public opinion on such research. Voices opposing the use of embryonic stem cells on ethical or moral grounds are strong in Georgia, and promise a renewed fight to legally restrict the practice. Proponents worry that even the threat of legislative limits might scuttle the industry here. The top two candidates to be Georgia’s new governor appear divided on the issue, too.

A historic procedure

The experimental treatment being carried out at the Shepherd Center and run and funded by Geron Corp., a California company, verges on science fiction. Geron calls embryonic stem cells “immortal,” able to “divide endlessly in tissue culture.” Such cells are a blank slate, with the potential to turn into cells that build parts of the body. In this study, researchers hope the oligodendrocyte progenitor cells, which produce a substance called myelin, will recoat spinal nerve cells and transmit impulses.

“It’s historic,” said Hans S. Keirstead, a University of California Irvine neurobiologist, who developed a therapy that made paralyzed rats walk again and licensed it to Geron. This trial “symbolizes the transition from discussion and thought in the ivory towers to real action in the clinic.”

The Atlanta patient is the first of 10 paralysis victims Geron plans to study nationwide. Its aim is not to get patients walking; it’s simply to see if the cells can be safely injected into the spine and then to determine if those cells grow normally in the body.

“We have done everything that we can or the community can think of to assure the safety of this treatment,” Keirstead said in an interview. “But a rat is not a human, and this is an experiment, and we have got to wait and see.”

Geron selected the Shepherd Center, Keirstead said, because it is “perhaps the most outstanding spinal cord rehabilitation center in North America.”

A single dose of 2 million stem cells was administered to the unnamed patient, who was injured in the mid-spine and paralyzed from the waist down.

The procedure had to take place within two weeks of the injury, before scar tissue formed on the spinal cord. The injection was performed using a sophisticated syringe-positioning device developed by Geron that attaches to the frame of the operating table.

A Geron spokeswoman said the Oct. 8 surgery went well but offered no update on the patient’s condition. She said Phase 1 of the trial could expand to as many as seven sites nationwide.

Keirstead described himself late last week as both “excited and worried.” A breakthrough would mean a step toward the holy grail of regenerating lost movement in the disabled. But tumors or other problems could set back such treatments for years.

Foes on alert

The tests, which have no government funding and were approved by the federal Food and Drug Administration, are being viewed with great interest by patients, researchers and those in the medical-business sector.

Members of Georgia Right to Life also will be watching. Dan Becker, the group’s president, said the landmark medical procedure will galvanize the state’s anti-abortion community. He promised to push for legislation banning embryonic stem cell research when the General Assembly reconvenes in January.

“For us the battle is not over. It’s not done in Georgia,” said Becker. “I think most Americans would disagree with the basic premise of embryonic stem cell research. With embryonic stem cell treatment, you are always taking a life.”

While many scientists dispute that claim, a 2006 survey showed Georgians are divided on the topic of embryonic research. While 63 percent of those polled supported research on stem cells using donated embryos from fertility clinics that would otherwise be discarded, those identifying themselves as Republicans were split down the middle.

Support drops below 50 percent when those surveyed were asked if they approved of research on embryos created in laboratories for the sole purpose of generating stem cells.

Emory ALS trial

The Shepherd-based trial is groundbreaking, but not the first time stem cells have been injected into a human spine.

That first happened nine months ago when Dr. Nicholas Boulis, a neurosurgeon at Emory, injected fetal stem cells into a patient with ALS, a disease that kills the nerve cells in the spine that control muscles. Since then, Boulis, who is working in conjunction with Maryland-based Neuralstem Inc., has performed five more such procedures.

The first phase was with ALS patients already unable to walk. But Boulis is set this week to move to a seventh patient, someone who is still ambulatory. The theory is the stem cells will do more good if injected earlier in the progression of the disease.

“As a surgeon, that is where the real Everest is,” Boulis said. “The stakes are higher.”

Boulis has developed his own patient-mounted platform to steady the needle as it is inserted. The device vaguely resembles a miniature oil rig floating atop a human back.

While the Neuralstem approach used cells from the spinal cord of an aborted fetus and uses a different device than the Geron researchers, Boulis is talking with Geron about merging the procedures. “It’s possible this landscape has room for different approaches,” he said.

“The chief medical officer of Geron is coming next week to watch us do our procedure,” Boulis said. “This is a story of collaboration, cooperation and also competition.”

John Cornick, a 51-year-old North Carolina man, was Boulis’ third ALS patient. Cornick said he has had no complications, although, he added, “we won’t really know until I die and they perform an autopsy.”

Cornick was advised his operation would do him little good. But he told researchers: “I’m in. Someone has to step up and do this.”

His disease has progressed since his operation in April and his speech is more difficult to understand. Cornick watched the news of the new study last week and said, “I’m 100 percent behind it. I’m still hopeful it will have an impact.”

His wife, Gina, supports such research but has concerns that those in the new study must quickly make a wrenching decision about their treatment. “I would worry those poor folks are still in shock,” she said. “But [researchers] probably pitch it that you have nothing to lose.”

Questions and concerns

Some scientists question the promise of embryonic stem cell treatments and believe adult stem cells will ultimately prove more successful. Jean Peduzzi-Nelson, a researcher at Wayne State University in Michigan, said embryonic stem cells are fraught with challenges, like the potential for developing into tumors or being rejected altogether.

Peduzzi-Nelson sees more potential in improving and saving lives by using stem cells from bone marrow or even by transforming ordinary skin cells into stem cells.

Right to Life’s Becker sees a slippery slope in the use of embryonic cells. “If we allow unregulated human research with no moral boundaries, we are opening a Pandora’s box to a society that will be unrecognizable 20 years from now.”

And while anti-abortion legislation in Georgia has often died in committee, Becker sees new momentum. He pointed to the increasing clout of the anti-abortion movement. This summer, Georgia Right to Life opposed Republican gubernatorial candidate Karen Handel, whom the group declared too liberal on its core issue.

Handel lost the primary to Nathan Deal. A spokesman for Deal said he supported President George W. Bush’s compromise for funding on a limited number of embryonic stem cell lines but prefers research to focus on adult cells. Democrat Roy Barnes supports embryonic stem cell research.

Todd McDevitt, Georgia Tech’s director of stem cell engineering, said not every fertilized egg will become a viable embryo. Some blastocysts — embryos just days old — are not high enough in quality to be used in fertility treatments, he said.

“In general, those will get destroyed,” he said. “But those nonimplantable blastocysts can be used to derive embryonic stem cells.”

Emory neurosurgeon Boulis understands many people say embryo cells can become a human and, therefore, should have rights. But he notes that they are frozen in liquid nitrogen and do not suffer, he said, getting a bit angry when talking about ALS patients who ultimately die of asphyxiation. “But it’s illegal to use [embryonic cells] to save someone drowning in his own saliva?”

A unique perspective

But even among the disabled, the ethical issues involved in this research can be wrenching.

David Jayne, a 49-year-old Rex man, was diagnosed with ALS 22 years ago. He lives on a ventilator, speaks through a computer and has become known nationally for his battles for the rights of the disabled. He is also politically conservative.

“Personally, if I was invited to participate, I would have to decline if embryonic,” he wrote in an e-mail. “Hell, I still struggle justifying being on the ventilator; I couldn’t live with myself taking a life to improve and/or extend mine. He or she might grow up and possess intelligence that makes Einstein look like Forrest Gump.

“With that said, I do support this research if the embryos are truly going to waste. I know this is a distasteful stepping stone that must be taken.

“The potential is unlimited. I dearly hope no legislation is passed in Georgia or across the nation limiting stem cell research. Yes, I still dream of this damn disease being a memory.”