First test patient gets embryonic stem cell treatment

Spinal cord therapy raises hopes and fears

THOMAS B. OKARMA The clinical trial “is a milestone for . . . human embryonic stem cell-based therapies,” said Geron Corp.’s chief executive. THOMAS B. OKARMA
The clinical trial “is a milestone for . . . human embryonic stem cell-based therapies,” said Geron Corp.’s chief executive.
By Rob Stein
Washington Post / October 12, 2010

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WASHINGTON — Doctors have injected millions of human embryonic stem cells into a patient partially paralyzed by a spinal cord injury, marking the beginning of the first carefully designed attempt to test the promising but controversial therapy, officials said yesterday.

The patient was treated Friday at the Shepherd Center, a 132-bed hospital in Atlanta that specializes in spinal cord and brain injuries, according to an announcement by the hospital and Geron Corp. of Menlo Park, Calif., which is sponsoring the research.

The hospital is one of seven sites participating in the study, which is primarily aimed at testing whether the therapy is safe.

Doctors will also conduct tests to see whether the treatment restores sensation or enables the patient to regain movement.

No additional information about the first patient was released.

The milestone was welcomed by scientists eager to finally move the research from the laboratory to the clinic, as well as by advocates for patients and by patients hoping for cures.

Although the cells have been tested in animals, and some clinics around the world claim to offer therapies using human embryonic stem cells, the trial is the first to have been reviewed by a government entity and aimed at carefully evaluating the strategy. After repeated delays, the Food and Drug Administration gave the go-ahead in July.

The move was criticized by those with moral objections to any research using cells from human embryos, and it is raising concern even among proponents. Some argue that the experiments are premature; others question whether they are ethical or fear the trials risk disaster for the field if anything goes awry.

“Without knowing more clinical detail, there’s little I can say,’’ said Steve Goldman, chairman of the department of neurology at the University of Rochester in New York. “In more general terms . . . I remain concerned about the long-term safety of unpurified grafts of embryonic stem cell derivatives. Time will tell.’’

David Prentice, senior fellow for life sciences at the Family Research Council, said: “Geron is helping their stock price, not science and especially not patients. It will be years before there is hard evidence about safety or effectiveness.’’

Supporters of the privately funded research are confident that it has been exhaustively vetted. The FDA has demanded extensive experiments in the laboratory and on animals to provide evidence that the cells hold promise and are safe enough to test in people.

“Initiating the . . . clinical trial is a milestone for the field of human embryonic stem cell-based therapies,’’ said Thomas B. Okarma, Geron’s chief executive, in a statement. “This accomplishment results from extensive research and development and a succession of inventive steps.’’

Donald Peck Leslie, Shepherd’s medical director, said: “Our medical staff will evaluate the patients’ progress as part of this study. We look forward to participating in clinical trials that may help people with spinal cord injury.’’

But some scientists worry that if patients are hurt by the cells — or if there is no hint that the cells help — it could be a devastating blow to the field. They cite the case of Jesse Gelsinger, whose 1999 death from a gene therapy experiment set that once-highly touted field back years.

Although Geron eventually hopes to test the cells for many different medical problems, the first trial will involve 10 patients who were partially paralyzed by a spinal cord injury in the previous one to two weeks. Surgeons injected the first patient with about 2 million “oligodendrocyte progenitor cells,’’ created from embryonic stem cells, in the hopes they will form a restorative coating around the damaged spinal cord. In tests in hundreds of rats, partially paralyzed animals walked.

In the meantime, officials at Advanced Cell Technology of Santa Monica, Calif., are hoping for the FDA’s go-ahead to start injecting stem cells into the eyes of 12 patients with Stargardt’s macular dystrophy. “Retinal pigmented epithelial cells,’’ also made from human embryonic stem cells, should replace those ravaged by the progressive loss of eyesight, which usually begins in childhood.

The company, which has laboratories in Marlborough, hopes that the approach will work for many conditions, including macular degeneration, the leading cause of blindness among the elderly.