The Future of Neural Stem Cell Therapy

In the early 1990s, as part of therapy designed to restore neural function in patients with Parkinson's, Curt Freed, a neurosurgeon at the University of Colorado, performed transplantations of cells derived from fetuses. Now, however, "we're moving into an era in which we'll create cells in the laboratory," Freed says. "I see the cell therapy area as becoming very sophisticated during this century. Two hundred years ago, drugs were not pure products but were ground-up leaves." Today, chemists can refine drugs to nearly 100 percent purity. "I think exactly [the same thing] is going to happen with cell therapy," he predicts.

That is the business model of Stem Cells Inc., which purifies fetal cells, multiplies them a thousandfold, and freezes them in "cell banks" for individual patient use. A cell population grown by this technique is much more uniform than the original sample, which should keep results consistent from patient to patient. "Because we can bank and store them," explains Rodney Young, CFO of Stem Cells, the company expects eventually to profit as the pharmaceutical industry does, by selling a product--"stem cells in a bottle."

Even though the primary goal of the initial Batten trial is to discover whether or not the treatment is safe, the OHSU/Stem Cells researchers expect that the doses of stem cells used in this trial could have therapeutic effect in the children. "We certainly will publish the results & whether they are positive, negative or neutral," Steiner says. In the meantime, "we don't think it's helpful to have a lot of hype about this study. We just want to do our work and take care of the children as best we can."

Twenty-eight days after the surgery, the parents of the first patient in the Batten trial held a press conference to thank the doctors involved. "There were doctors and nurses and so many specialists & it was a symphony of love for our son," says Marcus Koerner, the father. "The first news we got was that the first injection of neural stem cell--transplant cells--had gone in & everyone was so elated."

The boy emerged from surgery with a shaved head and incision scars. Steiner and the other surgeons warned the parents of possible side effects but all has gone smoothly so far. The child, who had seizures before the surgery, has had none since and has even spoken words the parents have not heard in a long time. With Batten disease, language ability can fluctuate and this improvement may have been caused by the support the child experienced in the hospital. "We are way too early in the uncharted territory [to be certain the surgery has had a beneficial effect] & but as parents," Koerner says, "we are overjoyed."

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