September 24, 2008

Stem Cell Experts Urge Clinical Trials At End of Summit, Scientists Discuss Possibility of Human Testing in U.S.


Madison -- At a talk on stem cells and spinal cord injuries, professor Wise Young of Rutgers University described what happened once he began a series of five clinical trials in China.

Americans told him they wanted to go to China to join the clinical trials.

"It got me deep down," said Young, chairman of the Rutgers department of cell biology and neuroscience. "We should not be sending Americans to clinical trials in China. We should be doing clinical trials here in the U.S. It's shameful."

On the closing day of the World Stem Cell Summit, speakers confronted a complex but inevitable question. After all of the talk about promising results when stem cells have been placed in animals and in laboratory dishes, has the science reached the point when stem cells can be tested in human patients?

"I really believe that stem cell technology is at or approaching the tipping point, where this technology is really going to start to bring new therapies to patients," said John McNeish, the executive director of regenerative medicine at the pharmaceutical giant Pfizer.

"We believe that in the future, and perhaps not so far in the distant future, cells will actually be therapies."

While stem cells transplants are an accepted therapy for a few illnesses such as non-Hodgkin lymphoma and leukemia, they have yet to reach human patients with many other ailments. The result is a series of complex dilemmas that confront patients, scientists and policy-makers.

Patients with spinal cord injuries and many other conditions can wait for the slow process of clinical trial approvals to grind forward, or they can go overseas, where the multibillion dollar medical tourism industry offers treatments that often come with a high price tag and without rigorous studies to show they actually work.

The U.S. Food and Drug Administration has steered back and forth between caution and aggression, said Alta Charo, a professor of law and bioethics at the University of Wisconsin-Madison. The FDA risks a patient uprising when it moves slowly, and debacles such as Vioxx, the troubled arthritis/pain-relief medication, when it moves more rapidly.

And then there are the scientists. If their stem cell therapies show some success in animals, they face a daunting task in proceeding to clinical trials.

Young said it was easier to begin clinical trials in China than in the United States. The reason, he said, "is simple. Money. There's currently no source of funding for clinical trials (in the U.S.).

"You're really talking about an issue of priorities for this country. There's plenty of money to bail out investment houses and insurance companies. But there is not money to do the clinical trials for therapies for spinal cord patients."

The clinical trials in China are costing about $30 million, money that was raised privately in Hong Kong, Young said. The trials would cost five times more to run in the U.S., in part because the process here is longer. The spinal cord injury trials are not cheap because they involve not only a week of treatment but an additional three months of rehabilitation and physical therapy.

Young said many of the patients who seek stem cell treatment overseas are not desperate -- they're determined.

"No matter what we do or what we say, it will occur until we start satisfying the demand for therapies," he said.

Clive Svendsen, co-director of the UW Stem Cell & Regenerative Medicine Center, agreed that it is more difficult to reach clinical trials in the U.S. than in other countries.

"They're expensive. A lot of people are skeptical that they'll work," Svendsen said. "But they are coming."

To keep public support for stem cells, he said, "we have to fund some large-scale clinical trials, particularly with childhood diseases and spinal cord injuries. I think America needs to do it. The question is: Who's going to pay for it?"

McNeish, the Pfizer executive, said it generally costs $1 billion to bring a medical therapy to the market, but he could not say whether a stem cell therapy is likely to be more costly than a drug. One promising sign, he said, is that most of the major pharmaceutical companies now have formal stem cell policies.

"Clearly," he said, "the pharmaceutical industry is making the move to enter the stem cell field."

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