Biotechs Look Overseas to Launch a Stem-Cell Revolution

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Photograph by Mauricio Alejo

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The Price of Approval

Three years ago, Mike Rumble could barely walk without having to stop and grab his chest. High-dose chemotherapy for leukemia had damaged his heart muscle back in the 1980s; then a bout of viral pneumonia "wreaked havoc on my already-weak heart," says the 53-year-old Boynton Beach, Florida, native. "I was in the hospital and had to have a defibrillator put in."

The diagnosis: congestive heart failure, which meant his heart wasn't strong enough to push blood out to the rest of his body. Some 550,000 Americans are diagnosed with the condition each year, and about 260,000 die from it. "My doctor kept saying, 'You're going to need a heart transplant,' " Rumble says, "and I wasn't too keen on that."

Clicking around online in search of some lead -- any lead -- that might improve his prospects, Rumble's wife stumbled upon the Web site of Toronto-based TheraVitae, a small company with offices in Israel and Thailand. The company was offering a therapy called VesCell at hospitals in Bangkok, Thailand, and the Dominican Republic. The treatment, the site explained, involved taking 250 ccs of a patient's own blood, extracting and growing the adult stem cells, and injecting them directly into the heart muscle. Once there, the cells would develop into mature cardiac-vessel tissue. This process was said to help regenerate dying blood vessels within the heart, reviving and strengthening the cardiac muscle.

VesCell has not been approved by the Food and Drug Administration for use in the United States. But after poring over the scientific data posted on the Web site, Rumble resolved to raise the $35,000 needed for the procedure and head to Bangkok -- against the advice of some of his doctors. "I didn't feel any fear whatsoever," he says, "because I knew exactly what I was going into."

Executives at TheraVitae have balked at the idea that FDA clinical trials should constitute the company's sole route to success. "To bring a biotech company through the FDA process over 10 years," says TheraVitae VP of business development Thomas Smeenk, costs hundreds of millions of dollars -- a nearly impossible hurdle for a young firm. Instead, TheraVitae has conducted its own low-cost clinical trials in Bangkok, which show that the therapy can improve the heart's ability to pump blood by more than 20%, according to Smeenk. (A trial evaluating VesCell's effectiveness for treating arterial disease is in progress in Hungary.)

Smeenk says the company is pioneering a new business model for the rapidly evolving stem-cell marketplace: conduct clinical trials overseas to demonstrate the utility of a therapeutic technique, then use those trial results to persuade investors to shell out the big bucks required for FDA approval. "When you take that overseas patient data and use it to generate an FDA clinical trial," he says, "you pretty much know the outcome before you start. There's less risk for investors in that case." Smeenk claims VesCell's overseas history will keep the cost of winning eventual FDA approval down. "Our Phase II trial in the U.S., for about 40 patients, is expected to cost $1.6 million to $2 million," he says, "and Phase III, $40 million," if the FDA were to require as many as 1,000 trial subjects.

These days, a quick Google search calls up a sweeping assortment of stem-cell treatment centers in places as far-flung as China, Russia, Mexico, and Ukraine. Many sites highlight testimonials from patients who claim to have benefited from the therapy. "People say their wife or kid went somewhere and threw away the wheelchair, but there are a range of providers from reckless to reputable," says stem-cell expert Robert Lanza, chief scientific officer at Advanced Cell Technology.

By most accounts, TheraVitae tends toward the reputable end of the spectrum. Papers on its scientific methods have been published in peer-reviewed journals such as the British Journal of Hematology and the European Journal of Heart Failure. In addition, the company presented early results from clinical trials in Thailand at an American Heart Association meeting in 2006. According to the report, 90.9% of patients tracked six months after their treatment topped their original performance in a six-minute walking test, and also improved in other measures of cardiac function. The World Economic Forum has also tapped TheraVitae as one of its "biotechnology pioneers."