Photograph by Mauricio Alejo

According to one small biotech, the best way to launch a stem-cell revolution is to do it overseas.

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."

Some doctors in the United States have given TheraVitae's treatment strategy the nod -- in principle, anyway. Cardiologist Warren Sherman, the director of stem-cell research at Columbia University's Center for International Vascular Therapy, has been investigating techniques for years that are similar to the approach TheraVitae is using. "The stem cells stimulate vascular growth, and that can either salvage heart-muscle tissue that would otherwise be susceptible to death, or recover the function of muscle cells that might be dormant," he says. Some U.S. clinical trials of related therapies are currently under way at the Texas Heart Institute and elsewhere, but since the enrollment in these trials is limited, they aren't a realistic option for most patients. "The majority of animal studies so far are strongly suggestive of benefits from this technique," Dr. Sherman says. "Also, the initial results from human clinical trials are positive enough that I think we're justified in moving forward on a larger scale."

Every country has unique regulatory standards for approving clinical therapies, and TheraVitae's Thai trials fall short of U.S. clinical protocols. They don't include a control group, for one thing, which would help establish how much of the patients' improvement was due to the treatment and how much to good basic care or even the placebo effect.

"When these findings are not reported in a refereed journal, you can't take them seriously," says Dr.Marius Wernig, a neuropathologist at Stanford's Institute for Stem Cell Biology and Regenerative Medicine. He won't recommend unaccredited overseas therapy under any circumstances. Even Dr. Sherman, who sees promise in the therapy, doesn't think its benefits are proven enough to give it to patients outside of clinical trials.

Smeenk insists TheraVitae's decision to operate overseas represents an attempt to maintain a delicate balance, providing patients with safe and effective treatment while turning a profit at the same time. (The company will not reveal its finances, though it says it has treated 450 patients.)

The firm's future aside, its decision to bypass the FDA approval process raises some important questions: Does the time and expense that approval requires make it a no-win prospect for small companies with innovative ideas but limited funds? Should the FDA ease the guidelines that authorize companies to give patients with few other options access to an experimental therapy like VesCell -- even if the therapy has not gone through the full FDA clinical-trials process?

"In Southeast Asia," Smeenk says, "if a therapy will do no harm and it may do good, and these people have no other options, they say, 'What's the issue?' "

But many physicians and scientists argue that, in a field like stem-cell research, cutting regulatory corners could usher in therapies that are ineffective or even harmful. "Stem-cell tourism is deplorable," says Hans Keirstead, director of the University of California, Irvine's stem-cell research center. "The best way to destroy a medical technology is to take it to a third-world country first -- no big pharmaceutical company will pick it up. A treatment will reach the greatest number of patients fastest by going through the FDA."

Rumble disagrees. Although he still technically suffers from congestive heart failure, he says his ejection fraction -- the amount of blood his heart pumps out with each beat -- has tripled in the 17 months since he returned from Bangkok. That boost in cardiac efficiency, he says, allows him to walk 3 to 5 miles a day without breaking a sweat. He says even his doctor at the Florida Heart Center concedes that his condition is much improved. "I would definitely get the VesCell procedure again, a hundred percent," Rumble says. "If I had just stayed the way I was, it would have been a death sentence."

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