South Korea is positioning itself in the vanguard of stem cell commercialization, with three adult stem cell treatments approved in the country over the past eight months. Yet, although officials inside the Korea Food and Drug Administration (KFDA) maintain that the therapies have been vetted according to international best standards, outside experts worry that the country's drug agency may be moving too quickly with its regulatory decisions.

“It took a lot of people by surprise that the KFDA was willing to approve these ahead of any country in the world,” says Douglas Sipp, a stem cell policy researcher at Japan's RIKEN Center for Developmental Biology in Kobe. “There has not been a lot of information released about the details of these studies or the regulatory approval process in English.”

The first approval came in July 2011 when the KFDA approved Hearticellgram-AMI for treating heart attacks. Developed by Seoul-based Pharmicell, the therapy consists of mesenchymal stem cells cultured from a patient's own bone marrow that are injected into the coronary artery to improve heart function. Traditional transplants of blood-forming stem cells from bone marrow and peripheral blood are regulated under separate terms from other human cell and tissue products in many countries, and so Hearticellgram is the first stem cell 'product' approved by a regulatory authority anywhere in the world. (The second approval came just four months later when the US Food and Drug Administration legalized its first stem cell product, a cord blood stem cell–based therapy called Hemacord, from the non-profit New York Blood Center.)

The KFDA then forged ahead in January with stem cell go-aheads for two more Seoul-based companies: one for Medipost's Cartistem, which uses banked umbilical cord blood stem cells to help regenerate knee cartilage for people undergoing orthopedic surgery and the other for Anterogen's Cupistem, an injection of fat stem cells derived from a patient's own abdomen or thigh that is intended to help people with Crohn's disease who suffer from painful anal fistulas.

Notably, Hearticellgram-AMI and Cartistem are the first licensed stem cell treatments that use bone marrow– or cord blood–derived stem cells to treat diseases outside of the blood and immune systems. Yet, some researchers worry about the efficacy of products of this sort, given the lackluster data released publically to date—and what is known about these agents is mostly through press releases by the KFDA and presentations, not peer-reviewed journals.

Cartistem's results look the most promising. In an 89-person Korean clinical trial, 26% more people who received the Medipost treatment experienced an improvement in knee function on a widely used cartilage repair assessment scale compared with those who underwent knee surgery alone. Meanwhile, a 59-person trial with Hearticellgram-AMI found that people who received the stem cell product could eject around 6% more blood out of their left ventricles at the end of the six-month study, compared with just 2% improvements in people who received standard medical therapy.

Show me the data

Company officials say that peer-reviewed results are coming soon. According to Francis Han, Medipost's head of research and development strategy, “the doctors running the clinical trials are now gathering more long-term follow-up data on the patients for publication.” Similarly, Anterogen spokesperson Kim In-ok says that the company is in the process of preparing a journal article based on findings presented at last year's European Society of Coloproctology annual meeting in Copenhagen.

The KFDA, for its part, stands by its record. Ahn Gwang-soo, deputy director of the agency's cell and gene therapy products division, points to the strict procedures in place to ensure that medicines are safe and effective. “Although there were some companies that conduct stem cell treatments outside of Korea,” he says, “they are not permitted to administer those treatments within Korea.”