Posted on behalf of David Cyranoski
On 1 July, the Korean Food and Drug Agency approved a stem cell therapy for heart disease. Reuters reported that it will put “South Korea back in the stem spotlight.”
The “back” is a reference to the glory days of Woo Suk Hwang, now a scientific pariah, who committed scientific, legal, and ethical transgressions on his way to embarrassing the country. Given that, I’m not sure South Korea wants to be back in the stem cell spotlight.
Nor, judging from the text of the story, will it likely be.
The treatment, called Hearticellgram-AMI, was developed by FCB-Pharmicell and is supposed to help hearts regenerate after heart attacks when directly injected. After six years of clinical trials, the company boasts that “6% patients showed a near 6 percent improvement in heart function six months after one dose of the injection”, Reuters reports.
The article quotes a university president saying it’s the “first stem-cell medication to be approved for clinical use not only in Korea, but the entire world.” While it’s true that stem cell therapies for specific applications have gotten hung up in regulatory struggles, notably in the US and China, stem cell therapies in the form of bone marrow transplants have been around for decades (see this NIH guide for more background on adult stem cells).
And after the big headline, the article let’s us know why we shouldn’t think it’s a big deal. Oh Il-hwan, professor of molecular biology at the Catholic University School of Medicine in Seoul, and the extensively-quoted source who “previously sat on KFDA panels overseeing stem cell research”, says:
“The lack of independent expert scrutiny and insufficient testing of the treatment’s effectiveness were disappointing for what could otherwise be seen as an indisputable breakthrough. Whether the government has done everything in its obligations to be transparent with the public, when it comes to the effectiveness of the treatment, there is some disappointment about that."
“We’re not quite at the stage of picking the fruit of our labor to eat it. The consensus around the world is that we are still very much at the stage of research.”
Which sounds like it shouldn’t be in the clinic. But it will be, and if nothing else, Oh says, “This approval means a certification that there are no major problems in terms of safety. What it also means is that the (KFDA) will be watching what the market says about the effectiveness.”
Efficacy, not safety, is the stumbling block for most drugs and medical therapies. Let’s hope that the KFDA is watching for reports from the clinic.