First Stem Cell-Based Therapy Gets Approved – In Canada | Hacker News Reader

First Stem Cell-Based Therapy Gets Approved – In Canada

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And absent the FDA, first generation therapies based on stem cell transplants would have been available in the US for much of the past decade, and we'd have had a few years of second generation therapies devoted to small patient populations like this one.

Meanwhile, the large patient populations (e.g. heart disease) continue to have to leave the country to find stem cell therapies. The FDA makes it prohibitively expensive to develop therapies, steps on groups that try to offer stem cell treatments akin to those widely available elsewhere in the world, and thus encourages a black market:

http://www.fightaging.org/archives/2011/11/an-unusually-clea...

http://www.fightaging.org/archives/2011/12/when-you-make-med...

http://www.fightaging.org/archives/2012/01/research-and-clin...

http://www.fightaging.org/archives/2010/08/fda-tries-to-shut...

Regenerative Sciences, Inc., a Colorado medical practice that specializes in the use of a person's own stem cells to help patients avoid more invasive orthopedic surgery, announced today that the US Food and Drug Administration (FDA) is seeking to enjoin the clinic physicians from practicing medicine using patients' own stem cells. The lawsuit will allow Regenerative Sciences to question the FDA's policy that adult stem cells can be classified as drugs when used as part of a medical practice. ... The FDA will finally answer our questions, in court, about their claims and jurisdiction as opposed to doing everything in their power to avoid the issue that we are not a drug manufacturer, but simply a medical practice.

This is mentioned in the article. So why does a company based in Dulles, VA have to go to Canada to get approval? Well, as promising the results are, they no doubt are surprising to some. In 2009 Prochymal suffered a major setback as two separate trials showed that, while the drug was safe, it failed to improve the condition of patients with graft-versus-host disease. This treatment was shown twice to be ineffective, and therefore not worth the risk of injecting yourself with an experimental batch of processed stem cells. Also, even with the failed 2009 trials, the FDA isn’t dragging its feet. Prochymal is currently being tested in trials that will assess, not only its ability to mitigate graft-versus-host disease, but Crohn’s Disease as well.

What's really disturbing is that the FDA has flunked Prochymal twice now (if I'm recalling correctly). Which is why Osiris went to Canada to get an approval, hoping to demonstrate safety and push it globally, as it's still an X factor whether the treatment will ever make it past the FDA.

Osiris even has the Pentagon / DoD in their corner (and had Genzyme as their partner for a long time to boot), as Prochymal is being developed under a government contract to treat skin radiation burns and similar. If you can't get a drug through with those boosters.......

It was proved safe in the US trials. It was also proved ineffective twice. That's why the FDA "flunked" it.

Patients were given infusions twice a week for four weeks. Soon after first infusion, 63 percent showed a “clinically meaningful” response (no further details).

I am pretty sure that in drug trials, "clinically meaningful" just means it's measurable beyond the threshold of statistical noise; it does NOT mean a what most people would think of as a meaningful or marked improvement in the patient's condition

What's so disturbing about the FDA's actions? Prochymal was found to be no better than a placebo when used as a treatment in two clinical trials for graft-versus-host disease. Sounds to me like the FDA is doing exactly what it is supposed to be doing -- preventing companies from selling snake oil as a legitimate medical treatment in the US.

http://www.nature.com/news/2009/090909/full/news.2009.894.ht...

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