Clinical trials inside: What happens with patients who get a placebo?

by Alexey Bersenev on December 31, 2008 · 5 comments

in clinical trials and cases

It’s known that to get your clinical trial protocol approved by the FDA in US is very tough and complicated. Since the FDA also covers all of the cell therapy clinical trials in USA, both companies or hospitals and patients suffer from very long approval procedures and bureaucracy. Many desperate patients, who are waiting for some kind of magic from “stem cell therapies”, are forced to look for non-proven cell therapy treatment outside of US. Other part of patients, who have no options to be treated by standard protocols, are looking for any news and trials launched by companies or hospitals to be enrolled.

But we (watchers) barely understand what’s going on inside of these trials. Today I’ve read a very interesting post by Gideon Sofer – a student at U of California, written for Wall Street JournalThe FDA Is Killing Crohn’s Patients and want to share it with you.

To date, half of my intestine has been removed to manage Crohn’s disease. Last year, at age 23, I enrolled in a clinical trial for a treatment that could save my life: an adult stem-cell therapy that helps damaged intestinal tissue regenerate from the relentless inflammation and scarring caused by Crohn’s.

The sponsor, Osiris Therapeutics, reported that Crohn’s patients in the therapy’s Phase II trial all experienced clinical improvement after receiving the cells. A Phase III trial for the treatment is now nearing completion, but Food and Drug Administration (FDA) approval could be years away, despite its FDA “fast track” designation.

Apparently he got into the “placebo group” and this is what happened next:


Foregoing all other treatments, I received the four scheduled infusions, and yet my disease progressed with a vengeance. In a matter of weeks, I became dangerously malnourished. I’ve since been readmitted to the hospital countless times, as my doctors continue to plead with Osiris for information. But Osiris has refused, citing adherence to FDA protocol.

I am now a lab rat. I have no right to know what happened to me in the study, nor do I have a right to try the promising treatment as my health deteriorates. It doesn’t have to be this way.

It’s a pain to read this and realize that some day you or your relatives can be in his place. Even though it’s a common problem of clinical trials regulation in US, I’m just curious about the experience of patients in other countries. In particular, I’m interested in experts’ opinions about “cell therapy clinical trials” regulation. Should it be under control of organizations regulating trials of “drugs and biotech products” or should it be separate, because “therapy of live cells is a special”? Should all of the patients in the placebo-group get the real drug at the end of trial if it really works?

{ 5 comments… read them below or add one }

Joanna December 31, 2008 at 1:01 am

This is an interesting story. In my opinion, in medicine there is always an imperative to treat the patient, even the control group in clinical trials. So, after the trial is done, I agree that those in the placebo group should receive treatment, if the therapy was shown to be effective. This guy’s case, however, sounds like it was complicated by the lack of transparency in the process of what happens when things go wrong. I don’t think patients’ health is prioritized at the top in today’s healthcare system at all. In order for patients like Sofer to really benefit, the whole perspective of the people launching such trials needs to change!

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Alex December 31, 2008 at 2:07 am

thanks Joanna, I agree.
I think placebo group should get a real drug at the end of trial if efficacy was confirmed. That actually the end of phase II.

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Alex December 31, 2008 at 3:34 am

also you can read discussion on Wall Street Journal forum –
http://forums.wsj.com/viewtopic.php?t=4865

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don margolis December 31, 2008 at 4:53 pm

Dear Joanna:
Your key sentence is far more appropriate than anything in the article itself:
“I don’t think patients’ health is prioritized at the top in today’s healthcare system at all.”

It is far worse than you imagine. What can you say about a system where every medical researcher in the country knows that adult stem cells can treat not only Crohn’s Disease but virtually every form of heart disease, but long-proven ultra-safe stem cell treatments are withheld for decades.

Why? Because “at the top” of the so-called healthcare system, controlling EVERYTHING, are the multi-billion dollar profits of the drug companies, HMOs and insurance companies. You will not see any serious inroads made by stem cells for decades as long as they continue to own congress and the White House. Patients don’t come in first, second, or third. Their motto should be: “We shall lie and you shall die, but WE will profit greatly.”

So you are lied to, being told that embryonic stem cells are the answer and adult stem cells are “snake oil.” That way, they KNOW that embryonics will NEVER provide one cure, and as long as you believe the “snake oil” argument they will be able to stop adult stem cells from ever becoming available. Clever, eh? Profits locked in while YOU suffer and die from treatable diseases.

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William Hardison September 14, 2016 at 12:47 pm

I’ve gotten a placebo participating in stanfords stroke trial. They drill a hole in your head and I promised to follow up for a year.

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