Provenance of Clinical Trials

Clinical trials are inherently risky - that's why they are clinical trials and not standard protocols. No amount of vetting or due diligence will tell you what to expect when something entirely new is tested, especially in Phase 1 or 2 trials. And even if there were animal tests previously, humans are not mice. Stem cells, immunotherapies, and various chemos have caused deaths in clinical trials. But some have caused almost miraculous improvements. Determining which is which and in whom is what clinical trials are for.

People seem to want it both ways without thinking about it. You should want it well vetted before it goes on market, not before it is experimented with. Without even reading more about it, this was clearly phase 1 or 2 (out of a 3 phase process). I say God Bless the pioneers.

Trials are treatment.

Could this one have been done differently? Probably. Was it done inappropriately? I don't know. Does the article cover the entire trial or just these three women? If a thousand had been successful what would be the report? Not to diminish their problems at all.

But to TA's point, there is risk inherent in all treatments whether in a trial or not, and that risk might be greater in trials as they are trying to figure out how best to deliver the treatment.

With MD these women were, unfortunately, going blind anyway, just more gradually. That is what MD does. I have a related condition (BRVO) that requires the same type of treatment (injections every six weeks in the eye). It comes with risk of infection that can blind the eye. One medication has a known higher risk so we don't use that one. I have known about the risk from the start and my doc keeps me well informed about problems he knows about. A few years ago a formulary pharmacy mixed some drugs incorrectly and that resulted in several people infected and thus blinded in the Augusta, GA area.

However I have maintained significant sight for more than six years so far by accepting the risk. Without the treatment I would now be nearly or totally blind in that eye. My doctor participates in clinical trials, but has not entered me into any. He contends that I am responding well to the current best treatment and he hasn't seen any breakthroughs yet that he would want me to try. If what we were doing wasn't working, we might be entering a trial.

You imply that trials are not regulated and that just isn't true. The NIH is not the only, or even the main governmental agency that manages the trial process. I suggest that you dig into the trial process and learn how regulated it is before making possibility uninformed judge-mental statements regarding trials. I am sure the Post article was in no way an exhaustive report on how trials are run. There are a myriad of requirements and oversight to first ensure safety and then to ensure the results are valid, reliable and repeatable. That is what is needed to bring a procedure, device or medicine into the mainstream.

You might reach out to fellow HW member and former moderator, Tony Crispino to learn more about the process. He is a patient advocate with the Southwestern Oncology Group (SWOG). They are one of the premier sponsors and organizers of cancer trials. Many of the methods and medicines you and I and other members of the board have used came to us through trials they managed.

yeah...clinical trials are a good thing

I would be extremely leary of any trial run by a private (for-profit) clinic...

And as Tall Allen already pointed out, even trials run by well-known medical scientists in a university setting may have disastrous results. Remember the U Penn gene-therapy trial?

I dug into the report and status of these trials and found the following.

The two ladies in Florida were treated in 2015. They have settled with the providers confidentially, so no reports of the settlement are available. The clinic reports they have used the procedure with more than 7,000 patients with no other adverse situations. The detailed reports indicate that the injected material was probably contaminated in the production, thus causing the infection.

The lady in Arizona suffered a detached retina. This is one of the most common side effects of MD, so not uncommon. It has not yet been determined whether her situation was caused by the injection.

The stem cell process is similar to the way Provenge, which we all know about, is produced. Either fat or bone marrow is extracted from the patient. The stem cells are extracted and used to manufacture a substance that is then injected into the eye. The expectation is that the stem cells will revitalize the macula densa and reverse or slow the degeneration. It is hoped that this would also reduce the frequency of injections as many have them monthly. Thus there are several steps in the process that could introduce contaminants just as in any other drug production.

I then searched for registered clinical trials. I stopped looking at around 100 trials for MD and stem cell use. It appears there are thousands of people in these trials. I found that most were sponsored by the big pharmaceutical companies as they are developing the drugs and have the corporate resources to fund the trials. Next were the trials sponsored by universities. A very small number are sponsored by firms that I didn't recognize. I researched a few of these and found them to be research subsidiaries of other well known companies and some were biotechnology companies that are smaller and newer. Many are directed by private equity firms funded by Silicon Valley and other technology money billionaires. This is how companies like Genentech and many others got started.

So from the research and development perspective, thank goodness for profits. They are serving us very well funding most of the R&D that benefiting all of us.