SCOTS Study on Stem Cells for Vision: Still Questions as Patient Alleges Harm

Eye injection

Eye surgery image from Wikipedia. Is clinics injecting stem cells into an eye a good idea? OK with FDA?

I’m hearing more questions from the community about alleged harm from stem cell clinics selling stem cells for a number of vision-related conditions and some concerns include the so-called SCOTS study.

I first blogged about SCOTS early this year and back then I myself had questions too. Some commenters then raised concerns or questions about SCOTS in weighing in on that post.

In my opinion SCOTS is almost certainly not a traditional FDA phased clinical trial, which raises issues right from the get go. I’m not aware of the trial having, for instance, an IND. Patients must pay $20,000 to participate and that brings additional questions.

The clinicaltrials.gov listing is here (reminder in general that a listing on that website does not equal FDA approval or some kind of NIH approval of the actual clinical science). The company mentioned as the responsible party is Retinal Associates of South Florida in collaboration with another business, MD Stem Cells.

A self-reported SCOTS study patient, George Gibson, made allegations to Scientific American that he lost vision after participating in the study:

“But not every story of stem cell therapy has such a rosy outcome. George Gibson was in his late 60s when he partially lost his vision during heart surgery. He says he paid $20,000 to get stem cells injected into his eye with the guarantee that he would be able to read a few more lines on an eye chart. Gibson claims that instead, he lost vision in that eye completely, but his assertions could not be verified. There have been other reports of vision loss in stem cell procedures performed elsewhere. Gibson didn’t get one of the first-come-first-serve slots to speak at the hearing; instead, he and his wife stood outside the meeting room during breaks with big signs that read, “I lost my sight to the SCOTS stem cell procedure!!!”

To be clear, this outcome has not been independently verified and the SCOTS investigators dispute the allegation.

Another SCOTS patient, Doug Oliver, who has also commented on this blog, has apparently had the opposite kind of experience. He self-reported a very striking positive outcome after receiving the stem cells in SCOTS and this has gotten a lot of media attention.

Two case report publications are listed as associated with the SCOTS study:

So what is going on here?

The short answer is that we as a community don’t know yet.

In a number of different stem cell studies over the years we’ve heard about individual patients having striking outcomes. Anecdotal reports of alleged potential harm or benefit should definitely be paid attention to and given consideration, but what we really need is comprehensive data from properly controlled studies to have confidence that an investigational stem cell therapy is (or is not) safe and effective.

Related to this specific study, I’m very happy for Doug, but the SCOTS study needs to show/publish all the data including any potential adverse outcomes before we can know what is going on with this investigational treatment. Last week I emailed Dr. Steven Levy, MD who is running the study asking if I could do a brief email Q&A with him for the blog, but so far no reply. Dr. Jeffrey Weiss is listed as the Principal Investigator so I may reach out to him as well if I don’t hear from Dr. Levy.

Study that injects marrow cells into eye, charges $20K raises many questions

Eye injection

Eye surgery image from Wikipedia. Is injecting stem cells into an eye a good idea? OK with FDA?

Whenever I see a stem cell headline like this one from a recent Baltimore Sun piece, it raises many questions: Stem cells apparently reverse woman’s blindness.

First of all, is the reporter, Meredith Cohn in this case, being cautious enough with that “apparently” in the headline? Part of the reason for this question is that the “treatment” in question is unproven, so far there’s no public data beyond 1 patient, and I wasn’t able to find evidence of an IND for this so it’s unclear if the FDA is OK with it.

Second, I immediately wonder what kind of stem cells are being used. In this case, the physician who is doing the study, Dr. Jeffrey Weiss in Margate, Fla, is injecting bone marrow stem cells into the patients’ eyes.

Third, does it make any sense to inject bone marrow cells into a person’s eyeball to treat a vision condition? I’m not a vision or bone marrow scientist, but there’s no obvious rationale to me. Even the doctor doesn’t claim to know for sure how it might work. Anti-inflammatory? Mobilization of or enhanced survival of endogenous cells? Other?

Fourth, is it safe? Who knows. There would definitely be some risks associated with transplanting these stem cells into the eye. Update: a mouse study from right here at UC Davis a few years ago suggests more broadly that this kind of approach might be safe for one particular type of retinal injury ( HT to @SurgeonRetina on Twitter), but that’s a long way away from being sure that what the human study that we are focusing on here is doing is safe.

Fifth, is there a charge? In this case, yes, it costs patients $20,000. This makes me even more concerned. Does 100% of this money go to running the trial? Or is there some profit (i.e. total funds generated exceed total costs of the trial)?

Sixth, will this newspaper article encourage other patients to take the same risk? I think it probably will. The article includes some caveats, but enough?

Digging more into the article, there’s some confusing text about FDA regulations and how they apply to this trial:

“The U.S Food and Drug Administration must permit the use of “investigational” drugs, and the NIH requires that researchers attest they have such permission to register a trial. While the FDA continues to tweak regulations, there are exceptions when stem cells aren’t considered drugs, such as when they are minimally processed and taken from and used in the same person.”

I’m not clear what this means.

The FDA “must permit” investigational drugs she writes, but the way it is written is confusing and again I’m not aware that this study has that permission. Does it? If so, I hope to hear about that. Cohn leaves this issue up in the air.

She also doesn’t touch on the crucial homologous use issue. Cells must be used in a clinically homologous manner or they are automatically drugs requiring pre-approval. Does bone marrow seem similar to the eye?

The clinicaltrials.gov listing is here and the business doing the study is Retinal Associates of South Florida in collaboration with MD Stem Cells. So far I haven’t been able to find evidence that this is a traditional phased FDA-approved clinical trial.

Unfortunately we are seeing more and more of these kinds of unusual listings at clinicaltrials.gov. The listings are called “clinical trials” by those doing them and the media, and patients have to pay.

This kind of human stem cell clinical experiment is exactly the type of thing that we need more clarity about from the FDA.