Stem Cell Treatment Alert: More (or less) than meets the eye?

August 14, 2017

by Jennifer Phillips, Ph.D.

If you, like me, have Google Alerts or similar settings to find new Usher syndrome stories online, you may have woken up this weekend to the surprising headline “Ushers [sic] Syndrome improves with stem cell treatment”. News breaks late here on the US West Coast, so I’m sure a lot of you saw this article before I did, but as soon as I read it, I knew I’d have to write something about it for the blog. I’ll write a bit more about the specific claims below, but first off, a few things stand out about the article right away:

It’s a press release, not a scientific article. There is no data presented by which the reader can judge the validity of the claims being made, no source material to follow up on.
Even as press releases go, it’s pretty weak tea, published by a generic clearinghouse for press releases, rather than, say, an affiliated hospital, university or professional organization.
Whoever wrote it seems to know very little about Usher syndrome.

A slightly deeper dive into the organization providing this stem cell treatment reveals a few more important points:

The entity apparently managing this experimental stem cell treatment is called MD Stem Cells, which has partnered with a doctor in Florida to embark on what they term SCOTS, or Stem Cell Ophthalmology Treatment Study.
The doctor doing the stem cell transplants appears to be trained as a retinal specialist, but is also affiliated with an organization called ‘The Healing Center’ which offers generic ‘treatments’ for a wide variety of conditions. This is a giant red flag for me, as it is the premise for alternative treatments well outside the bounds of science-based medicine.
To put the fringe on that giant red flag, I note that, on both the MD Stem Cells and Healing Center web pages, there are wordy disclaimers about the unreasonable restrictions of government funded, peer-reviewed biomedical research, prickly FDA approval, and the like. It seems to be a built-in defense against having to provide, well, any evidence for the claims they make.
Although SCOTS (and the new iteration SCOTS 2) boast an Institutional Research Board approved status and registration on the NIH Clinical Trials page, the treatment they offer is not well explained and *NOT * FDA approved.
In an unusual move for a clinical trial (which in the vast majority of cases is a funded study of an experimental treatment), patients participating in the SCOTS trials pay tens of thousands of dollars out of pocket. It’s not specified anywhere I can find precisely what this money is used for.

So, what did the press release say, exactly? I can’t bear to link to it, honestly, but in a nutshell, it reports that one individual with ‘Ushers syndrome’ (type not specified) was treated with a stem cell injection to the retina, and had unspecified improvements in vision and in hearing. That’s…incredible, to say the least. Regarding the stem cells, previous information from this company shows that they extract bone marrow cells from the patients and transplant them to the retina(s). There are indeed stem cells in bone marrow, but, absent some significant in vitro manipulation, they’re not the kind that has ever been known to spontaneously become retinal cells. There’s no proposed mechanism for how this works, no cell preparation protocol to inform us here. Beyond that, it’s extremely hard to imagine a plausible biological mechanism for how stem cells delivered to the retina could affect hearing.

So, on the one hand, we have a handful of individual case reports or press releases reporting extraordinary outcomes from injecting patient-derived bone marrow stem cells into the retina. On the other hand, there are more than a few concerning questions about the plausibility, oversight, safety, and ethics of this operation.

My reservations are deepened by the fact that earlier this year, another similar stem cell therapy company made news when three patients were blinded by experimental stem cell transplants. Additionally, although the cases aren’t clinically documented, some patients participating in the SCOTS trial have complained of poor outcomes, as described in a recent BBC documentary. Stem cell researcher Dr. Paul Knoepfler has expressed serious misgivings about the standards, ethics, and risks of human trials of non-FDA approved stem cell therapies. I’m not an expert on stem cells, but Dr. Knoepfler is. Regarding these types of clinics, he cautions:

"Overall, stem cell-based approaches to vision loss have real promise, but in my view should only be done in FDA-approved trials with [clearance to test stem cells as a ‘new drug’] based on extensive rigorous pre-clinical data."

I completely agree. Scientists who study how stem cells work, and how they can be programmed, are the ones with the best perspective on how feasible this type of treatment can be. We know the potential is there, but the molecular regulation of stem cells—how to get them to become what you want, to integrate into living tissue and perform the desired function without doing anything ‘rogue’ or disruptive—is extremely complicated. Knowing this, I can’t help but be highly skeptical of a company whose business model seems to be “hey, we just threw some non-neuronal stem cells into the eye and all kinds of fabulous things happened!”

In summary, this treatment can offer no confirming data for its claims, engages in a lot of special pleading to excuse its lack of FDA approval, and carries considerable risks with no clear gain. I am keenly aware that the clock is ticking for members of our community who are losing their sight. I can only imagine the hope that headlines like this might ignite, in a world going dark, but please, my USH family, do not rely on testimonials and press releases to influence your medical treatment decisions. In the very best case scenario, the doctors conducting these SCOTS trials will eventually disclose enough of their research findings, with sufficient supporting data, that some sense can be made of the claims they’re making now. Until then, please proceed with extreme caution in cases where reports of individual improvement cannot be verified or vetted.

For further reading, Dr. Knoepfler has several posts about the SCOTS trials and the entities behind them on his blog.