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.

18 thoughts on “Study that injects marrow cells into eye, charges $20K raises many questions


  1. Dr. Knoepfler,

    I really enjoyed reading your recent blog entry regarding the private clinical trial using bone marrow stem cells to treat retinal disease.

    I’m an MSW (Masters of Social Work), with a 20-year career in medical social work under my belt, so I can understand both sides of the perplexing issues you raise about the process of this trial’s research, its financial interests (being patient-funded), and its compliance with FDA regulations, which are, as you mentioned, quite vague at this point over the nature of this kind of clinical research.

    Research like yours is producing amazing results. But, so are the small shop trials. Especially those who have done international research.

    The reason I’m responding to you is because I also believe the small research programs, especially the apparently EFFECTIVE ones, must be supported and encouraged by big research and social policy. . Doing so will only provide MORE amazing data that both large efforts and small efforts can develop into future advancements… much more quickly.

    Also, on the concern you express regarding patient-funding. I argue that it used to be a stronger ethical issue when only non-profit or governmental funding sources were used. But today that is less common, so extraordinary measures, which don’t always work, need to be in place to keep from inviting bias into a study. These days, it seems a rare situation that a clinical trial isn’t somehow subsidized by private for-profit funding.

    With the trial in question, the procedure is carried out at an accredited surgery facility. Board-Certified Ophthalmologist, Anesthesiologist (M.D.), Interventional Radiologist, Orthopedic Surgeon are all present in the OR, along with their teams. I’m pretty sure the $20K is well-covered as far as “costs of the trial” is concerned (not even considering overhead and administration of the trial itself).

    Here’s the thing. I, too, had treatment through this clinical trial. I have macular degeneration, and have been legally blind for 10 years.
    In August, I was 20/400 in one eye and 20/2000 in the other. I had treatment at this clinical trial on August 18th.
    Now I’m 20/30 and 20/40 and my field of vision improved from 40% compromised to nearly normal in both eyes.
    I have Vanderbilt Eye Institute documents to prove it.

    And to top it off, I’m one of eight trial subjects that has gotten their drivers’ license. For me, in December.

    Also know that there are many other patients, some of whom I am acquainted with, who have experienced real improvements that are verified by their own retinal or ophthalmologic specialists.

    I hope my credentials on this matter and my comments are meaningful to you, especially since someone like you is in a position of influence and, if inclined, could do a great service to many, many people by rethinking the intensity of national stem cell holy grail competitiveness. And maybe even concluding as well that we should NEVER discard something that is working. And changing lives.

    Let some organic, inductive research take place. It’s fun to reverse engineer it and help others.

    Best to you and thank you for your contribution to some wonderful efforts.

    Doug Oliver


    • @Doug,
      Thanks for sharing your experience as a patient and your thoughts on the trial. I’m glad you are doing well. If the experimental therapy being administered is really that effective (and frankly, it does sound too good to be true overall), then those running the trial should collect the data, analyze it, and publish it in a peer-reviewed journal. Too often that doesn’t happen, but maybe it will in this case.
      If you have the contact info for those running the trial, I’d like to interview them for the blog so if you could share that it’d be great either here via a comment or my email.

      I’m still also concerned about that $20K price tag for many reasons. Just one issue is that it means access to the trial is only granted to those who can pay that big chunk of cash. Many cannot. Is that fair?
      Best,
      Paul


      • Yes, that’s the contact information. If you need a copy of my Vanderbilt Eye Institute validating medical records, let me know. It may not be too good to be true in the cynical sense, but I assure you, it is true. I actually think this trial is more of a study. I know they’ve already published a couple of articles in Neural Regeneration Research Journal and have a couple more in the peer-reviewed pipeline.

        Doug Oliver
        Nashville, TN


  2. @Doug – great to read a post from someone actually participating in a stem cell trial – also glad to hear that you experienced some positive news.

    Do all volunteers in the trial pay $20,000 (i.e. $6 million in fees for 300 enrolled)? How is this fair in a “trial” where some folks may get no response at all? Or are these simply routine customers of the clinic who pay at their own risk, and who’s data is now collected as for a volunteer trial?

    That would explain why this is a non-controlled, non-randomized study, which unfortunately will only give limited data on real effectivity.

    You said, “I believe the small research programs, especially the apparently EFFECTIVE ones, must be supported” – I can’t agree with support for studies that are “apparently” effective, as this is the misleading data that dubious clinics use to confuse patients. We should support controlled trials with peer-reviewed data or we waste money and patients’ time.


  3. TO Douglas Oliver.
    Did you get subretinal or intravitreous injection So? Did your druses and geographic atrophy improve following stem cell injections? Did your visual acuity remain stable without further treatments? Thanks. Gavan


    • plz email me ahmadfarouki7@gmail.com. I have done the stemcell whit Jeffrey N. Weiss, I have lost so much site after stem cell surgery . I can give you many other people who have done the surgery and lost so much sighte after the surgery . If you do it you will regret it .plz do not do it and contact me first .


  4. I have maintained acuity for one year. Longevity is not known, but it’s a treatment, not a cure. My condition is genetic, so it may express itself again if given the same conditions. I had one eye treated subregion ally and the better eye intraviteously. The sub retinal eye appears to be laying in new RPE cells, and may help the atrophy, but we’ll know more in a few months. The other eye still has atrophy that is stable. The drusen is receding or resolving from the maculas of both.


  5. I m sufferng friom bulabr mnd since late 2012 right now everyhing is outof control i have severe swallowing prblem anybody out there can help me please tks
    don ng


  6. This so called trial is a sham , these doctors ( Steven Levy & Dr Weiss) both do not even follow up after the procedure and if the procedure impacted negatively then they would not want to reply to your emails. My night vision has severely decreased after the procedure.


  7. dear paul, i am a clinician reading , researching about stemcells for the last 8 years. Your doubt about the logic of injecting bonemarrow stemcells into eye surprises me because i presume you to have some background knowledge on normal human physiology. Whenever any organ like heart, kidney, brain etc undergo a damage say ischemic insult resulting in heart attack, stroke etc. cardiomyocytes, neurons die. The dead cells release enzymes [ like CCK,LDH etc which we measure and confirm the diagnosis of heart attack] that go through blood stream and knock at the doors of bone marrow which releases immediately a small stock of HSC,MSC,EPC and a concoction of stem cells which travel to all parts of the body but home only to the affected organ sending chemical signals of damage[.You know that Homing is an important attribute of stemcells]. Subsequently for some days these cells are released continously from bonemarrow to help the affected organ.Animal studies with green flurescent dyes with male donors to female recepient animals have shown such transplanted bone marrow stemcells travel ,lodge and start working to repair/regenerate any such damaged organ like heart, brain etc. I was not surprised when one such animal study I came accross confirmed the presence of bone marrow stemcells in even CENTRAL RETINAL ARTERY OCCLUSION IN THE EYE. A PROPER SEARCH OF LITERATURE WILL CONFIRM MANY SUCH STUDIES . so bonemarrow stemcells go to the eye whenever there is a need normally as we are sleeping without us knowing it. This is the beauty of our own regenerative apparatus of which bonemarrow plays a dominant role.Now I don’t doubt the logic of injecting stemcells to the eye from bonemarrow. The best route, dose and other parameters are being worked out by so many well meaning doctors who do not have the means to organise a proper trial with so much funds you know.


    • Hi Dr. Ravi… appreciate your comments. I believe this treatment study has published 3-4 peer-reviewed pieces in international research journals, which haven’t been cited often here in the U.S., but are getting more attention now with Japan’s dominance in the area. They’re legit.


  8. Dr. Ravi – you are confusing cells circulating in the blood stream (which supplies every tissue) with cells being injected into the vitreous humor of the eye (which is what the article is describing).

    Their claim is to treat conditions like age-related macular degeneration with bone marrow stem cells, but these cells are never normally found in the neuroepithelial layers of the retina, even after injury, hence this is a clear case of non-homologous use.

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