Remember that FDA public meeting on stem cells? Here’s the video

The FDA held a historic public meeting on stem cells a few months ago. Many people have been eagerly awaiting the video archive.

Here it is!

My memory from watching most of it live was that there was quite a bit of really interesting material at the meeting from the diverse presentations.

Stem cell wish list for 2017

Last week I posted my list of 2017 predictions for the stem cell field. Today a couple of days into 2017 I’m more focused on hope than realism. What would I wish for in the stem cell and regenerative medicine arena in the coming year?stem-cell-wish-list

More stem cell clinical trial data posted and published. There are few things as exciting as stem cell and regenerative medicine clinical trials across the full spectrum of stem cell types including adult, embryonic, and IPSC. But we need to have actual trial data be peer reviewed and published or at least posted. Clinical trial updates only by press release are not helpful to patients or the field.

Clinicaltrials.gov adapts. This vital resource of trial listings adjusts to new realities. It either filters its listings to screen out for-profit entries that aren’t real trials or it provides more practically useful information such as at a minimum clear indications of whether a listing has an IND (this doesn’t need to violate any confidentiality rules) and whether the listing requires payment as an inclusion criteria. See my interview with the leader of Clinicaltrials.gov.

FDA speaks clearly. Whatever the FDA does or does not do in terms of actual stem cell & regenerative medicine-related actions in 2017, it is clear about it. This year I hope the FDA provides concrete, consistent explanations in the public domain that don’t require an FDA-ese jargon dictionary to try to understand.

FDA and its CBER center are consistent with good & bad citizens of the stem cell arena. The FDA has a tough job overall and its CBER branch focusing on biologics including stem cells has its own specific challenging task set. However, for years CBER has held different players in the stem cell arena to different rules and expectations. Paradoxically, essentially the better a citizen you are, the more the FDA expects from you. On the flip side, if you are for instance a stem cell clinic with no intention of following the rules (no BLA, no IND, no pre-IND, no expertise in stem cells, no data, etc.) CBER has historically generally left you alone.

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Top 20 Stem Cell Predictions for 2017

stem cell crystal ball

Stem cell crystal ball

Each year I make a list of predictions for the stem cell and regenerative medicine field for the coming new year. Later in this post I list my top 20 stem cell predictions for 2017. In looking at my past predictions I realized this will now be my 7th year doing stem cell/regenerative medicine yearly predictions.

You can see below links to these predictions for past years, which sometimes seems rather far removed from today and in other cases strike me as strangely apropos of our times.

What will 2017 bring? Below are my top 20 predictions in no particular order except starting with a few hopeful visions for the coming year.

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Open letter to FDA: take action now on stem cell clinics before Trump

Dear FDA,

The stem cell clinic clock is ticking on you.

Before the Trump administration rolls in to possibly tie your hands on many important areas of oversight including stem cell clinics, you should take bold action now.

Your CBER branch has been preternaturally quiet on taking actual regulatory actions on stem cell clinics for several years now even though there about 600 such clinics in the U.S. operating without any FDA approvals putting thousands of patients and the stem cell field at risk. Everyone knows that you are now aware of these clinics. One warning letter in a period of years is a drop in the bucket.

For these same past few years you have issued draft guidances that if implemented would substantially change how you regulate stem cells in ways that would quite helpfully put a stop to the mushrooming stem cell clinic industry.

You also held two public meetings on stem cells in 2016, which was historic, and you received both verbal and written comments from stakeholders. The REGROW Act is history and the Cures Act is now law, with important language reinforcing your role in stem cell oversight. The clinics do not by any stretch of the imagination meet the hurdles specified in Cures.

In short, much uncertainty is over. And you have done your due diligence. The time is right to tackle the dangerous stem cell clinic problem. You have sent clear signals that you feel strongly about proper stem cell oversight including via a late November opinion piece in the NEJM only a few weeks ago. But words are not enough.

Issue a large coordinated series of warning letters in the next couple weeks to the scores of clinics grossly violating your regulations by experimenting on thousands of patients for profit with unapproved drugs. Now is the time.

After Trump’s inauguration all bets are off as to whether the new administration’s FDA could still do anything about this serious problem. Strike now before his inauguration and make a profound positive difference.

Best regards,

Paul Knoepfler

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.