Huge clinical trial patient fees allowed by FDA at times, details often secret

stem cell monopolyI am often critical of for-profit stem cell clinics on this blog for numerous reasons. For instance, one thing that concerns me greatly about these clinics is that they charge patients to get experimental “treatments” that have not been proven to be safe or effective.

But as some patients have pointed out to me over the years, certain FDA-approved stem cell clinical trials also charge patients to receive unproven stem cell therapies.

Should someone (even if that someone is an academic clinical researcher) be able to charge a patient a large access fee to be in a clinical trial in which that patient will be subject to an experimental therapy that could ultimately be proven unsafe and ineffective?

I have serious doubts about this.stem cell monopoly quote

You might be surprised to learn that the FDA in some instances allows patients to be charged to be in trials including stem cell trials and others. Those running the trials can request permission from the FDA to charge patients. The agency then has to evaluate the request. The FDA has a draft guidance on this, but frankly it’s difficult to learn that much from that document in terms of details in how this plays out.

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Predatory stem cell clinics are winning the war; what can we do?

For years I have been working to educate the community about the predatory stem cell clinics out there. These clinics prey on vulnerable patients and their families. The clinics use hope as a marketing tool.

A weapon.

As the number of such clinics has mushroomed in the US and elsewhere the risk to both patients and to the larger stem cell community proportionately rises too. We are in a situation today where the dangers from such clinics have never been higher.Stem cell facelift comic

They are making millions in profits with little-to-no accountability. Their therapies don’t have to work or even be proven safe. It’s odd because many consumers seem to expect more from non-health-related businesses such as McDonald’s or computer companies than from stem cell clinics.

Thousands of patients just in the US alone are regularly being subjected to experimental, non-FDA approved interventions. They are spending millions of dollars and being put at substantial risk.

Many patients are desperately looking for hope so they are very driven to find something that may help and are often willing to take unknown risks.

Some of us in the stem cell community are working to try to make some positive impact in this area. I believe we are making a difference, but overall the dubious stem cell clinics are winning the war.

Why are the clinics prevailing so far?

In part it is because they’ve been very smart about how they do business.

For instance, they do PR like pros, they manipulate some members of the media to almost in essence work as their spokespeople, they use social media to great effect, and they’ve won over some powerful allies in the form of certain patient advocates who have become in effect stem cell clinic advocates.

An illustration of the cleverness of the clinics is their move to take advantage of Clinicaltrials.gov to list their non-traditional, profit-driven business as if it were real clinical trials. They even go so far as to say that just because their work is listed in that database that their offerings are FDA-approved. They aren’t.

The stem cell clinics are also winning because the FDA has been so passive and ineffective, particularly during the last two years. Further in the past the FDA and more specifically the CBER division within the FDA that is tasked with dealing with stem cells took steps to regulate the stem cell clinic industry through actions such as warning letters. In contrast, lately CBER hasn’t done anything (at least apparent in the public domain and via FOIAs I’ve submitted) on the stem cell clinic problem.

This apparent regulatory passivity couldn’t come at a worse time either as the stem cell clinics proliferate like crazy in the US. There’s certainly a connection there. Less regulatory action = more dangerous clinics. It’s frustrating because CBER of course remains very active with the good citizens of the stem cell world such as those in academia and legit biotechs with appropriately high expectations for them.

Hello, CBER, are you home? Are you paying attention? Patients need to be protected.

I’ve tried talking with the FDA to get at the root of the stem cell clinic problem, but things remain nebulous. Are they afraid of being sued? Just too slow? Don’t have the budget? Maybe part of the problem is the leadership transition at the FDA where there hasn’t been a commissioner…but I think that’s only part of the story.

The FDA took a healthy step last year in issuing draft guidance (see my interview here with the FDA on the draft guidance) to regulate fat stem cell products that are almost certainly biological drugs requiring approval, but that’s been about it and those draft guidances have not been finalized. Until finalized, the draft guidances have no teeth. Meanwhile the fat stem cell clinics and others that sell unapproved stem cell biologics of various kinds such as amniotics, take advantage of this gray area to milk patients for millions of dollars all the while putting such patients at risk. The clinics are literally laughing at the FDA all the way to the bank.

Why should you care about this as a stem cell researcher, patient advocate, or other interested party? As has happened in the past, people are going to get hurt or killed at these clinics, and not only is that a tragedy unto itself, but also it will reflect badly on the whole stem cell arena. This magnifies the negative impact.

The unchecked stem cell clinic industry also has other negative effects such as muddying the waters for patients over just what is (and what isn’t) a legit stem cell therapy and research. We are also seeing some at academic institutions starting to give in to temptation and work with the dubious clinics too probably for the big bucks involved. In short, the war isn’t going well and the risks are growing.

So what do to?

We need to push the FDA to act more consistently, quickly, and forcefully on this problem. Maybe they think they are acting on it, but from my view it seems to be in slow motion. A recent poll on my blog indicates a larger sense within the stem cell community that the FDA isn’t being effective on stem cell clinics. And by my own calculations, the number of stem cell clinics in the US alone is skyrocketing. The FDA doesn’t have much time.

If more patients are injured or even die after getting questionable stem cell therapies, in a sense the FDA will bear part of the blame because of their ineffectiveness.

We also need organizations to step up to the plate and confront the clinics as well. When individuals such as myself and others including Leigh Turner and Doug Sipp do this, we have had some positive impact, but at great risk to ourselves. It is literally dangerous for us. I have been threatened with litigation and literally threatened to be attacked or killed.

Educational efforts can also be helpful and that is a major mission of this blog.

In the absence of timely FDA action, an out of control stem cell free market is churning. In January of this year I called it a “wild west” of medicine. It really feels that way. There’s demand so there will be supply. Something fundamental needs to change or the war is over and patients lose out, as does the stem cell field.

Stem Cell Person of the Year 2012: Roman Reed

Roman Reed Stem Cell Person of the YearThe Knoepfler Lab 2012 Stem Cell Person of the Year is Roman Reed.

Congratulations, Roman!

Roman made a tremendous difference in 2012 in many ways.

One of the most notable was catalyzing the TJ Atchison Spinal Cord Injury Research Act in Alabama, which provides $400,000/year in funding for research. Of course, TJ and many others who helped make this possible also deserve great credit and have my admiration, but Roman provided key leadership. Here in California, Roman’s Law supported its 11th year of grants all eligible for all forms of stem cell research. Roman informs me that it funded $749,00 overall and approximately $200,000 in stem cell funding.

In addition, Roman in 2012 mentored and energized a whole new generation of advocacy from young, energetic leaders: TJ Atchison, Katie Sharify, Richard Lajara and Tory Minus.

I would also say Roman has been an inspiration to me too and many others. One of Roman’s mottos is “A rising tide lifts all” and he doesn’t just say that, he lives it. He lifts up many others.

Here is a link to Roman’s foundation. Go take a look!

Roman also received the most votes in the online voting (you can see the final poll results here). More than 4,500 votes were cast in total. Only 6% behind Roman was the amazing activist Ted Harada followed by Roman’s dad the remarkable Don Reed, the wonderful Judy Roberson, and the super Katie Sharify nearly all tied for third. Next after them was the relative new kid in stem cell town, Leigh Turner (more on him below).

Roman is still moving mountains already in 2013. AIMing for some amazing developments in Alabama.

I made my own life very difficult with a stellar panel of 16 finalists for this award, but Roman stood out this year, something that the voters in the on-line poll and many of the other finalists also recognized as I asked them for their favorites amongst each other.

The runner-up for the 2012 award is Dr. Leigh Turner of the University of Minnesota. Leigh took the courageous, outside-the-box step in 2012 of contacting the FDA to investigate Celltex when he perceived patients could be at risk. As “thanks” for his action, he was put under enormous pressure and there was talk of possible litigation against him. Pressure was applied to his employer, the University of Minnesota. We’ll never know for sure, but from everything that I know I believe that Leigh’s actions directly led to prompt FDA action, which otherwise might not have happened at all or until much later. In my opinion, Leigh’s act of courage, helped make hundreds of patients safer in a direct way and indirectly may have set a higher standard for the field of stem cell treatments. It may also inspire other folks to contact the FDA if they perceive specific entities are putting patients at risk. I’d also like to note Doug Sipp here, who sometimes works together with Leigh, for his courageous efforts to educate the community on unlicensed stem cell operations as well, often “thanked” with threatened litigation too.

In a way I’m an additional winner from this contest because I got to know even more amazing people in the stem cell field as well. Their accomplishments inspire and motivate me even more! I also hope that by being nominated and chosen as finalists that this process was a win for everyone involved.

Overall, it was a tough thing to do to judge this thing as you might imagine. I’m betting that the 15 finalists who didn’t win for 2012 are going to be top candidates for the award in 2013, but I hope we have more newcomers as well. I’d encourage people in industry and in research labs to get more involved too in this award process.

Yes, I’m doing this again this year and the prize will be $1000 again for the winner for 2013. I’m going to do it a bit earlier, starting with calling for nominations in just 10 months from now. Time flies!

Keep up the good work! 2013 is going to be a banner year for stem cells.

Knoepfler Blog Stem Cell Person of the Year Finalists: time to vote

I’m giving out a Stem Cell Person of the Year award for 2012 along with all my other annual awards (e.g. best stem cell journal, paper, blog, comic, activist, etc).

The Person of the Year winner will get recognition for being a difference maker, someone who takes a risk to help others.   

The winner also gets $1000 in cash from me.  

About 30 people have been nominated, consisting of a truly amazing group of people. Many of them were nominated independently more than once for this award. I originally said I was going to list the top 5 finalists, but I decided to list the top 16 (~half of total nominees) since they are all so awesome. Scroll down below the poll to see information on each nominee. 

Ultimately I will choose the winner, but your votes (see poll below) could have important influence, especially in the case of a tie-breaker situation where I might have trouble picking between a few outstanding candidates. Given the stellar group of nominees the likelihood of this happening is not so small.

So please vote and tell your supporters to vote.

The voting deadline is 11:59PM December 31.

Listed below in alphabetical order by first name (I’m a very fortunate person to know most of these folks personally) are the finalists along with my short introduction of them to you including paraphrasing or actual quotes from nominators:

  • Alexey Bersenev. Alexey is a self-described “Cell trialist”, who amongst other things runs the fabulous stem cell assays blog. Alexey makes a difference through at least in part amazing educational outreach. Nominator: “He is arguably the most educated person I know about cell therapies from essentially every angle and is very helpful in educating others”. He calls it like he sees it.
  • Bernard Siegel. Bernie is the head of GPI and Stem Cell Action. He is the organizer of the annual World Stem Cell Summit. He is the tireless engine that drives stem cell advocacy and accountability. It’s hard to imagine the stem cell community today without Bernie’s contributions over the years including this year.
  • Don Reed. Don is a long-time stem cell advocate (blog here) who has inspired and empowered a whole generation of stem cell advocates. His nominator said of Don “He never ever stops working for stem cell research and advocacy.”  Here is an interview published with Don, who played a key role in Prop. 71 and the creation of CIRM. On Twitter, Don describes himself thusly: “sponsored California’s Roman Reed Spinal Cord Injury Research Act, board of directors for Prop 71, Vice President for the Americans for Cures Foundation.”
  • Doug Sipp. Doug is someone whose actions make a huge difference in protecting patients and promoting accountability in the stem cell field. Nominator says: “More than anyone else, I think, he has taken fire for so many others in the stem cell advocacy arena, and yet doesn’t make a big deal out of it.” Doug’s work includes, but goes well beyond his blog.
  • Jeanne Loring. Jeanne is both a great stem cell researcher (see her lab page here) and also an educator. She is the Founding Director of the Center for Regenerative Medicine at The Scripps Research Institute in La Jolla. For years Jeanne has made herself available as an advocate, going beyond the lab to make an impact in multiple ways. Nominator: “Jeanne is an exceptional scientist who takes that step outside the lab to engage the wider community in powerful ways.”
  • Judy Roberson. Judy is a Huntington’s Disease advocate who, as described by nominator, “has positive spirit and energy beyond anyone I know.” She is the President of the Northern California Chapter of the Huntington’s Disease Society of America. She makes amazing things happen and I believe has contributed in tremendous ways to bringing transformative funding to research into Huntington’s Disease. Here is a radio interview with Judy that tells you so much about her efforts.
  • Katie Sharify. Katie is a hero and inspiration to so many. She is a participant in the Geron hESC-based clinical trial for spinal cord injury and a patient advocate. Katie is that rare person who has the courage to tell her story and inspire others. One way she does this is through YouTube Videos (e.g. here and an interview with CIRM here) . Nominator: “She really connects with people in a special way that makes a difference.”
  • Keri Kimler. Keri is a long-time stem cell advocate. She is a passionate worker for advancing stem cell research. Keri is the founder of Texas Cures Education Foundation. She is currently the Director of Constituent Relations at Texas Heart Institute and is former Vice Chair of Texans For Stem Cell Research . Keri also runs a great website on stem cells. Nominator says “Keri is someone who, in sometimes hostile territory, makes practical, positive things happen. She talks the talk, but also walks the walk.”
  • Lee Buckler . Lee describes himself on Twitter as “Blogger, analyst, consultant, and entrepreneur in the cell therapy & cell-based regenerative medicine industry.” Nominator: “Talk about your Renaissance Man, huh? Lee should include on there ‘educator’ because he is mindbogglingly informed about every aspect of the stem cell/cellular medicine industry.” He’s also quietly (although less quietly nowadays) working to help educate others including through his blog.
  • Leigh Turner . Leigh (pronounced “Lee”) is an associate professor at the University of Minnesota in their ethics center. On Twitter he describes himself as “Prof of Bioethics & Public Health; Interests include bioethics, global health, medical tourism, organ trafficking, health fraud, patient safety”. Nominator: “In the midst of an intimidating environment, Leigh took the revolutionary step of being a scientist asking the FDA publicly to look into a company that he felt was putting patients at risk.”
  • Mary Schneider. Mary is a long-time stem cell advocate who interfaces with politicians at the very top to make a difference. She has worked very hard specifically as a patient advocate for children to promote safe and effective therapies for children. I asked Mary about this and she wrote that her goal is: “that other children may some day have the gift of good health and opportunities that my son was blessed with.  I knew from the very beginning of the journey to help my child that the other children who were afflicted were equally as important and no matter what the outcome was for my child, I would never stop advocating to help the others.  I’ve kept my promise and the hard work is paying off in ways that I could never have imagined.” You can learn more about Mary’s great work here.
  • Roman Reed. Roman is a long-time spinal cord injury (SCI) research and stem cell research advocate (see his foundation here). Roman goes beyond educating others to make tangbile things happen. The SCI research act named after him in California funded groundbreaking research into SCI that was crucial for laying the foundation for Geron’s work. This year, through a lot of hard work as part of a stellar team, he helped get the TJ Atchison SCI Research Act passed in Alabama. Nominator says: “Working to make that a reality in a ‘red’ state is a great achievement.”
  • Sabrina Cohen
. Sabrina is a long-time stem cell and SCI advocate. She is the founder of the Sabrina Cohen Foundation, which does amazing things such as raising awareness through lecture series and funding of SCI research. Sabrina is a high-profile advocate who extends our community’s messages to an incredibly important, larger audience. Nominator: ” she’s given around 20,000 motivational talks, mentored hundreds of the newly disabled, and raised hundreds of thousands of dollars for health-related cause, which includes $75,000 in medical grants directly to the nation’s s leading stem cell researchers.”
  • Stephen Sullivan.  Stephen is the founder and Director of the Irish Stem Cell Foundation. He is one of the few scientists who is also an advocate, which is very admirable. Stephen is another one of those people who works tirelessly to make the stem cell arena a better place and safer for patients, but without claiming a lot of media attention for himself. No self-promotion there. Nominator: “He works in a hostile environment with opponent groups going after scientists and stem cell advocates personally and by name, but maintains a positive outlook.”
  • TJ Atchison. Nominator: “TJ is an inspiration and hero”. He suffered a SCI and became the first ever hESC-based clinical trial participant. He worked to make The TJ Atchison Spinal Cord Injury Research Program a reality via the TJ Atchison SCI Research Act. Nominator: “The impact of this new effort will be tremendous”.
  • Ted Harada. Ted was one of the patients in NeuralStem ALS trial and had a striking response. He describes himself on Twitter as follows: “Husband, Father of 3, ALS Patient & Advocate, politics & sports junkie and (hopefully) Catholic Gentlemen”. Nominator: “He became a vocal advocate for stem cell trials in ALS community.” You can read more about Ted here on CNN Health.

Some important notes. I note that CIRM as well as Drs. Ellen G. Feigal and Patricia Olson of CIRM were nominated and I believe are deserving of tremendous recognition, but I do not list them in my dozen finalists because as a CIRM grantee myself I feel there would be a conflict of interest for me. But they are wonderful!

President Obama was also nominated, but I frankly think he hasn’t done quite enough on the stem cell front yet. I do have hopes for his second term.