ISSCR 2014 Day 2: Navigating Balanced Stem Cell Path to Clinic

I’ve really enjoyed day 2 of ISSCR 2014.

So far this day has already packed in a slew of great talks both in the lecture halls and outside one-on-one with old friends and new ones. How great is that?

I’ll focus on just a few highlights to keep it concise.

Dubious stem cell clinics and the FDA: a match not made in heaven?

The day started off a session entitled “Therapies in the Clinic” and my colleague Leigh Turner kicked things off with a great talk on regulatory oversight of stem cell interventions and dubious clinics.Leigh Turner

Leigh rightly pointed out how dubious stem cell clinics are exploding right here in the US. He used the stem cell clinic network, Cell Surgical Network (CSN), as a case study. For background, you can read my blog interviews with the leaders of CSN here and here as well as my specific concerns about how they are operating here. The leaders of CSN were remarkably frank about their views in these interviews. CSN seems to make the case in a general sense that they are not subject to any FDA regulation, but you can see for yourself based on their comments in the interview pieces.

Leigh pointed out that CSN is a growing network of dozens of clinics operating without any apparent licensing or approval from the FDA, they use devices not specifically approved for production of specific stem cell products for human use, and they offer to treat a host of conditions.  For the first time, Leigh also publicly revealed today that he sent a letter of concern about CSN to the FDA way back 18 months ago. It seems that the FDA has not specifically had any reaction to CSN. Who knows, maybe it never will.

In fact more broadly I don’t see any evidence of FDA action regarding the more than 100 total such clinics throughout the US. What is going on and why no apparent FDA action? I believe the FDA is on course to address the growing issue of dubious stem cell clinics, but is moving far too slowly…kind of like a glacier.

Meanwhile the number of such clinics and as a result from my view the population of patients being put at risk is rapidly rising. My message to the FDA would be that you need to speed up and make clear whether you are (or are not) going to regulate such clinics or it will result in default deregulation. To the members of ISSCR and other stem cell researchers I would say these clinics are not only putting patients at risk, but they are also a serious danger to reputation of the whole stem cell field with the public.

Stem cell translation to the clinic for Type I Diabetes and Parkinson’s Disease.

Then came two great talks on clinical translation of stem cell-related therapies for two major illnesses: Type I Diabetes and Parkinson’s Disease (PD).

James Shapiro of the University of Alberta gave an intriguing talk on the history and future of stem cell treatments for Diabetes. Islet cell transplantation conducted in the past has yielded clinically very significant outcomes in about 15% of patients. It was exciting to hear that more recent adaptations of such transplants are giving about half of treated patients insulin-independence over a period of a few years of follow up. Adverse outcomes only rarely occur and include infection and immune responses. The new protocol includes the key added feature of incorporating alemtuzumab treatment. He also mentioned alternatives to islet transplantation including stem cell-based approaches such as those being done by Viacyte. Very exciting.

Roger Barker of the Cambridge Center for Brain Repair talked about fetal stem cell transplants for PD. One thing that surprised me was the stat he quoted that 1 in 800 people get PD, which means a huge number of people are impacted. Dr. Barker presented a very concise, clear overview of how hard it is to develop a therapy for PD that would be considered a success. Nonetheless his talk gave reason for optimism. Past fetal grafts resulted in some degree of benefits to recipients who had PD, but there was also a risk of dyskinesias. There will be a new trial (called something like EuroTrial?) based on this approach starting in a few weeks for which there is hope. hESC and iPS cells are additional alternative bases for treating PD.

Patient Advocacy

We also heard from patient advocate Jennifer Molson who gave an inspiring talk about her experience with Multiple Sclerosis (MS). Molson had a devastating form of MS that derailed her life at a very young age. A hematopoietic stem cell transplant in 2002 led to a dramatic recovery for her to a point of regaining her life. She is celebrating her 12-year anniversary of treatment. My favorite quote from her talk: “I am living proof that stem cell research can change lives!” Patients such as Molson are the reason why many of us work on stem cells.


Some thoughts on patient contact about stem cells

Lately on average patients or patient relatives contact me about stem cells more than once a week. That’s about 50 times a year.

The patients or their parents have questions about stem cells most often. Sometimes they have comments about this blog. Other times they ask for medical advice from me.

Because I am a Ph.D. scientist  I do not ever give medical advice, but I often have fruitful discussions with patients about stem cell science and the realities of stem cell interventions.

The most frequent kind of contact I have is with parents of autistic children. The second most common contact is made with me by patients who themselves have Multiple Sclerosis (MS). Many other diseases come into play as well in these contacts.Stem Cell Patients Questions List

The parents of autistic children are often desperate for some kind of hope and are exploring stem cell therapies. Often they ask me for recommendations for specific clinics or my opinion on “which type of stem cell is best for autism”.

I do not believe that any stem cell treatments by any stem cell clinics currently offer any realistic hope for treatment of autism. So to be ethical I must tell them that, but I try my best to be understanding and supportive at the same. I also tell them about my concerns about safety. I also tell them I have doubts about the clinical trial ongoing for autism with stem cells. Finally, I tell them there is hope for the future for the use of stem cells, but it is still early days for the field.

The same kind of situation holds true for how I discuss things with MS patients. I just do not believe that IV injections of stem cells realistically do anything that has been proven to be good for MS patients. Here again there are safety concerns. Perhaps at some point stem cells will in some form be shown to safely help MS patients, but that is not true today.

I point them towards my top 10 list of questions that they should ask doctors and clinics selling stem cells. See the list in summary form above that can be printed and taken with you to the stem cell clinic.

In both the case of autism and MS (as well as other diseases) I point out that clinical trials are available, but I also stress that clinical trials have risks too. At least, however, an FDA-approved clinical trial usually means patients will be treated with respect, not charge exorbitant fees, and will receive proper follow up by a trained physician.

I also point out to potential stem cell patients that by traveling to other countries they may increase their risk or the risk to their children because regulatory laws are weaker in most places outside the US. In many countries there is no such thing as malpractice law either and many clinics have no contingency plans for what do if something goes wrong such as if patients become septic or have catastrophic autoimmune reactions.

I have thought long and hard as well as gotten outside expert advice about how best to be supportive of patients without giving medical advice. I believe the approach I described above is the best even if it is imperfect and still evolving.

In the end I hope through these overall educational efforts and my being a respectful listener that I help patients and their families overall.

Guilty plea in ‘evil’ Texas stem cell ‘scheme’

The Houston Chronicle reports a guilty plea from one Vincent Dammai (pictured at right) for his role in criminal behavior using stem cells. The FBI also posted a news story on this itself.Vincent Dammai

Dammai was employed by the Medical University of South Carolina, a position he reportedly used to conduct stem cell isolations and processing without university permission or approval from the FDA.

The stem cells involved were ultimately used for medical interventions in patients.

Reportedly, Dammai’s attorney said “his client mailed stem cells he thought would be used appropriately for research and not for unproven, highly-expensive treatment of terminally ill people.”

According to the Chronicle, victims who ultimately received the stem cells administered by others (not Dammai) included an ALS patient, Mike Martin. It appears a number of patients with ALS and Multiple Sclerosis (MS) may have been treated.

Martin’s sister, Katie, was opposed to Mike getting the treatment, but understandably Mike was looking for hope.

“Mike needed a medical miracle,” Martin said. “They said he’d be able to live and would never even need a wheelchair.”

We know now that was untrue.

Dammi reportedly could be sentenced up to a year in prison and be fined.

Three additional people involved in various ways in this situation (Update: info sourced from press releases & other material by the FDA (scroll to bottom of page), FBI, and Southern District Court of Texas and news articles) apparently will spend time in federal prison: Lawrence Stowe (at left on 60 Minutes), Francisco Morales and Fredda BranyonUpdate: Note that Dammai disputes the links publicly stated by these federal and state law enforcement agencies (as well as newspaper items) between himself and some of the others involved in this case (he has said so in comments on this blog and on a legal defense webpage here).Lawrence Stowe

According to the Chronicle reporting on this case:

The indictment accuses Stowe of marketing, promoting and selling stem cells – along with other drug and biological products that had not been reviewed or approved by the Food and Drug Administration - for the treatment of cancer, ALS, multiple sclerosis and Parkinson’s disease.

At one point, Stowe was a fugitive on the run from the law, but he eventually turned himself in to the feds.

According to a DOJ press release quoting Special Agent Patrick J. Holland:

“These pleas are a victory for the American public, in demonstrating the FDA’s commitment to investigating cases of individuals and businesses that prey on the sick and vulnerable with phony medical treatments,” said Holland. “The FDA will continue to aggressively pursue perpetrators of such acts, and ensure that they are punished to the full extent of the law.”

Katie Martin said of law enforcement handing the situation and those who perpetuated what the Chronicle calls a “scheme”:

“I trust them to make the predators pay for their evil action”

Predators, as Martin calls them, who use stem cell hype to take advantage of vulnerable patients deserve to prosecuted to the full extent of the law in my opinion.