November 27, 2020

The Niche

Knoepfler lab stem cell blog

More Duke autism program backstory: stem cell ‘magic’ & money

Last week I wrote a long post detailing the troubling multimillion-dollar reverberations between the Duke autism program and a for-profit stem cell clinic in Panama called The Stem Cell Institute. The potential entanglements between the Duke group and the Panama clinic raise a bunch of questions and concerns. They center on questionable, unproven autism “treatments”. In Duke’s case, their own clinical trial showed there was no benefit, but they forging ahead anyway.

Muddy waters

This Duke-stem cell clinic situation is a prime example of muddy waters between a place overall doing robust clinical science and for-profit clinics. In terms of the actual biomedical science in this case, it seems to me like this is likely the realm of stem cell “magic” where one’s hopes and ideas prevail over actual data.

I wanted to give you as readers more background on what led up to that post and what has followed it, including more information and concerns.

In addition, stem cell policy and ethics researchers Jeremy Snyder and Leigh Turner coincidentally just recently published a crowdfunding analysis paper that highlights the resonance between Duke and The Stem Cell Institute so I want to briefly discuss that as well.

Duke Curemark autism study pamphlet
Duke Curemark autism study pamphlet.

Duke-clinic story background

It’s difficult in some ways for me to do a post like last week’s one. I’ve only done a few such posts in a decade of blogging here on The Niche, including about Northwestern’s now-defunct autoimmune stem cell program. Who wants to take the risk to be critical of the practices of a team led by a fellow academic stem cell researcher? In the case of the Northwestern program, the leader Dr. Richard Burt pushed back with a piece in Science. Also, Dr. Kutzberg has done important, pioneering work on blood stem cells for decades, mostly unrelated to autism. Her overall research track record is fantastic.

Given all of that, why do a post like last week’s?

The well-being of pediatric patients and their families is the primary reason I decided to pull the trigger on this particular post.

I’m also very concerned more broadly about the practice of some universities requiring large fees for patients to receive unproven cellular therapy offerings, often in ways such as via off-study or compassionate use where the data from the patients often does not end up published. These arrangements may have less chance for benefit and more risks than are portrayed.

Some universities are at the same time hyping or exaggerating on-going clinical trials as though the cell therapy drug in question is already known to work.

What happened next

After doing my post last week on Duke, I heard from a wide variety of people in the stem cell and autism research field with generally positive feedback and the need for more transparency on this situation. Many were puzzled as to why Duke would be in such a situation.

Fairly quickly after I put my post up, David Gorski over at Respectful Insolence wrote a scathing piece about this same Duke – stem cell clinic connection. There’s more info there in his piece so it’s a worthwhile read. We both agree that many children are being put at risk. Gorski also doesn’t pull any punches. For example, he used the term “quackademic”.

Snyder – Turner Paper

The relevant, new Snyder-Turner paper is entitled, Crowdfunding, stem cell interventions and autism spectrum disorder: comparing campaigns related to an international “stem cell clinic” and US academic medical center.

The paper, which came out just days after my blog post, provided some additional helpful insights. The clinic in the title of their paper is the same Panama The Stem Cell Institute and the academic medical center is indeed Duke.

There seems to a large, ever-changing constellation of families dealing with autism centered around both the Duke and the Panama clinic, with fundraising and other connections. To participate in at least some of what’s offered, you apparently have to shell out a lot of money.

Even if the Duke autism team is not intentionally promoting the Panama Clinic, I believe that how Duke is handling all of this does help the clinic, and as I wrote in my previous post, it seems to me that the clinic is helping the Duke autism team too in some ways. There’s some concerning symbiosis there in my view.

Another odd autism trial involving Duke

A colleague also earlier tipped me off to a different Duke autism trial listing that is in my view another red flag. The trial, called “blüm: The Study of a Biological Autism Medication”, has a questionable rationale in my view, perhaps even weaker than the cord blood work they are doing. Duke is one of several sites for this study. It is sponsored by Curemark, LLC.

The Duke page for the study says:

“Investigators at the Duke Center for Autism and Brain Development are studying an investigational drug in children ages 3-8 with autism.  The investigational drug, CM-AT is an enzyme that is sprinkled over food three times a day that aims to improve digestion of protein and reduce harmful gut/brain interactions and therefore may improve symptoms associated with autism.”

In essence, as best as I can tell, the study sprinkles some enzyme digestive supplement on the food that the autistic children eat and hope for the best.

In rare cases, some children with an enzyme deficiency can develop autism, but it seems this trial wasn’t focused on kids who have the deficiency.

I’m not kidding. Check it out for yourself.

Sure, there are links between the GI and the brain including in the context of specific health conditions. There also could be a meaningful link between digestion, metabolism and some unique cases of autism like in kids with the enzyme deficiency. Even if there is, why would this supplement help kids with no enzyme deficiency? This seems like another uber long-shot clinical trial. Note, that although this trial is still listed on the Duke autism website, it might already be completed.

The favorite stem cell trap

It is surprisingly easy to fall into the trap of viewing your favorite particular kind of stem cells as some kind of medical magic. I actively try to avoid this with one of my favorite types of stem cells, IPS cells. Over the years I’ve posted many times about limitations and even setbacks to clinical IPS cell research along with the milestones and stepwise successes.

Stem cells in a general sense are remarkable cells with unique qualities, but that doesn’t mean that any particular kind of stem cells can treat or cure a wide array of diseases.

Take cord blood. The stem cells in there have proven power for a narrow range of diseases related to bone marrow transplant kind of applications, but if you stray away from that very specific area, you find statements even by a few academic researchers that suggest cord blood cells can do almost anything including helping autism, COVID-19, and just about any other health condition you can think of at the moment. I believe cord blood is likely to have newer applications as well that are going to be proven and have big impact, but I also believe if you’re going to do any particular clinical trial using cord blood, and especially on kids, your rationale better make good sense and be based on excellent data.

The same kind of thing goes for MSCs. They are probably going to have some specific, proven safe and effective applications in coming years, but they aren’t a panacea. MSCs from fat in particular have been injected into just about every body part and for dozens of conditions, often where it just makes no sense such as into the eye. The results have in some cases been tragic and have more often than not given patients a huge hit to their finances.

Looking ahead

In the end, it should always come back to the strength of the data.

What do we really know about a specific kind of stem cell in terms of risks and its potential use for a specific condition? Often, next to nothing, even if it is already being injected into people, sometimes with a big price tag. Where there are data, they are often confusing or point in different directions.

In the Duke autism case, again the best data out there (much of it their own) even says there is no benefit. So just do another similar trial with somewhat different design or cells? Then another and another?

Is this really helping any children or families?