Overplaying N=1 in the stem cell therapy world

The regular emails and phone calls I get from patients asking questions about stem cell therapy are a powerful reminder of the fact that research in our field can be very personal.

Sometimes individuals who receive experimental stem cell therapy (or other cell therapies) and seem to have a positive outcome can end up becoming powerful symbols.

They can embody the potential of specific stem cell approaches and be effective advocates. However, in certain cases these situations get complicated and raise difficult questions.

stem cell therapy, star patient
Stem cell therapy becomes very personal for those getting it and sometimes for scientists too.

Firms using people as symbols of unproven stem cell therapy as successful

Stem cell therapy and the broader area of cell therapy research is not just about testing just some fairly abstract idea. It’s not strictly related to advancing knowledge for knowledge sake. The stakes are high here in a translational and clinical sense. People are hurting and looking for treatment options based on experimental cells for themselves or their loved ones.

Maybe it’s not so surprising then that sometimes in a specific area one particular person becomes the face of the clinical research at least for a time.

What are my concerns here?

I’m mostly thinking about when for-profit firms or their advocates hold up individual trial (or even non-traditional pay-for-play study) participants as evidence that a still experimental approach works.

There’s a lot of gray in this area.

The case of Neuralstem stem cells for ALS

Thinking of research on stem cells for ALS often brings Ted Harada to mind for me. Ted was an effective patient advocate in that space and a wonderful person. I got to know Ted and considered him a friend. Unfortunately, Ted passed away from a brain tumor a few years back. He is really missed.

Many years ago Ted received an experimental intervention from the firm Neuralstem, which became Seneca Biopharma and then part of Palisade Bio. He felt that the cell therapy strongly helped him with his ALS symptoms.

Some folks including in the media and even in politics got carried away when discussing Ted’s situation.

Many other ALS patients probably got the wrong impression from the media.

Where do things stand now?

At present, the Clinicaltrials.gov status for the Neuralstem ALS trial is “unknown” and I’m not aware of any indication that this effort is particularly promising overall. I didn’t find any trial listings for Seneca Biopharma and Palisade Bio only has one listing, which is unrelated.

Unproven stem cells for sale for macular degeneration

Let me give another example. A patient named Doug Oliver received bone marrow stem cells for vision loss due to a rare form of macular degeneration. Doug reported a very positive outcome. He got the cells within a non-traditional, pay-for-play clinical study called the “SCOTS trial” that charges for participation.

Doug has become a stem cell patient advocate as well. Good for him.

People often reach out to me with questions on SCOTS and it’s not unusual for them to cite Doug’s experience as a possible motivating factor for them to participate as well. This is a complicated situation though. The SCOTS study is very controversial. I don’t see concrete evidence that the cells being used are doing most people getting injected any good.

There are also risks and some patients have reported major negative outcomes. People who want to get injected have to pay large amounts of money, often around $20,000.

It’s still not clear to me if the SCOTS trial needs FDA pre-market approval (which it doesn’t have as far as I know) because it uses autologous bone marrow cells. However, in my opinion those cells are likely being used in a non-homologous manner with marrow cells going into the eye. The bone marrow and eye are not related tissues.

Since to my knowledge the FDA has taken no action on SCOTS does that mean that in that particular case non-homologous use doesn’t come into play or unexpectedly this use of cells is considered homologous use?

It’s puzzling.

Results may vary

It makes sense to me that individual patients who get experimental cell therapies and perceive a big benefit may want to spread the word. Their hope is that other people can get help too. I totally get that.

They can become high-profile advocates.

Yet the experience of just one participant in a clinical trial may have little-to-no bearing on how others may do. Many different factors could explain why one person has an atypically positive experience, while others do not.

Genetics. Environment. Age.

Different manifestations of the disease could also be important. For instance, Doug Oliver apparently has a rare form of macular degeneration.

There can also be variability in the cellular preps that each patient receives that could contribute to outlier responses.

The advertising phrase “results may vary” is a common disclaimer and applies in the cell therapy world too.

Money priorities and media

Where I get especially concerned in this area is when firms profiting or seeking to profit off of experimental cell therapies in a sense use a prominent patient for marketing. Sometimes investors jump on this too.

Even politicians.

To me this doesn’t seem appropriate while a trial is still ongoing and the overall results on safety and efficacy still remain unknown. This often occurs with unproven stem cell clinics but can involve biotechs and even universities too.

Part of the draw to do this kind of trial promotion is the potential financial benefit directly from getting more paying customers or such as by raising more money from investors based on an N=1 trial participant story.

Examples of media hype: diabetes & skin

It’s not unusual for the media to hype the story of one trial participant.

The most recent example is when the New York Times raised the possibility of a stem cell therapy diabetes cure on the front page. This story was based on the experience of just one participant in a Vertex clinical experiment. He had only been followed for a relatively short time too. Also, it seems that Vertex’s main approach for their clinical trial will use a somewhat different approach (a capsule delivery of cells) instead of the IV infusion that this patient got. I saw a more recent report that the patient is still doing well now, which is great, but it’s still N=1.

I’ve interacted with biomedical science writers for a very long time. They and their editors love stories of real people who had powerful experiences with experimental offerings. That’s understandable to help readers relate to the story. However, such stories, if not backed up by data from far more than one person, can sometimes end up being very misleading.

There sometimes also seems to be the temptation to sensationalize.

Another recent news headline hyped results from skin cell rejuvenation as though it had helped a woman’s actual skin. It was just about cells in a dish.

Risk of false hope from media or corporate hype

The media problems here most often manifest with over-the-top headlines that hype one patient’s experience as somehow far more meaningful than the reality suggests. In some cases, the body of the story provides more depth and balance but it’s hard to overcome a hypeful headline.

Again, for the one patient involved I can imagine how exciting their experience may be to feel so much better.

However, that doesn’t mean that many others will benefit from a particular experimental stem cell therapy. When there is little solid data to stand on and companies or the media play up such stories too much, it often raises false hope.

Another somewhat different manifesting of the N=1 kind of case is when individual celebrities promote unproven stem cells, often for a large fee or in exchange for free/discounted injections. Some celebrities are even investing in stem cell clinic type firms.

2 thoughts on “Overplaying N=1 in the stem cell therapy world”

  1. I’ve been reading about VSels, the pluripotent type of stem cells that are being used for many different injuries and diseases. I read they also have an effect on the telomeres lengths, and can been done on a repeated basis for DNA longevity. What is your thoughts on these cells.

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