Stem cell tourism – from anecdotal and speculative cases to observational studies.

by Alexey Bersenev on August 30, 2010 · 0 comments

in clinical trials and cases

 
Stem cell tourism is here. It exists and, apparently, it’s a good business. But does it really help patients? What are the results and long-term outcomes? Professionals (physicians and scientists) are staying skeptical and referring to lack of any reported data about “stem cell treatments” abroad. There are stories of many cases you can read online, but almost all of them are advertised on clinic web-sites, “told by patients” and positive. Sorry, but I don’t believe in this magic, I don’t buy it. Absence of any “no effect” cases or “unsuccessful treatments”, actually makes me cautious and even scared. I think that such innovative intervention as cell therapy can not avoid negative results or complications. What we really need right now is that observational studies. What is it? These studies do the following:

    1. evaluation of the patients who have returned from “stem cell tourism” trip by physicians;
    2. collection of data about all patients who went through such treatments;
    3. unbiased analysis (not by clinic-providers and not subjectively by patients);
    4. evaluation and analysis of published cases and phases I-II of clinical trials;
    5. publish in peer-reviewed professional scientific and medical journals.

It is needless to say how badly we need this kind of studies in cell therapy. Until observational studies and conclusive clinical trials, professionals will consider all magical cases of cure as anecdotal and speculation. Well, the good news is that we have got some observational studies right now. I’ll quote one of them below.

The study, evaluating cellular therapies for amyotrophic lateral sclerosis (ALS), is published by our Spanish colleagues in September’s issue of Cytotherapy journal. This is the best observational study I have ever read! Strikingly, unlike advertisements online, data is negative!

The increasing availability of stem cells in many countries, together with straightforward laws of supply and demand, makes it imperative that clinical trials demonstrating the effectiveness of these treatments be undertaken, before ‘stem-cell tourism’ becomes more widespread and ends all opportunities for this promising new treatment. Therefore we present the results from a series of 12 patients who decided to seek cell therapy of their own accord in the clinics mentioned above.

They evaluated three groups of patients:

    1. Three patients treated with fetal olfactory ensheathing cells by intracerebral injection in China by published methodology;
    2. Two patients received mesenchymal stromal cells by intrathecal and intravenous routes in Greece;
    3. Seven patients received intrathecal enriched CD34+ cells in Germany.

None of the three centers providing treatment maintained a specific follow-up protocol after TX. No information about the anticipated result of the treatment was offered in writing at any of the centers except for the one in Israel. We have no information regarding the expectations of success given verbally to the patients by the medical teams.

The authors evaluated patients by commonly used in neurology functional scales – ALS functional rating scale (ALSFRS-R) and forced vital capacity (FVC). Data were compared with scores, reported for natural progression of ALS. The study showed no difference in the progression of the disease and was no changes observed in comparison with pre-treatment status. Some adverse effects were noticed:

There were two adverse effects reported during the TX procedures. Patient 1, who received intracerebral ensheathing olfactory cells, presented with fever and deterioration in consciousness, lasting 5 days, 48 h after surgery. One of the patients that received intrathecal MSC presented with intense back pain 4 h after the lumbar puncture, which required morphine derivatives for 72 h. No late complications attributable to the TX were observed during the follow-up period.

Importantly, authors retrospectively analysed all available data about current ALS clinical trials. 550 patients underwent cell therapeutic treatments and outcome was reported. All possible cell types were injected (hematopoietic, mesenchymal stromal cell, total mononuclear, fetal neural), mostly autologous. None of these trials and cases were found positive. Authors conclude that current cell therapy can not cure or even offer better life for patients with ALS.

I like the last 2 columns of their trials summary table. First one showed that outcome judged by subjective impression of the authors is mostly positive (6 out of 8 reports). Second one showed the opposite – objective evaluation in all reports is negative.

Regarding secondary effects, one of the patients who underwent treatment with olfactory ensheathing cells presented with serious central nervous system (CNS) complications. Three cases of meningitis were identified in an observational study of seven patients with chronic spinal cord injuries treated at the same center. Of the nine patients receiving intrathecal stem cells, one presented with back pain lasting 3 days that required morphine, and two complained of sensory symptoms in their legs. A similar prevalence and intensity of adverse events were found in the English- and Chinese-language literature. Of the groups using intracerebral injection, 4.9% of patients in a Chinese group experienced complications including headache, short-term fever, seizures, CNS infection, pneumonia, respiratory failure, urinary tract infection, heart failure and possible pulmonary embolism; 25% of this group experiencing complications died.

As you can guess, conclusions are the following:

In view of these negative results for cell therapy in humans, further insufficiently controlled clinical trials should be avoided. Furthermore, these treatments are often offered by ‘for profit’ clinics, which raises ethical implications. ALS experts could prevent the worrying new type of medical ‘tourism’ arising from patients’ desperate searches for effective treatment. The ever-increasing range of fraudulent procedures provides therapies that are not proven and that may cause serious psychologic and economic distress to the patients concerned, and endanger the legitimate progress made by scientists in the field of stem cells. In conclusion, the results obtained to date in these clinical trials suggest that cell therapy in ALS has not been shown to be sufficiently effective in curing or halting the disease, and its presumed ability to slow down the disease’s progression has yet to be proven.

I love this study! Very well done and unbiased. Extremely important for community! Based on this data we can design better trials and avoid possible complications. We can answer to patients seeking treatments, if they ask for advice.

Of course, it is very sad news for desperate ALS patients, but it’s how it is. Cell therapy is not a magic. Stem cells are not a panacea.

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PS: I’m ready to send this paper out to everyone who is interested. Contact me.

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