Stem cell therapies: is it all hype?

Stem cell therapies come under attack. #MSBlog #MSResearch

“I read an alarming piece in Science this morning on my way to work. It is about the use of stem cell therapy in heart disease. My heart fluttered as many of the issues raised in the paper are directly relevant to the field of stem cell therapy in MS. Whenever I am asked to comment about the future prospects of stem cell therapies in MS, my heart sinks (excuse the pun) and I am forced to make many of the same points highlighted in this very well-written article. I have extracted a few excerpts from the paper, but I would suggest you read the full article.”

Harvesting bone marrow stem cells

Jennifer Couzin-Frankel. The elusive heart fix. Science. 18 July 2014


…… In April, a maverick British cardiologist rocked his field with an assault on one of its cherished hopes. He suggested that a strategy for rejuvenating a damaged heart with infusions of cells, which has tantalized doctors and patients for more than a decade, does not work……

…..  Francis began digging. He and his colleagues combed through 133 reports on 49 clinical trials of cardiac cell therapy—specifically, a type that uses a mix of a person’s own bone marrow cells. …. All but five trials contained what Francis labeled “discrepancies,” from minor errors in tables to dead patients listed as still taking drugs. And the more discrepancies a trial had, the more effective the therapy appeared to be. His disquieting conclusion: Knowingly or not, some cardiologists were massaging their data to lend them a veneer of efficacy……

…… Published in BMJ, the study is the latest salvo directed at a promising field marked by hope and hype. Since the first heart patients received experimental cell therapy 13 years ago, controversy has come in waves, and it’s now at another peak. Trials built on uncertain science have led to fights about why some can’t be replicated, charges that the animal data supporting trials are too skimpy, accusations of sloppiness, and worse……

…… As cell therapy shifted from mice to ailing humans, uncertainties arose. Doubts were emerging about whether adult stem cells were as versatile as thought. And in 2004, three groups reported independently that they could not replicate Anversa and Orlic’s mouse findings. But by then, “the clinical trials were out of the barn,” says Jonathan Epstein, a cardiologist at the University of Pennsylvania……

…… Cardiologists acknowledge that cell therapy was marked by outsize expectations. “We all hype our work,” says Steven Houser, a cardiac muscle biologist at Temple University in Philadelphia, Pennsylvania. “We want to tell people our work is important. These patients, many of them coming to enroll in these trials, they have no other hope.” And, Houser suggests, in this case “the hype got ahead of the science.” ……

….. “In mouse studies there’s always dramatic improvement,” says Joseph Wu, a cardiologist studying stem cells at Stanford University in Palo Alto, California. “Once you go to a large animal study, it’s moderate improvement, once you go to a phase I trial, it’s decent improvement, and once you go to phase II, phase III, there’s no improvement. This happens again and again and again. … It’s the entire field of biological research.

….. Cell therapists have also been rocked by two recent scandals. In April, a paper published by Anversa in 2012 on the regenerative power of the human heart was retracted, and another was slapped with an “Expression of Concern.” Harvard is investigating the famed cardiologist. Another giant in the field, Strauer, was investigated by the University of Düsseldorf, which announced in February that it is launching disciplinary action. Neither university has said whether the scientists’ early work is in question……

….. For many patients and doctors, however, the key question is practical: Does cell therapy buy time for a person with a failing heart? No clinical trial, for any cell type, has been designed to look specifically at survival, but one is about to: a 3000-person, $8.1 million study now recruiting across Europe. It is funded by the European Union and aims to resolve, finally, the bitter dispute over whether a mix of cells from a patient’s own bone marrow can help. Yet it is far from universally welcomed in the fractious field. The trial, called BAMI, is “unethical,” Arnesen charges, because, he believes, the therapy it’s testing has already been shown not to help…..

….. Even so, BAMI launched with an elation that echoes the earliest trials a decade ago. A 54-year-old man named Neal Grainger, the first British patient treated, told BBC that “it’s fantastic to be part of this.” A London hospital signing up volunteers put out a press release in February highlighting that it is “recognised for its pioneering research into stem cell therapy for heart patients.” The BAMI website notes that “clinical data now exists supporting the concept that autologous bone marrow derived cells can restore cardiac function” after a heart attack. The trial, says the site, could show that the cells “will reduce the mortality rate by 25%.”

….. Left unmentioned: the many studies of the same therapy, in patients with the same illness, that have failed to make a difference…….

About the author

Prof G

Professor of Neurology, Barts & The London. MS & Preventive Neurology thinker, blogger, runner, vegetable gardener, husband, father, cook and wine & food lover.


  • “In mouse studies there's always dramatic improvement"

    Looking at the EAE work published the improvements shown to date have been marginal at best

  • Prof G,

    Sounds like you don't think stem cells will do much for MS repair. Is there anything in the pipeline which shows promise in terms of repair (I don't mean the highly effective DMTs)?

  • The use of stem cells to directly repair cardiac tissue is different than altering the micro-environment of the CNS by way of stem cell secreted factors that can promote endogenous repair. Haven't studies shown that stem cells need not survive to aid in repair mechanisms?

    • Correct in many studies the transplanted stem cell studies are detectable for a few days and therefore presumed dead after that…..So this is all well and dandy that they release factors that cause the existing immature oligodendrocytes to make new myelin, however if you are expecting these cells to turn into new nerves to regrow what is lost then if the cells only last a few days then this route for repair is simply hype.

    • Maybe. We will just have to wait to see the results if it works great if it does not then some people have to eat humble pie

    • The reporting of the paper in the press was accurate. There's no doubt that HIV patients have a much lower incidence of MS than the rest of the population. Whether this is due to the HIV virus itself or the retroviral drugs used to hold it in check we'll have to wait for the trial to see if the hypothesis holds true.

    • These are drug treated patients so the dramatic reduction of T cell numbers seen in full-blown AIDS does not apply here. Hence the study to investigate whether it's the anti-retrovirals that are responsible for the reduction in MS rates in HIV positive subjects.

    • A normal CD4 count is 500–1,000 cells/mm3, AIDS therapy are effective when the CD4 count falls below 350 and it appears that the therapies restore CD4 counts to the lower end of normal.

      It just may be that AIDS patient on therapy have a lower CD4 count than the normal population which would obviously lead to a reduction of MS.

    • In the anti-CD4 clinical trial CD4 T cells were depleted to less than 300/ml This depletion, however, did not result in a significant decrease in the number of active lesions on monthly gadolinium-enhanced MRI over 9 months.

    • Which trial was that? I think it is clear that all therapies that deplete or sequester t-cells such as stemp cell therapy, as well as all of the other immunosuppresive theapies (altiluzumab, natiluzumab, fingolomod, tecfedera, etc, etc.,etc……) have a profound effect on the disease.

      Time to start paying attention to what is going on.

  • Two references given to original paper reach a paywall. Could you please note this when you give such a reference – so that the indigent among us don't continue to spin our wheels.

  • Re London hospital signing up volunteers put out a press release in February highlighting that it is “recognised for its pioneering research into stem cell therapy for heart patients.”

    The hospital is "The London Chest Hospital', part of 'Barts Health'

    The press release is HERE on the website

By Prof G



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