Careful with the heart strings: Promise vs. potential

Author: David Kent, 06/26/14

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The cellular therapy plenary at the ISSCR conference on June 19 was an incredibly interesting mix of hope and despair. On one hand, the opening talk by Leigh Turner made us imagine thousands of people in the United States getting duped into stem cell therapies as a result of pre-mature commercialization and ethically contentious marketing practices. On the other hand, talks by Luigi Naldini and Roger Barker catalogued excellent progress in clinical trials on rare blood disorders and Parkinson’s disease – I’ve written previously on the Naldini group’s work. Overall, it was really encouraging to see well-controlled, well-monitored trials taking place and being presented at scientific meetings – things have come a long way in the last decade.

But the highlight talk, easily generating the most post-session chatter, was from Jennifer Molson, a patient advocate who was involved in a Canadian-based Multiple Sclerosis (MS) stem cell trial. In 1996, Ms. Molson was diagnosed with MS at the age of 21 – she was a healthy, happy and physically active young woman working toward her career goal of becoming an RCMP officer when she received the news about her crippling degenerative condition.

Jennifer’s story was nicely summarized in the news several years ago but it had flown under the radar for many in the crowd of 3,000 stem cell biologists. Her powerful tale ensued as she charted the destruction this condition inflicted on her life – numerous relapse/remission episodes, various therapies, lots of side effects, and a devastating blow to family life. Her dreams of an RCMP job were eviscerated; she managed to get an office job, but in September 2000, new symptoms arose which forced her to quit her job the following February. There were new lesions in the brain stem, options and hope were fading fast and she entered the MS stem cell transplant trial run by Harry Atkins and Mark Freedman.

The results were incredible – Jennifer was articulate, mobile, and charming. The process was lengthy, uncertain, and full of ups and downs, but this stem cell treatment clearly gave Jennifer a new lease on life. She detailed her new ability to participate in the lives of her family members and could now undertake activities like skiing, hockey, yoga, driving, and a return to full-time work. She is living proof that stem cell research can change lives. As session chair Irving Weissman said, Jennifer’s story was “a great reminder why we’re doing this”.

Despite such an inspiring story, I was conflicted about the appropriateness of the talk for this meeting. Earlier this year, I attended an epidemiology lecture in Cambridge about breast cancer, which chastised early screening programs for their severe effects on people who do not actually have cancer. (e.g., people receive chemotherapy when they do not have cancer, and this represented the majority of people who test positive). The argument was that the success stories (i.e., cancer caught early = good) were far more persuasive and dominated public policy from a public relations point of view and nobody properly considered the costs of treating people who do not have cancer.

Jennifer is one of many patients who underwent the treatment, and while she is not the only person who responded well (another example is Alex Normandin), she does represent the “positive side” and we certainly do not hear talks at conferences of people who have undergone failed stem cell treatments. One cannot argue that the treatment can work, but the purpose of doing a larger trial (as Drs. Atkins and Freedman are doing) is to know how frequently it works and how frequently it is actually bad for patients. Indeed, in the early phase of the trial, one patient died due to chemotherapy-related toxicity.

To me, it seems more prudent to wait for the final trial results before showcasing success stories – public policy regarding stem cell trials needs to be based on data with the complete picture available and not heart string tugging. I’m delighted that patients like Jennifer have had such a strong positive response to treatment, but we really need to consider all patients and the potential damage that such treatments can cause.

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David Kent

David Kent

Principal Investigator at University of Cambridge
Dr. David Kent is a Principal Investigator at the University of Cambridge in the Cambridge Stem Cell Institute (http://www.stemcells.cam.ac.uk/researchers/principal-investigators/dr-david-kent). His laboratory's research focuses on fate choice in single blood stem cells and how changes in their regulation lead to cancers. David is currently the Stem Cell Institute’s Public Engagement Champion and has a long history of public engagement and outreach including the creation of The Black Hole in 2009. He has been writing for Signals since 2010.
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4 Responses

  1. Sonja B says:

    Well said Dave. I agree that Jennifer’s story was powerful and inspiring. Ultimately, though, I see a lot of similarities between ISSCR presenting stories like this one, and dubious “stem cell clinics” publishing patient testimonials on their websites – both highlight individual positive outcomes without necessarily putting them in the context of results from larger trials, and both do it ostensibly to win public support.

  2. Christopher McCabe says:

    Great blog.

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