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A few weeks ago, Italian private company Stamina came under considerable pressure regarding their controversial use of mesenchymal stem cells in patients. The Italian government, it seems, had authorized an unproven stem cell therapy for use in patients. The International Society for Stem Cell Research issued a statement of concern and numerous leading stem cell scientists authored a letter in EMBO which describes the entire case as it stood in April 2013.
Last week Laura Margottini reported in Science on the Italian government’s parliamentary response. Instead of prohibiting the therapy, parliament opted to pass a bill that would demand (and fund!) a clinical trial to assess the claims of Stamina’s therapy. This brought to my mind, two very important questions:
1) Should parliament be involved in deciding which therapies have sufficient merit to support? and
2) Would the public money to pay for a clinical trial be better used elsewhere?
The first is quite tricky – on one hand, I like the idea of a parliament stepping in to say this is important to get right and the only way to know is to fund a proper trial. On the other hand, however, there are many potential stem cell therapies and choosing the one to support should be grounded in the best scientific evidence possible. No matter what the clinical need is for patients, a company should not be allowed to disregard safe manufacturing of cells or skirt the process of a standardized clinical trail to demonstrate efficacy in a small number of patients. Moreover, as Margottini points out in her article, there is significant disgruntlement in the Italian science community since stem cell research at the national level is only moderately higher than the cost of this single trial.
Indeed, it is an interesting question: Should public money be used to support such clinical trials? Clinical trials are a large component of the costs associated with bringing a therapy to market and are often cited as the main reason for exorbitant therapy (or drug) pricing. Therefore, unless the Italian government is planning to gain some of the financial benefit should the therapy prove successful, I am loath to support it. This case represents a single company getting a boost to defray its drug development costs because of a political uproar and represents an unfair advantage. While there may well be a case for publicly funded clinical trials, this seems like an odd way to start.
I am reminded of a talk by Cathy Prescott at the Royal Society meeting a couple of years back where she listed the challenges of recouping costs for cell therapy as a major hurdle for a field in which it is not as easy to gain patent protection. Cell therapies (which potentially represent major savings for nationally funded medical systems) are quite different from standard drug development and thus warrant serious consideration – not merely a reaction to public protest – from policy makers in all countries considering the best way to develop them.
David Kent
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Is this a welcome break from the bloody-minded mantra — by FDA and ISSCR — that all stem cells=drugs?
The issue is important because, the way I see it, the ISSCR/FDA is trying to gain exclusive control over therapies that could also be considered the practice of medicine.
With regards to safety, it is very relevant that the Italian therapy uses autologous MSC. We are not talking about induced pluripotent cells or ES from someone else…
As for efficacy, the ISSCR uses a false argument when they oppose on the basis that there is no “compelling evidence from clinical trials that such cells provide benefit to patients with neurological conditions.” This logic would always prevent every clinical trial!
It’s a bit vain-glorious to say “manufacturing cells”. Cells were manufactured by 3.5 billion years of evolution.
I’m not impressed by either side in this particular dispute.