Winds of change are blowing through the corridors of many pharma companies as executives develop more concrete ideas to optimise stem cell technologies in terms of efficiency and responsible use. As one would expect, the first to profit are those in the industry itself capable of delivering solid results rather than promises and hopes for the future. More surprising is that patients are also reaping benefits from these changes, at least those who belong to patient organisations. It is not ‘biotech’ per se, as one might assume, but differences being introduced into pharma leadership. That and the use of methods and analytical instrumentation able to reflect the trajectory of stem cells.

  In June 2005 Roger Perlmutter, CSO of Amgen, told the Financial Times about plans to enter into “business with stem cells”. Interestingly, his answers were not the usual honed and polished product of a corporate communications department, but delivered ‘straight from the hip’, so to speak, in direct response to the journalist’s questions. His message has broad appeal, and remains so more than four years on, despite the ubiquity of commercial research and stem cell products that began to emerge a decade or so ago. The viability of the science is underlined when one counts the number of pharma executives either taking major investment decisions in the field, or actually sitting on the boards of biotech companies dedicated to stem cell research.

  So, back to Amgen and the FT. In the interview, Perlmutter repeated what was already known at the time. In addition, he was especially careful to repeat the standard industry statement that Amgen is not interested in any commercial uses of “embryonic stem cells”. But he may not have been careful enough. Insiders report that Perlmutter came under fire back at the office for voicing outspoken views on strategy in such a public forum. What is certain is that his message did not align with that of corporate communications.

  In 2007, BioWorld Europe*, a print magazine for life science professionals, (note, not the general public) launched a survey among pharma companies and their evaluation of available stem cell technologies, their shortcomings, and recommendations to overcome these weaknesses. At the time, there was a growing malaise among industry scientists doing lab work on stem cells. Major science journals introduced ethical debates in their technical sections – not seen often but necessary in order to capture the public mood. Patenting issues were contaminated by politics, especially in continental Europe – in particular France and Germany, somewhat less in Sweden, and even more muted in the UK.

  It would be normal for industry scientists working in the field to attend scientific stem cell conferences. So far, so good. But what most found irritating was a feeling that they were being ‘muzzled’ by their companies during these conference visits. As editor of BioWorld Europe I attended BIO in Boston - the US pharma industry’s largest partnering event for biotech - in order to meet business developers for and corporate spokespersons of big players such as Genentech, Pfizer, and Amgen. Now, BioWorld Europe was not FT, and has limited appeal other than marketing for the average US pharma company. However, even a European-based trade magazine can expect some positive response to requests for interviews. Sadly not. I began to realise what Dr. Perlmutter must have felt after returning to the office after his much-discussed FT interview. The industry scientists I spoke to informed me about their companies’ upgraded communications policies regarding in-house stem cell projects when talking to the outside world. Depending on the company, reactions range from failure to respond, to delays and subsequent abandonment, to ridicule – even lies. Of course, such a ‘head-in-the-sand’ approach is counter-productive when it comes to building trust, open innovation, or what Pfizer’s Jeffrey Kindler calls a “scientific powerhouse”. It introduces a cynical world view, both among employees and observers outside company. Let’s take another look at the FT interview. Even before the article was written, Amgen had not only externalised stem cell research to hospitals, universities and biotech firms such as Stem Cells Inc. and Viacell, they were also working in-house with (mice) embryonic stem cells – developments which the FT reporter did not pursue and about which Dr Perlmutter kept silent in the interview.

  There is a myriad professional bodies, industry organisations and scientific societies who rely on the silence of pharmaceutical companies when it comes to discussing R&D, despite the temptation felt among all scientists to discuss their work among a wider professional audience. It must be remembered that these professional bodies have their own agenda separate from, and sometimes in opposition to, the individual needs of their members. Thus, debates about stem cell technologies are often reduced to chatter about how to acquire public research funds, or a means for political manoeuvring. If we accept the overall goal of speeding up technology development and the need for reasoned and intelligent advocacy, the result of all these diversionary tactics is that they let the more important questions fall by the wayside. Such as: How do we properly finance the translation of stem cell discoveries into safe and valuable therapies? How do we develop modern and appropriate regulations to govern clinical use of stem cells, that will give greater certainty to investors? And, moving beyond ethical guidelines and clinical trials development issues: what technical standards need to be followed in order to validate toxicity testing with stem cells? What is the scope of a single discovery? or: Does a successful lab experiment with stem cells entitle the researcher to publicise the discovery by suggesting a novel ‘therapeutic’ has been ‘discovered’, even though the normal requirements of independent confirmation, upscaled studies, expanded clinical data collection and regulatory approval have still to even be considered?

It is true that any responsible application of these technologies has to be governed by consensus within society (e.g. on the ethical framework) and also legitimate political decision-making (e.g. leading to regulations), but it is equally true that scientific and medical research needs guidance in where to direct basic research (which need not contradict the norms of disinterested and curiosity-driven research!). Biotech suppliers and tool developers need guidance from pharma companies and other major customers on standards necessary once development leaves the research labs or when funding by the tax payer runs out. And this in turn requires a more forward-thinking attitude on the part of pharmaceutical companies, and a preparedness to invest in internal infrastructure, at least those interested in growing advanced therapy businesses. Even a vertically-integrated pharmaceutical company is unable to call all shots, if only because of the high risks of failure which go hand-in-hand with medicines research. A topical example comes from Genzyme on how this successfully works for employment security, profit generation and patient benefits. Another example are autonomous patient organisations like Association Française des Myopathies which operates basic research (like I-Stem), runs technology centres, establishes biotech companies, invests into pilot plants to manufacture (orphan) drugs, and supplies academic advisors to national regulatory bodies as well as EMEA.

  B2Bioworld is to contribute to the debate about growing ‘the business of stem cells’. B2Bioworld is proud to offer to our readers an edition, wherein pharma leaders of eight of the worldwide largest and most innovative companies of the industry share their experiences and views together with scientists, industry consultants and patent lawyers. Enjoy reading..

Notes

* BioWorld Europe (2007): Regenerative Medicine and Stem Cells.Edited by Wolf G Kroner, Editor and Founder of Biworld Europe.

** In this and upcoming issues B2Bioworld will publish further details about some of these companies. In 2005, and well ahead of others, GE Healthcare (including former Amersham in the UK and Pharmacia in Sweden) published a position statement on their understanding and approach to use stem cells (www.gehealthcare.com/company/position_statements/stemcell6_05.html). Make no mistake: this is no press release or cosmetic platitude. It is no less than an element of leadership, directed towards internal change, as well as a bold statement to the outside world this is a learning organisation. Content may be debated, but this statement from GE Healthcare stands out as a proactive landmark, a claim that they aren't afraid to deal with these issues and encourage their employees to help them critically evaluate stem cell technologies.