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October 05, 2011

Unconstrained thinking: the link between computer chips and clinical trials

by Drew Lyall

The World Stem Cell Summit taking place this week in Pasadena, California, occupies a unique place in the stem cell calendar. The summit brings together patient advocates, policy makers, industry and scientists from around the world to take stock of progress in the field; to discuss common political, regulatory, financial and scientific barriers to therapies reaching the clinic; and, to actively collaborate on moving efforts forward.

One of the joys of this meeting is that is usually includes a thought-provoking keynote talk from an icon outside of the field, but with a deep personal interest in its success. This year's keynote came from Andy Grove, who founded Intel Corporation in 1968, and in various roles including Chief Scientist, President & CEO and Chairman grew the company to the multi-billion dollar behemoth it is today, ranking right along side alongside Microsoft, Google, Apple and now Facebook as one of the giants of the Information Age.

In 1999, Grove was also diagnosed with Parkinson's disease.

In recent years, Grove has spoken widely about how the lessons he learned in Silicon Valley might be applied to biotechnology in order to speed the identification and delivery of cures to the clinic. While skeptics have suggested that "people are not chips", and that a single human cell is significantly more complex than even the fastest chip, Grove remains undaunted, investing more than $22 million of his own funds in partnered projects with the California Institute of Regenerative Medicine, and reportedly pledging another $40 million to the Michael J. Fox Foundation.

In his talk on Monday, Grove had two key themes. The first related to the way in which biotechnology research is conducted, and his strong belief in the imperative of collaborative translational research programs. He even went so far as to suggest that all health research should be conducted in hospital-based research institutes, so that patient needs and clinical practice drive the innovation process. While the need for physical co-location could be debated, the focus on collaborative translational research has been a core value of entities such as Canada's Stem Cell Network for the past 10 years (it’s our belief that the rapid advances we have catalyzed in Canada flow from the establishment and support of multidisciplinary research teams that included stem cell biologists, bioengineers, and clinicians).

The second theme related to his frustration with the current regulatory approval process of multi-phase trials prior to a treatment being approved by the FDA / Health Canada and other national regulators. As Grove pointed out, the problem with this process is that it can take 5-10 years to complete, which may be beyond your life expectancy if you have one of the many devastating diseases stem cell research is targeting. Furthermore, not everybody can enlist in a trial -- the "exclusion" criteria (i.e. the necessary characteristics of patients that can be enlisted in the trial to ensure it is statistically valid and methodologically sound) can be quite narrow, leaving nowhere for patients to go (except perhaps to one of the many unregulated “clinics” -- see other blog articles). The frustration of patients is completely understandable, especially if the treatment is eventually validated, and could have helped them had they been allowed in the trial.

Grove's solution is to give the right to a patient to "opt in". In this scenario, a patient would be given the right to take a drug the regulatory agencies have deemed to be safe (albeit safety cannot be certain until the drug is released into the general population), even if there is insufficient evidence of efficacy. The patient would not be part of the trial data, but, Groves argues at least the patient would have the right, as a fully-informed adult, to take the risk in the hopes that they may be lucky, and it does.

Grove makes a fairly compelling case, although of course it opens a myriad of questions:

  • Who will pay for the treatment, and who is responsible for complications if the treatment does not work out?
  • In a resource-constrained health-spending environment, should we allocate dollars to treatments that have not yet been shown to work, when there are still people with other conditions waiting for treatments which do?
  • Will patients still be willing to enter "proper" clinical trials where half of them will receive a placebo, when they know they could opt-in and receive the drug, and if so, how will we assess drugs moving forward?

Grove's proposal would require a radical rethinking -- indeed a revolution -- of our whole drug-approval process, and a discussion of the rights, obligations and expectation of government with respect to our health. Maybe that was the final unspoken point of his talk to that audience, and why it made sense to deliver it at the World Stem Cell Summit. If therapies are going to get to the clinic faster, we need to think beyond our own domain to consider the whole innovation process.  Each of us: patients, scientists, doctors, regulators have an important role to play; in doing so, we must not let our thinking be constrained by convention. Constrained thinking is not how Intel became the dominant chip company on this planet.



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Conceptually I am all for free choice to opt in. Certainly the money issue can be addressed as a strategic choice by individuals, companies or government. However, as I have continued to reflect on this, the big challenge to me seems to be how we develop sound trials, and determine if a drug actually works - what incentive will there be for a patient to enter the clinical trial, and "risk" getting the placebo, when they could opt in and take the drug? Knowing which drugs work and which don't is a huge societal benefit....

With all the foundations and capital out there why is medical attention always a function of money? How much money will be saved by curing people to live productive lives? If we needed to go to war tomorrow the funds would be there...why not make war on disease?

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