Aug 2013
Snipped Slamon and Mak

Not an easy job, but the demand is there

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Welcome to the first instalment of the Stem Cell NewsDesk, the Foundation’s attempt to help Canadians better understand where a “breakthrough” fits on the research lab-to-clinic continuum.

Essentially, the aim of NewsDesk is to try to answer one question: how does [insert news-making development/discovery/breakthrough here] contribute to finding a treatment or a cure for a currently untreatable or incurable disease?  The idea is not to hype stem cell science but to provide realistic reports on developments as they occur.

It won’t be easy. Stem cell science is complicated and it can be hard to decipher whether a discovery represents a monumental leap forward or is just an incremental improvement in understanding how stem cells function. Sometimes it is obvious, as with Dr. Shinya Yamanaka’s 2006 Nobel-winning discovery of how to make embryonic-like stem cells from almost any cell in the body – cells we now call induced pluripotent stem cells. Sometimes it’s not. Remember that the first demonstration of the unique properties of stem cells 50 years ago flew in under the radar.

In email correspondence, Dr. Connie Eaves, a Vancouver-based researcher whose team was the first to isolate breast stem cells, shared her thoughts on why this is such a challenge:

  • Every ‘new’ piece of information about how cells work and how their behaviour can be predictably manipulated is potentially a breakthrough – but it may take years to understand whether/when/where/or for what that will be true. So, making a fast judgment is rarely possible.
  • Current efforts use unknowns (new molecules with an experimental rationale) to treat unknowns (human tumours we don’t understand).
  • Clinically, an improvement of long-term survival from 5% to 15% would be considered a big advance. But if you were an affected patient, you might not see it that way, as overall your survival chances would still be pretty bad.
  •  What is useful clinically requires a controlled trial and this usually takes a long time (10 years) and the result may appear sort of boring by the time the answers are all in.

Case in point: the ‘sharpshooter’ story

Dr. Eaves is part of the 100-person team led by Princess Margaret Cancer Centre’s Dr. Tak Mak and Dr. Denis Slamon, (pictured at right) of the University of California, Los Angeles that made headlines in mid-June by announcing they had developed a new kind of “sharpshooter” anti-cancer drug.  Given the excellent track record of the two scientists – Dr. Mak revolutionized how scientists think about the human immune system by cloning the T-Cell receptor and Dr. Slamon developed the breast cancer drug Herceptin – it’s not surprising the announcement garnered major media attention.

As the Toronto Star explained, the new drug, which has been tested on mice for ovarian, breast, pancreas, lung and colon cancer is called a sharpshooter because it goes after a specific enzyme to shut down cancer.  Unlike chemotherapy, which can kill healthy, quick-replicating cells, the drug, called CFI-400945, takes aim only at the cancer cells.

On CTV’s Canada AM, host Bev Thompson described it as “being hailed as a major breakthrough in cancer research” and said while “we’ve talked about breakthroughs before … this seems like a cut above.”

In the Globe and Mail, however, Canada’s leading health writer André Picard, pointed out that CFI-400945, has “not been tested on a single person” and that “even in a best-case scenario” a new cancer drug “is at least a decade away.”

As excited as they were, the Princess Margaret researchers also urged patience. On that Canada AM segment, Dr. Philippe Bedard explained that the three-phase clinical trial process is a marathon, not a sprint, stressing that there is a long road ahead and it “can take many years.”

So where does that leave cancer patients?

Officials at Princess Margaret say there has been lots of interest from people who want the new drug. That will take some time: Health Canada approved CFI-400945 for use in human trials in mid-July. Next, it goes before the University Health Network’s Research Ethics Board for approval. A trial involving a small number of patients to see if CFI-400945 is safe – likely will begin in November.

So why did Princess Margaret bang the drum so loudly at such an early stage? The sharpshooter announcement actually came from The Princess Margaret Cancer Foundation to make donors aware of the potential advances that are critically dependent on the funding support that their donations provide. Makes sense:  Canadians support medical research through their charitable donations as well as through their taxes and want to know how their investments are doing.

No quick fixes

But the reaction shows that there is a real and growing need for a resource to help people understand how a treatment may have an impact on them. As stem cell research moves closer to providing new treatments, people will want to know more.

NewsDesk hopes to help in this. Again, not an easy task. And there will be lots of cautions and caveats attached to our discussions of breakthroughs.  Because the reality is: there are no quick fixes or magic bullets. But progress is being made – almost every day.

So let’s go back to Dr. Eaves, who is a member of the Foundation’s Science Leadership Council, and her thoughts on the sharpshooter announcement:

“The Tak Mak result looks very exciting in the experimental models studied to date. But there is not much history yet to know how these will correlate with patient outcomes. I can’t say much more than that and don’t think anyone can at this early stage.”

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