I attended the first Sage Bionetworks Congress in 2010 and it left a powerful impression on my thinking. I have just attended the third congress in San Francisco and again the challenging nature of views, the real desire to make a difference, and the standard of thinking in the room will take me some time to process. But a series of comments, and soundbites over the course of the meeting have made me realise just how seriously bad our situation is.
- Attempts by a variety of big pharma to replicate disease relevant results published by academic labs failed in ~80% of cases (see for instance this story about this commentary in Nature[$])
- When a particular blood cancer group was asked what factor about their disease was most to them, they said gastro-intestinal problems. No health professional had ever even considered this as a gastro-intestinal disease.
- Jamie Heywood spent $25M of his own money on attempting to replicate around 500 published results that were therapeutically relevant to ALS and could not repeat the findings in a single case.
- A cancer patient, advocate, and fundraiser of 25 years standing said the following to me: “We’ve been at this for 25 years, we’ve raised over $2B for research, and new patients today get the same treatment I did. What’s the point?”
In a room full of very smart people absolutely committed to making a difference there were very few new ideas on how we actually cut through the thicket of perverse incentives, institutional inertia, disregard for replicability, and personal ego-stroking which is perpetuating these problems. I’ve been uncertain for some time whether change from within our existing structures and systems is really possible. I’m leaning further and further to the view that it is not. That doesn’t mean that we can’t do anything – just that it may be more effective to simply bypass existing institutions to do it.
Related articles
- Sage Congress: The synthesis of open source with genetics (radar.oreilly.com)
“[…] new patients today get the same treatment I did.”
I understand the frustration all too well. And advances are slow, in part because cancer is such a complex group of diseases. But it really isn’t true: treatments are improving, though the improvements are incremental rather than of the major-breakthrough variety.
For one of the most common cancers, bowel cancer, the current drug mixes and schedules are a real improvement in both survival and comfort over 20 years ago, and there’s new drugs such as cetuximab that improve the chance of other drugs working and presage new lines of approach. Control of the serious side effects have improved a lot, which means more patients are able to finish their regimens and have better quality of life during treatment (the point about blood cancer being a gastrointestinal disease highlights just how important this is). Improved surgery techniques means fewer get metastases, more metastates are surgically treatable, and mortality is lower for what is, sometimes, very invasive procedures.
Depending on how you see it, these advances are very modest or very significant. Overall survival is up, so is average years of life, and so is quality of life.
 “ I’ve been uncertain for some time whether change from within our existing structures and systems is really possible. I’m leaning further and further to the view that it is not. “What are the options? Foundations like Sage? New startups that try new ideas? Maybe an option is to bring brilliant people from other fields to see if their different frame of mind comes up with solutions.Â
It is indeed a sad situation but at the same time it is clear that there are many things to be done.
Cheers,
Julio
Julio, good question, we should focus on the positive recommendations. There are many options I think, Sage is one. Research taking place outside of traditional institutions is another, patient directed projects, and new funding approaches provide others. There are many things to be tried and many things to be done and where possible the main thing is that people can get on and do them without being stuck down and held back by existing incumbents and systems.
Jan, this is true, but it wasn’t so much the specifics that got me as that this was emblematic of a deeper problem. That medical research is configured, funded, and run for the benefit of medical researchers rather than patients. This is institutional rather than personal but I think there is a real risk that we are heading towards a situation where those people who have been pounding the pavements fundraising, and are our most natural allies in persuading government to support research are becoming disillusioned by what they are supporting.
“That medical research is configured, funded, and run for the benefit of medical researchers rather than patients.” Â Or put another way: in a world where the sole reward for research is (high-impact) publications, we don’t solve problems.