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For Scientists, Collaborative Efforts Could Speed Medical Advances

September 23, 2010 at 5:15 PM EDT
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Special correspondent Dave Iverson reports on a new push to get scientists to work together to find medical cures sooner.
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JUDY WOODRUFF: Now: new developments in the field of biomedical research.

Special correspondent David Iverson was diagnosed with Parkinson’s disease in 2004 and has been reporting on the search for effective treatments for that disease and others.

He filed this report for our Health Unit, a partnership with the Robert Wood Johnson Foundation.

DAVID IVERSON: Scott Johnson has had multiple sclerosis for 34 years. He’s coped with everyday challenges, like how to drive a car.

SCOTT JOHNSON, president, Myelin Repair Foundation: It’s impacted, you know, virtually all my adult life. You know, my — my right hand doesn’t really work very well, or really at all. My right leg doesn’t work great.

DAVID ANDERSON, Kidney Disease Patient: I actually need someone to show me what I weigh now. That’s good.

DAVID IVERSON: David Anderson’s challenges are different. He has kidney disease. And a transplanted kidney failed three years ago. Today, he depends on dialysis to survive.

DAVID ANDERSON: When I need dialysis, mostly, I notice that I feel sort of muddy in my thinking. You know, the toxins in the blood are not just in your leg muscles. They’re all over your body, including in your brain.

DAVID IVERSON: David Anderson and Scott Johnson contend with very different conditions, but they share a similar frustration: the slow pace of medical advancements.

So, why isn’t more progress being made toward solving a variety of complex diseases? After all, the National Institutes of Health spend over $30 billion a year in research. The problem may not be how much money we spend on research, but the medical research system itself, and a problem sometimes known as the valley of death.

A problem that frustrates patient advocates like Scott Johnson.

SCOTT JOHNSON: The valley of death really is a gap in the overall process. So, you’ve got — at the front end of the process, you have got academics that are doing basic science research.

And the result of that is about 800,000 papers published every year. That’s a lot of papers. At the other end of the spectrum, you have got the pharmaceutical and biotech industry that — that actually has to turn things into medicine.

DAVID IVERSON: To bridge the gap, Johnson says, requires scientific grunt work that often doesn’t get done.

SCOTT JOHNSON: You need to replicate experiments many times. And that just doesn’t get funding. That’s not something that is interesting for an academic to do. It’s not something that helps their career. It’s not something that you get a grant to do.

And so those steps just don’t happen. And, on the other end of the spectrum, you say, well, why doesn’t pharma do it? Well, because, how do you wade through 800,000 papers and figure out, what is the gem that I should put all this money in to do these steps?

DAVID IVERSON: So, nine years ago, Johnson decided to leave his career as a Silicon Valley executive and start a foundation aimed at speeding the research process and finding a new treatment for M.S. To do, he immersed himself in the world of science.

DR. BEN BARRES, Stanford University Medical School: One of the exciting things is that we found that it looks like it’s turning on some genes, but also turning off other genes.

Johnson got to know Dr. Ben Barres who, as chair the neurobiology department at Stanford University, was knee-deep in the world of academic research.

DR. BEN BARRES: We work very hard for a few years, and we publish a paper in a good journal. And I always assumed that that discovery, if it were immediately relevant to a new treatment, would be picked up by one of the big drug companies and then develop the next step. And, so, I thought I was doing my part.

DAVID IVERSON: Like most researchers, Barres had focused on intriguing scientific questions, but not on the ones that might help patients most.

In multiple sclerosis, the body’s immune system attacks something called myelin, which sheathes the nerves. Without it, the brain can’t tell muscles what to do. To date, most research had focused on why myelin goes missing, but not on the practical question of how repair it.

Then Scott Johnson came along.

DR. BEN BARRES: And, so, he sort of embarrassed everybody, you know, from coast to coast into saying, we have got be working more on this question.

DAVID IVERSON: Johnson’s Myelin Repair Foundation has now raised $43 million for research, including much of the work that Ben Barres is now engaged in.

But the funding deliberately comes with strings attached. The research must be patient-centered. Scientists must work collaboratively and provide the kind of data that would persuade drug companies to take the next step, a model that could work for other diseases, too.

(APPLAUSE)

DR. SUSAN DESMOND-HELLMANN, chancellor, University of California, San Francisco: I’m Sue Desmond-Hellmann, the new chancellor.

DAVID IVERSON: The new chancellor at University of California, San Francisco, knows something about getting private industry to move forward.

Dr. Susan Desmond-Hellmann came to her new position at UCSF fresh from Genentech, where she oversaw the development of breakthrough cancer drugs like Herceptin in the 1990s.

But Desmond-Hellmann thinks private industry has become less willing to make those investments.

DR. SUSAN DESMOND-HELLMANN: There is concern today that that kind of innovation, that kind of basic science and following it with great translation and clinical science is too rare.

DAVID IVERSON: To reignite innovation, Desmond-Hellmann believes academic scientists have to take on the collaborative approach she once witnessed at Genentech.

DR. SUSAN DESMOND-HELLMANN: The experience that I had in private industry, I always worked as a member of a team. And that was great fun. I couldn’t be and wouldn’t be the expert at all things, but if, in the end, something good happened, I was happy and rewarded for that, for a good outcome, not whether I published.

DAVID IVERSON: And — and is that something that you’re trying to inculcate here at UCSF, that more collaborative, interdisciplinary approach?

DR. SUSAN DESMOND-HELLMANN: Absolutely.

DAVID IVERSON: And that collaborative principle is gaining adherents.

At the San Francisco Veterans Administration, an Alzheimer’s brain imaging project uses a novel data-sharing approach. The project draws on funding from government, industry and academia. It’s led by Dr. Michael Weiner of UCSF and the Veterans Administration.

DR. MICHAEL WEINER, Veterans Administration: When any scientific project is done, the scientist collects the data, keeps it in their laboratory, and then writes a paper about the data, that is, the raw data is not released, just a paper which is a description of the raw data.

DAVID IVERSON: But, this time, scientists did the opposite.

DR. MICHAEL WEINER: We decided, for a number of reasons, to just share all the data that we collect from this study, and to share it widely on the Internet. And the results of that have just been amazing.

DAVID IVERSON: The Alzheimer’s study followed 800 patients at 50 different centers. The researchers all had instant access to the same data, and worked collaboratively to figure out a way to detect Alzheimer’s early on, a potentially significant breakthrough.

DR. MICHAEL WEINER: What’s extremely interesting is that we’re seeing signs in the completely normal people…

DAVID IVERSON: Early on?

DR. MICHAEL WEINER: … that they have biomarker signatures of Alzheimer’s disease.

DAVID IVERSON: So, I mean, in a way, is this like having real-time, open-source research?

DR. MICHAEL WEINER: Yes.

DAVID IVERSON: Is that what is different?

DR. MICHAEL WEINER: Yes, yes.

DAVID IVERSON: And it’s research that’s both collaborative and patient-focused, which chancellor Desmond-Hellmann says has to be the primary academic goal.

DR. SUSAN DESMOND-HELLMANN: Increasingly, what people call translational science, or I would call clinical science or human biology, is allowing us to say, that science question you just asked and answered, first of all, is that relevant to a human being? And, secondly, what about the patient?

DAVID IVERSON: Which takes us back to David Anderson, the kidney disease patient we met at the beginning of this story. Right now, he faces the prospect of being hooked up to a machine three days a week for the rest of his life. But a new development has changed those prospects.

DAVID ANDERSON: The information I got recently about the possibility of creating an artificial — an implantable artificial kidney, well, you know, that’s something worth hanging around for.

DR. SHUVO ROY, University of California, San Francisco: So, what we have here is a little model of our device.

DAVID IVERSON: An artificial kidney is in the works at the laboratory of Dr. Shuvo Roy.

And this would actually be implanted and would be roughly this size, but lighter?

DR. SHUVO ROY: This may look big to us, but, actually, to give you some context, it certainly would fit within the abdomen of most of us.

DAVID IVERSON: Dr. Roy is an engineer by training, but the project brought together cell biologists, material scientists, engineers, and practicing physicians.

DR. SHUVO ROY: By finding those right people together, I think we can sort of take out the traditional way of doing academic research in silos, but saying, let’s put our arms together and brains together, and solve a problem collectively. And, for a project like this, you absolutely need that to succeed.

DAVID IVERSON: It’s the same collaborative approach that Scott Johnson’s Myelin Repair Foundation is promoting, changing the model for how research works and solving the problem of multiple sclerosis that’s challenged him for 34 years.

SCOTT JOHNSON: And to be able to prevent others from having to go through that, I think, is — to me, would be something I would like to do. But, probably on a larger scale, it’s really about this model, because I think that this model can be applied to any disease research.

DAVID IVERSON: And, of course, there’s no shortage of diseases still to tackle.