The New York Times reports on a successful collaboration, the Alzheimer’s Disease Neuroimaging Initiative, which created a platform for sharing data on the biological markers (or biomarkers) of Alzheimer’s:

The key to the Alzheimer’s project was an agreement as ambitious as its goal: not just to raise money, not just to do research on a vast scale, but also to share all the data, making every single finding public immediately, available to anyone with a computer anywhere in the world.

There were many barriers they had to overcome to get scientists to  share their data. In the end, they managed to get scientists, government agencies, pharma companies, universities and nonprofits to work together to better diagnose Alzheimer’s and measure the  progression of the disease.

It’s great to see yet another example of a collaboration working in medical research.

Read the rest here.

The end of July will bring another opportunity for scientists, entrepreneurs and philosophers to gather and talk about what they think and know about anything and everything within the scientific realm of information. The event is called the Open Science Summit and will take place in Berkeley, CA. If you would like to know more about the event, check out this great article by Joseph Jackson at http://bit.ly/cjaOXg.

Joseph Jackson is a philosopher, entrepreneur, activist, and organizer in the Open Science Movement. He is CEO of Lava-Amp, a company building a very inexpensive hardware platform for portable PCR, ideal for use in developing countries and in biology education. He is also co-founder of Biocurious.org, the first Bay Area community lab for citizen science.

It’s no secret that the process of taking discovery biology and turning it into a treatment for any disease, like Parkinson’s or multiple sclerosis, is broken. It takes too long and it costs too much money. However, as with most questions we ask these days, there may be a search engine out there that has the answer. Or at least one of the brains behind the search engine does.

Sergey Brin, one of the master mathematical minds behind Google, is using his knowledge of large data sets and the information they can provide, as well as his large wallet (he’s worth about $15 billion) to do two unthinkable things: significatly fund research for a disease he does not have and rethink the entire way scientists conduct that research.

In a recent article by Thomas Goetz, Senior Editor at Wired Magazine, Sergey Brin exposes the Parkinson’s predicament he has found himself in. Learning that his genetic code contained LRRK2, a gene that has been associated with an increased likelihood of developing Parkinson’s, Brin has begun the process of decreasing his potential to incur for the devastating disease bit by bit.

Goetz writes about how Brin is making adjustments to the environmental factors that affect his life, such as his exercise routine and caffeine consumption. But these changes, to Brin, are not enough to lower his potential of developing Parkinson’s to the nearly impossible level he’d like. So instead, Brin decided to take matters into his own hands, by not only funding research for a disease he has yet to, or may never, develop but also taking the time to hypothesize how one might fix the broken system of medical research.

Image of the LRRK2 Gene

So now you may be wondering why you are reading this article on the Myelin Repair Foundation blog when it seems to have nothing to do with MS and everything to do with Parkinson’s research. But here’s the thing. At the MRF we like to surround ourselves with people who have innovative ideas. People who take what’s presented before them and ask, how can I make this work better? What we, at the MRF, see in Sergey Brin is someone who is as equally as frustrated with waiting for cures as we are.

By financially supporting Parkinson’s research, Brin is speeding up the pace to find a treatment. He says, “Generally the pace of medical research is glacial compared to what I’m used to in the Internet, we could be looking lots of places and collecting lots of information. And if we see a pattern, that could lead somewhere.” But it takes more than just money to make something happen – it also takes an idea. And that’s just what he had in mind when he came up with his cutting edge way to slice the time it takes to develop treatments into a fraction of what it once was.

Brin, an expert in algorithms, wants to use what is called market-basket analysis, a computer science term that names the process of mining large data sets for useful associations. This way, scientists can begin with tons of data, formulated by an outpouring of observational patients, and then wade into the muck of it all, searching for “patterns and correlations.” Now, working with the Parkinson’s Institute, the Fox Foundation and his wife’s company, 23andMe, Brin will begin experimenting with his new take on the scientific method. They will use 10,000 people in their search to attempt to find a meaningful connection between the participant’s environmental factors and or family history and their disease progression etc.

With people like Brin and the MRF, who can back up their innovative ideas with funds, it looks like they’re going to give the current drug development system a run for its money. And if it pans out for Parkinson’s, who knows what the minds at Google could do for MS.

To read more about Sergey Brin’s philanthropic work, check out Tom Goetz’s article on the internet version of Wired Magazine at http://www.wired.com/magazine/2010/06/ff_sergeys_search/

We’ve all heard the phrase “secrets don’t make friends.” Well, they don’t make drug treatments either.  One of Forbes magazine’s blogs, The Science Business, is publishing a series of articles on how secrecy is hurting drug research. Matthew Herper, a senior editor at Forbes, authored a piece published yesterday, June 1, on the topic and it has struck a chord here at the Myelin Repair Foundation.  He discusses the idea that it may be “a companies’ tendency to keep their research under wraps” that is holding them back, which is one of the reasons we use a collaborative model in our research method.

Herper also takes a moment to pinpoint some of the other locations across the country,  such as the University of California, San Francisco and the headquarters of the huge drug developer, GlaxoSmithKline, that have started to share information with each other in hopes of minimizing drug development costs and maximize the number of therapies and/or cures they can find. He says “Traditionally, drug companies don’t share data with each other as studies progress. Increasingly, it seems that might be a mistake.”

If you want to read more, visit:  The Science Business Blog,  Forbes Magazine

There will be articles discussing the secrecy of drug research all week. Also, check out how the Myelin Repair Foundation is using the idea of research collaboration in their ARC model at MyelinRepair.org’

Crossposted at Myelin Repair Foundation Blog

Amy Dockser Marcus writes in the  The Wall Street Journal (April 13, 2010) about the challenge of creating a cultural shift of collaborations and sharing of information amongst scientists.

Scientists worry that if they share data before publishing their findings, someone else might claim credit for a discovery they made. And even after they mine information for themselves, they frequently cling to the notion that more may be discovered, and so continue to hoard the data.

Many organizations and government institutions are finding that collaborations need to be managed throughout the whole process in order to make progress on research. Read the full article “My Data, Our Data, Your Data.”

Margaret Anderson, CEO of FasterCures, recently moderated a webinar with the Myelin Repair Foundation, the Kauffman Foundation, Robert Wood Johnson Foundation and Scott Cook & Signe Ostby Foundation on the topic of “Developing Cures with Less Time & Capital.”

She also just published an article in the Milken Institute Review on the “perfect storm of discovery” brewing which may refocus a search for cures for more diseases.

In the article, Anderson discusses the fact that most basic science research funded by the National Institutes of Health are focused on revealing the underlying mysteries of biology, which is necessary but not sufficient to develop patient treatments.  She sees a growing role for non-profit disease research organizations in sparking innovation in the process due to their close relationships with patient communities,  tools such as trial registries and tissue banks, and their ability to move quickly to address emerging translational and clinical opportunities.

Read her entire article in PDF format here.

We posted a question on LinkedIn asking others how they would define or explain cures and treatments because many Patient’s Manifesto signors thought of them as mutually exclusive terms where cures end a disease and treatments only treat the symptoms.  However, we thought there needed to be a more nuanced discussion of these definitions especially in light of our initiative, “Where Are The Cures?”

Many responses came back explaining the differences and similarities, but the best response by far was from Mark Davison in the UK. He wrote up a longer blog article on the subject below:

Treatments and Cures: Must-Have versus Nice-To-Have?

“Where are the cures?” is an understandable reaction to the slow progress of biomedical research.  Why do things take so long? The scientific method is inherently cautious: to get meaningful results, we design controlled experiments to isolate and test one variable at a time.  The human body, however, has millions of interacting processes occurring all over the body and changing over time.  Translating results from test tubes to people is therefore a tough task.  That research breakthrough we hear about on the evening news may not become a marketed drug for years, if ever.  If there were big shortcuts we’d have found them by now, but maybe a change of emphasis could help.

The success of modern medicine means that most of us see disease and health as opposite states; black and white.  We expect doctors to convert one to the other when required – to diagnose, to treat and to eradicate our illnesses. However, the disease-health relationship is often more like a continuous spectrum than our all-or-nothing perception.  If we view medicine in this way there is value to be had all along the rainbow, even if we never find the pot of gold that is a cure.

In the nineteenth century life expectancy was half what it is today. In the pre-pharmaceutical era survival was often due more to luck than to medicine.  In the Civil War, non-sterile amputations to treat gangrene frequently caused death by septicemia.  Joseph Lister’s innovation of antiseptic surgery occurred in 1867, just after the war ended, and was followed sixty years later by Alexander Fleming’s discovery of penicillin.  By the time of the Second World War, the chances of surviving battlefield wounds (and other previously life-threatening conditions) were much higher.

In the twentieth century, scientific progress made medicine ever more effective. The underlying causes of many dangerous diseases were uncovered.  Today, ailments that would have killed our great-great-grandparents can often be cured or prevented.  In the 21^st century, all the “simple” diseases have now been addressed, if not necessarily eradicated, but more complex problems such as multiple sclerosis still remain to be conquered, despite many billions of dollars spent in pharmaceutical research.

As noted above, we know that drug treatment and cure aren’t necessarily linked in a straight line. Some medical conditions (depression, for example) are difficult to study because of well-known placebo effects. The drug administered may be biochemically no more effective than breath mints, but the medical attention makes the patient feel better and gives the appearance that drug treatment was successful.

Similarly, some patients with chronic illness are never fully cured but effective treatment can liberate them from the tyranny of their condition.  “Absence of pain” or “increased mobility” is usually more important to the chronically-ill person than “freedom from disease”. Maybe we should look for more “functional cures” – treatments that don’t eliminate underlying problems but allow us to live with them and thrive.  This isn’t a new concept – many neurological and auto-immune diseases are already managed in this way, but cure or regression of disease is stillthe primary commercial aim.

In my view, maximum quality of life should be the first goal. Treatments need to reduce symptoms and minimise further damage – still very difficult to achieve, but maybe just fractionally easier to find than a full cure.

Cures will always remain the ideal, and we should continue to pursue them, but they are a home run play and usually the R&D bat swings and misses.  Maybe a drug-based remedy will never be found for some complex, multi-factorial diseases.  Perhaps instead of swinging for the fences we need to bunt and run like hell to first base.

We live in symbiosis with a load of aberrant biology already – we carry more bacterial cells with us than human ones. Maybe some diseases will also be seen as harmless passengers when we find the right medical approaches. Treatment and cure are nineteenth century concepts, when medicine was all or nothing. In 21st century healthcare, continuing research and new treatments can help patients even without curing them.  As Voltaire said, perfect should not be the enemy of good.

About the Author: Mark Davison has held a number of research and commercial roles in pharma and biotech during a twenty year career, including responsibility for CNS therapeutics at a biotech start up.  He is a drug industry consultant and writer.

See Mark’s blog at: http://pharmapieces.blogspot.com

If you would like to contribute an article please contact us at info@wherearethecures.org with your pitch.

MRF  President Scott Johnson recently spent some time talking with writers at the Arabella Philanthropic Advisors who were working on an issues brief to advise their clients on best practices in making philanthropic gifts for medical research. The  Issue Brief, produced in collaboration with FasterCures, explores strategies donors can employ to help improve the current medical research process and to support the search for breakthrough treatments and cures. The MRF’s research model was featured in the brief as one of the success stories.

According to the brief, the philanthropic sector provides $918 billion to support medical research, small compared to the billions spent by the NIH & pharmaceutical companies, but philanthropists operate with fewer constraints and have the opportunity to support higher-risk, yet potentially high-impact research — work that is crucial in making breakthroughs.

There are many challenges in moving treatments through the therapeutic drug discovery pipeline:

- The lack of incentives for translational research – the Valley of Death between basic science discoveries and commercial development.

- Institutional constraints - As R&D costs rocket, companies are becoming more risk-averse. Academic scientists are also incentivized to only focus on basic science which isn’t sufficient to develop therapies for patients.

- Roadblocks to collaboration - The pressure to publish, tenureship and the peer review system which determines grant funding are factors that discourage scientists from collaborating. There are no cross-disciplinary approaches being used to further a discovery.

The issue brief advises how philanthropists can encourage change in the system by investing in certain activities or encouraging innovation in existing organizations:

• Help close the translational gap: funding efforts to collect data and samples for patients, support programs to expand networks of clinical trial sites or invest in new models for testing treatments.
• Invest in validation work: To ensure a product’s commercial relevance, companies & investors need to see replicated results or published papers indicating a discovery’s promise.
• Fund collaboration: Support organizations as they develop processes for sharing data & establish metrics for measuring success. Fund online tools & infrastructure to facilitate collaboration. Support efforts to bring scientists together to exchange ideas and share best practices.
• Tell the story: Support documentaries or education programs that raise awareness and help the general public understand the current system and its limitations.

As supporters of the Myelin Repair Foundation know, we are already working on all four areas listed above. We have also recently launched our WhereAreTheCures.org site to educate the general public on the barriers in the current medical research system.

If a donor is currently giving to an organization working on a disease they care most about they should ask to see a research and development strategy, hold the organization accountable by asking how they measure progress, find out their interim milestones and ask to see their management plan and list of advisors.

Download the PDF of the Medical Innovation Issue Brief to read more.