Take, for example, Lilly Fang. You remember, she won an award for research into predicting the progression of ALS (Lou Gehrig’s Disease). She is young to have won such a prize.

But it was not the fact that she is young that caused indignation among beneficiaries of the status quo. It was the fact that she is not a doctor. Nor is she a scientist. Nor has she studied any aspect of medicine. Nor did she even know, at the beginning, what ALS is.

We’ve got an unidentified intruder in Lab 4. Please call security.

Lilly Fang has not one, but two advanced university degrees, and not one, but two careers. She is a lawyer. And she is a mechanical engineer. So what is she doing in the science lab?

Well, Albert Einstein never set foot in a science lab, and neither did Lilly.
Lilly had 3 months off between university sessions. So she spent a lot of time at her computer.

And there is this website, you see, that gives the complete data of each patient, of the 8,500 ALS patients who have given themselves to clinical trials, posted on the internet in hopes that it would click with somebody out there. It clicked with Lilly. The lawyer and mechanical engineer saw things in the data. Things that no one had seen when the data was being kept hidden.

Remember when AMGEN decided to pull the plug on their Parkinson’s volunteers, and made no effort to determine why, for example, one of the patients had an improvement of symptoms estimated at 80% - for three years. AMGEN, trying to keep a straight face, said it was a placebo effect. Yeah, sure, a 3 year long placebo effect giving 80% improvement. They think we are stupid.

AMGEN made no effort to find out why one patient got so much healthier than others. As Andy Grove of Intel said at the time, in direct reference to the AMGEN GDNF scandal, the VCR was invented by accident, when one attempt came out very different from the others. That is when AMGEN ran away and hid the data and turned off the lights and pretended that they were not at home, at the exact point where real lovers of science jump in to find out why one is so different from the others. Researchers created the VCR by mistake. They jumped on the mistake, figured out what made it different, open-sourced it, and made digital history, along with billions of dollars. Typical socialists.

Task:
… to confront a basic puzzling question in ALS: most patients are like Lou Gehrig, with a rapidly progressing disease course, surviving for 2 or 3 years. Some patients, however, turn out to be more like Stephen Hawking, where the disease progression is delayed for decades. What separates the Lou Gehrigs from the Stephen Hawkings? … If you had ALS, you would find that to be an important question .

Hello? Mega-Pharma? Open source data? You say it can’t be done for data from clinical trials? You can’t post the patient data on that new-fangled electrical inter-nets; the earth is flat and the sun orbits around the earth. To believe otherwise is blasphemy.

You can’t change the way scientific research is done; it has to be done by the scientific method, which means promoting whatever your peer reviewers believed when they got their doctorate 30 years ago. It’s not possible to go open source, like they did in the computer and internet and video-game industries. (All open source, which is why there are thousands of apps from thousands of code-writers, which is why it all grew so fast).

Cast thy bread upon the waters and it will be returned to you tenfold.

But that’s not how they teach it at Harvard Business School, right? Genuflect and make it look sincere. And publish or perish.

Pharma says they can’t go open-source. We would steal their oh-so-brilliant ideas. We would all start producing agonists in our basements.

So your Apple will never be able to send a G-mail through Firefox to post a video on You Tube. Unless they open-source their code. Which of course they did.

Ha! Microsoft, Apple, Google, Intel, Facebook, Dell, and all the video game creators – all a bunch of socialists, if you ask me.

Hello? Parkinson’s research? There seem to be more citizen scientists with Parkinson’s than any other cohort. No one pays attention to them.

Your citizen scientists need access to the raw data. And you have no way of knowing who they are or where they are. They start to emerge from the shadows when you say, “Here is the day-by-day full information about every patient who ever volunteered for a clinical trial for ALS. Tell us what you see.”

If Lilly had been aiming at Parkinson’s she would not have had access to the data. Because the data behind Parkinson’s research is private property, and certainly not available on the internet for the likes of Lilly Fang, who had no experience whatsoever with any disease.

The Parkinson’s Archipelago has to realize at some point that research is not best expedited by keeping people in ignorance.

Lilly says:
… ALS causes progressive loss of motor function in patients and typically leads to death in about three years. Currently, there is no cure, and the causes are not well understood.

The task set out was to take patient data from the first three months of a clinical trial and use it to predict the rate of progression of the disease over the following nine months… the data was provided by the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database.

Lilly said:
One of our biggest challenges was the lack of information about what aspects of the available data would likely be predictive, as the academic literature had thus far only identified a few relevant variables. This lack of information, combined with our lack of medical expertise, led us to extract a broad spectrum of features from the data and to choose an algorithm that would be robust to irrelevant features. In the end, we hope to have identified some unexpected but useful features.

PRO-ACT contains thousands of fully de-identified unique clinical trial patient records, including demographic, lab, medical history, functional scores, and other data elements. The dataset includes both placebo and treatment-arm data from late stage ALS clinical trials, resulting in over eight million longitudinally collected data points.

"… the ALS community and researchers around the world have access to enough data to answer previously unanswerable basic questions, such as, how much does ALS differ between men and women," said Prize4Life's Chief Scientific Officer, Dr. Melanie Leitner. "PRO-ACT will also help to answer more complicated questions, such as, can we identify subgroups of people who may actually have responded to treatment in any of the completed trials."

Although the average life expectancy of an ALS patient is about three years, some people live for decades, while others succumb within months. This lack of predictability makes the design of clinical trials for potential new treatments a long, costly and complex process. One key to better predictability in the future lies in the past - ALS research will move forward when scientists are able to identify the patterns hiding in the millions of data points in PRO-ACT...

"As clinicians who see ALS patients every day, we recognize the huge potential impact of having access to vastly more patient data than anyone has ever had before. As just one example, being able to identify factors that determine the rate of progression in people with ALS will allow us to improve clinical trial design," said Dr. Merit Cudkowicz, Neurology Chief at Massachusetts General Hospital. "Because of PRO-ACT, ALS researchers will be better able to design trials that need fewer participants."

The PRO-ACT database is freely accessible to the global research community for analysis and downloads at www.ALSDatabase.org.

Prize4Life believes that solutions to some of the biggest challenges in medical research will require out-of-the-box thinking, and that some of the most critical discoveries may come from unlikely places.

Founded in 2006 by Avi Kremer, who was diagnosed with ALS at the age of 29, Prize4Life encourages and rewards creative approaches that will yield real results for ALS patients. For more information, visit www.prize4life.org.

Dr. Neta Zach, Scientific Director for Prize4Life, says;

Understanding the variability of the disease can mean a lot for ALS patients going through diagnosis and can lead to a substantial reduction in the cost of clinical trials for ALS treatments. Providing ALS “Big Data” to a global community of researchers speeds up the process while driving down the costs of discovery, which is good news for both the scientific and patient communities we serve.

We are currently assessing ways in which the algorithms could benefit day-to-day clinical practice as well as using them in a clinical trials context. We already know two important and immediate benefits: they will increase the likelihood of clinical trial success, and our experts estimate that these algorithms can reduce the number of patients in a clinical trial by 23%.

Bob says;
Hello? AbbVie? Pfizer? Sanofi? Parkinson’s charities? Doctors? You are either on the train or off the train, but the train is leaving the station. All aboard all who want to be aboard.

Some guy said the earth is not actually flat, it just looks that way.
They burned him at the stake. Now THAT was a peer review.