Saturday, August 29, 2015

Looking for Answers

We all hope some day to be able to stop Parkinson's in its tracks. We want to stop Parkinson's forever, and even reverse the changes in the brain that result in Parkinson's, despite the serious challenges there are in making changes to the wiring of the brain. In my job, I have funded some of the work to make a pill that would stop Parkinson's, including work on the Parkin protein and alpha synuclein.

As I do this work, I am left with a question: what if we already know secrets that will change the course of Parkinson's, or they are right under our noses, and we haven't noticed?  Working with a smart new neuroscience Ph.D., I did some research to identify the impact of exercise on Parkinson's disease.  The results were dramatic.  Regular exercise -- just 2.5 hours a week -- seemed to turn back the clock on Parkinson's by about a year.  We don't have evidence that exercise changes what is going on in neurons in your brain, but we do think that exercise helps the cells in your brain to fight Parkinson's.

That's not it.  In another study, working with the Mayo Clinic's Anhar Hassan, we showed that there are many patients who out-perform the average, living with Parkinson's for 20 years or more.  While some in the PD20 cohort exhibited advanced disease, there did seem to be a group of what we might call "elite controllers" of Parkinson's disease.  These patients have been treated for years by leading experts at top academic medical centers, we think that they really have Parkinson's and are not misdiagnosed.

What if the secret to controlling Parkinson's disease isn't in a test tube in a lab somewhere but is, in fact, floating in the cells of one of these long-term thrivers?  I am hoping to launch a study to determine just this question: are there people whose cells are specially designed to fight Parkinson's, or are their cells deficient in some way that prevents progression?  If we could answer this question, we could then challenge our pharmaceutical company partners to figure out how to deliver this benefit to everyone with Parkinson's.

The first cure for AIDS was discovered in the bone marrow of a man who was immune to HIV.  We can cure "bubble boy disease" with tissue from unaffected people.  Small pox was eradicated using a virus found in nature.  We believe that many of the clues to Parkinson's in humans will be found by studying people with Parkinson's.  Many of the hardest questions for scientists are found to have been solved by nature, if we just knew where to look.

PS: many totally brilliant research findings are coming from biologists and researchers who are looking at molecular, cellular, or animal models of Parkinson's.  However, one of the most exciting findings of the past decade was a result of insight from studying patients who had a particularly bad form of Parkinson's, as I detailed earlier.  Studying people in the clinic and studying drugs in the lab form a virtuous circle.

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