An Overview of Parkinson’s Disease Research: Questions and Answers
Sunday, 6th October 2013

On Tuesday, February 19, The Michael J. Fox Foundation (MJFF) hosted the first event of the year in its Hot Topics in Parkinson’s disease (PD) Research Seminar Series: “An Overview of Parkinson’s Disease Research.” The Webinar, which is now available on demand, was hosted by MJFF Contributing Editor Dave Iverson, and panellists included MJFF CEO Todd Sherer, PhD, Bernard Ravina, MD, of Biogen Idec, and Irene Hegeman Richard, MD, of the University of Rochester.

Sherer, Ravina and Richard answered some participant questions live, but time ran out before they could get to everyone. As promised, we answer a few more here:

How will the newly announced brain mapping initiative from the Obama administration aid Parkinson’s research?

The short answer is that it remains to be seen. Certainly, any investment in learning more about how the brain works is a good thing for people with PD. But we’re yet to be sure that the planned initiative will definitely happen.

In the way that the Human Genome Project famously provided researchers with a sort of owner’s manual to our DNA, it’s possible that the “Brain Activity Map” could similarly provide critical information regarding the even more complex structure of the brain. Better understanding the chemical processes taking place in our brain could help researchers to, in turn, learn how these processes are altered by the presence of a neurological disease. This could go a long way toward developing drugs to counteract these harmful changes in brain chemistry.

But mapping the brain won’t be easy, and will likely take many years, and lots of research dollars, to complete.

It was said during the webinar that Deep Brain Stimulation (DBS) can be done earlier on rather than later, but it was not said why. Are there new facts available about DBS?

To date, most research has pointed to DBS as an option for those with later stage PD. But a new study published this past month suggested that DBS is effective against the “early motor complications of PD.” This is the first study to show that surgical treatment might be proposed in an earlier-than-anticipated phase of PD.

A couple of caveats from MJFF Associate Director of Research Programs Maurizio Facheris, MD, MSc, who was quoted in a Forbes article referencing the study:

“Treating ‘earlier motor complications’ doesn’t necessarily mean treating younger patients (earlier refers to numbers of years with a disease, whereas younger is of course in reference to actual age).”

“Also, we’re still far from understanding if DBS has any disease-modifying effects; for now it’s still considered a purely symptomatic therapy. And DBS is not for everyone: It’s important to discuss the procedure with a healthcare provider to discuss eligibility and the pros and cons of having brain surgery.”

The DBS field is one of increasing potential. Learn more about upcoming developments from DBS pioneer Andres Lozano, MD.

I know that the Neupro patch has returned to the market in the past year. Can you tell me more about how this drug works?

Neupro is a dopamine agonist, or a drug that mimics how dopamine operates in the brain, supplementing function that has been lost as dopamine-producing neurons die during the course of PD. Neupro, and other extended-release dopamine agonists (such as Mirapex ER), maintain as constant a level of the drug as possible throughout the course of the day. Clinical studies have shown that taking Neupro reduces patient “off-time,” which is when the effects of the medication they’re taking wear off.

In addition, the patch is also now approved to treat Restless Leg Syndrome (RLS), an uncontrollable urge to move one’s legs when at rest. Extended release treatments for PD such as Neupro could have important implications for the many people with PD who experience difficulty sleeping. In addition to complications from RLS that often make it hard to sleep, rigidity can make it equally difficult to get some rest. Unable to toss and turn, many spend countless waking hours during the night. A more consistent treatment for PD such as Neupro could help to alleviate this stress.

You can learn more about drugs used to treat PD on our Web site. The Michael J. Fox Foundation does not endorse any one particular therapy, and we encourage all patients to discuss therapeutic options with their treating physicians.

I’ve recently heard some about the protein tau as a potential target for Alzheimer’s disease but was surprised to learn it may have a link to PD as well. What do we know about tau’s role in Parkinson’s?

Last fall, the Wall Street Journal explained tau’s role in Alzheimer’s: “Like all of the body’s proteins, tau has a normal, helpful function—working inside neurons to help stabilize the fibres that connect nerve cells. But when it misfires, tau can clump together to form harmful tangles that kill brain cells.” Research is moving forward to find drugs that might un-tangle these twisted brain fibres, which could, in turn, slow the disease.

In recent years, tau has also become a point of focus for PD as well. In addition to being genetically linked to a higher risk of developing PD, scientists from the Parkinson’s Progression Markers Initiative (PPMI) are looking into whether levels of tau in a person’s cerebrospinal fluid (CSF) might also provide a biomarker for who might be more likely to go on to develop Parkinson’s. Very early studies suggest that lower levels of tau in CSF may, in fact, be representative of PD. (Watch this episode of the Parkinson’s Minute to learn more about the search for a biomarker that is taking place in PPMI).

But there’s an interesting discrepancy between tau’s apparent role in AD and PD, says Mark Frasier, PhD, MJFF’s vice president of research programs. In AD, there is a clear pathological marker, or clear physiological relationship of disease related to tau – those tangles in the brain mentioned above. But as of yet, there’s been no sign of a genetic mutation in tau that might lead to the disease.

In PD, it’s the opposite: While there have been genetic studies linking changes in tau to PD, there has been little evidence of tau pathology (those tangles seen in AD) in most PD brains. For this reason, most current work into tau and PD is to determine if the protein might one day provide a biomarker for the disease.

The PD community will watch closely to learn more about the effectiveness of therapeutic approaches to tau in Alzheimer’s, before moving forward with targeting the protein for potential Parkinson’s drugs.

What can people with Parkinson’s do to help advance PD research?

The good news is, there are many ways to get involved. In addition to donating to Research, people with Parkinson’s and their loved ones can participate in and support fundraising activities.

There is also a real need for people with Parkinson’s to participate in clinical research. Clinical trials are the final phase in the lengthy drug development process, but a persistent shortage of volunteers for clinical trials slows scientific progress. Patients pay the ultimate price for under-enrollment in clinical research, facing few new treatment options.

MJFF has launched a Web-based matching tool called Fox Trial Finder to address this shortage of trial participants to connect willing volunteers with the clinical trials that need them. Sign up today to get started in finding a trial near you!