Changing the course: Parkinson’s disease research in 2016

By Patrik Brundin, M.D., Ph.D.
Director, Center for Neurodegenerative Science

The time has come to shift the paradigm on Parkinson’s disease.

Never before have we had the scientific capacity and the breadth of knowledge about Parkinson’s that we do now. At the same time, the level of collaboration between scientists, clinicians, people with Parkinson’s, advocates and industry is nothing short of breath-taking—individuals from all over the world, working together to beat Parkinson’s.

The shift can’t come soon enough. There have been few major breakthroughs in therapeutic development for Parkinson’s in the last 50 years, with the exception of the gold standard therapy levodopa, and deep brain stimulation surgery.  While these therapies mitigate symptoms, they do not correct the underlying cause of Parkinson’s nor do they repair damage.

But there is hope on the horizon. Through individual and large-scale collaborative efforts, we are working to change the standard of care for Parkinson’s and to find ways to drastically improve quality of life. At VARI, we focus on three points:

Biomarker discovery

One of the biggest challenges in Parkinson’s is also one of the most basic—how do we accurately diagnose and define it?

Unlike many other diseases, which may be diagnosed with a blood test or a biopsy, there are no definitive tests for Parkinson’s. While this certainly presents a challenge for scientists and clinicians, it is most problematic for patients who deserve a cut-and-dry explanation of what’s causing their symptoms.

This is where biomarkers come in.

As the name implies, a biomarker is a biological characteristic—for example, altered levels of a protein, a hormone, or a genetic signature—that can be objectively measured to indicate a normal process or the presence of a disease. In fact, it is a safe bet that most people are aware of at least one or two biomarkers— for example, using body temperature (fever) as an indication of infection or using the levels of certain hormones to detect pregnancy.

Finding new biomarkers will give us further insights into the disease’s underlying mechanisms and help us stratify Parkinson’s cases into subtypes, which aids in choosing the best therapy for a particular patient. Importantly, it may also allow us to definitively diagnose Parkinson’s before the onset of symptoms, giving clinicians and patients a solid answer.

Stopping disease progression

Parkinson’s marches through the brain slowly, damaging the connections between cells and eventually killing dopamine-producing cells, leading to the onset of the disease’s hallmark motor symptoms—rigidity, tremor and slowness of movement. Slowing and, ultimately, halting this process is a vast unmet need.

Some of these therapies may be hiding in plain sight. Through the Linked Clinical Trials initiative, which is spearheaded by The Cure Parkinson’s Trust in collaboration with Van Andel Research Institute, we are investigating drugs approved to treat other conditions as potential therapies for Parkinson’s. The drugs on our list have all demonstrated promising neuroprotective effects in the lab and have already made it through a gauntlet of safety trials, reducing the time and cost required to translate them into potential Parkinson’s therapies. Unlike current treatments, which only impact symptoms, these repurposed therapies hold promise for actually changing the course of the disease. (Read more about one of these drugs—ambroxol—and a recently launched clinical trial here).

Brain repair

Stopping Parkinson’s in its tracks isn’t enough—we also must find ways to repair the damage and to restore the function lost along with dopamine cells in the brain. We are in the beginning stages of building this research program at the Institute in collaboration with colleagues around the world and are hopeful that repairing damage brought on by Parkinson’s will one day be a reality.

Going farther, together

While each of these goals is important on its own, the collective impact of achieving them means a completely new way to view and treat Parkinson’s. We envision a future in which biomarkers will be used to catch the disease before extensive damage has occurred, a future in which that person may be treated with a disease-modifying therapy to prolong symptom-free years and quality of life, and a future in which damage may be repaired. We can realize this future through collaborative efforts between scientists, clinicians, industry and, importantly, the Parkinson’s community. The shift is coming, and it will take all of us to make it happen.

About Parkinson’s

  • The hallmark motor symptoms of Parkinson’s disease include tremor, rigidity and a progressive slowing of movement. Non-motor symptoms, such as loss of sense of smell, gastrointestinal issues, cognitive deficits, depression and sleep disorders, may occur years before diagnosis.
  • Approximately one million people in the U.S. and seven to 10 million people around the world have Parkinson’s disease.
  • Motor symptoms are caused by the loss of dopamine-producing cells in the brain. Dopamine a chemical called a neurotransmitter that is responsible for smooth movement, among other functions.