New Parkinson's treatment described as potential 'game-changer'

September 28, 2017  By Bryce Airgood

Neurologist and professor Thomas Foltynie

GRAND RAPIDS, MI - A new potential treatment to slow the progression of Parkinson's disease is being discussed by specialists at Van Andel Institute this week.

This potential breakthrough stems from a recent clinical trial led by neurologist and professor Thomas Foltynie in the United Kingdom. In the course of a 48-week period, Parkinson's patients who received the drug exenatide retained more motor function than patients who received a placebo.

Although more research needs to be done, professor and Director of the Center for Neurodegenerative Science Patrik Brundin described the trial as a "total game-changer" if the research holds up.

"It will be the first medication in history in Parkinson's that will change the course of the disease," Brundin said.

Parkinson's disease specialists and advocates from around the world gathered in Grand Rapids for the Grand Challenges in Parkinson's Disease symposium. This scientific event, which runs through Thursday, highlights the latest breakthroughs in Parkinson's disease and this year included discussion of Foltynie's study.

Foltynie described the clinical trial as a double-blind randomized placebo controlled trial. Exenatide, a licensed drug for treating Type 2 diabetes, was given to some patients with moderate severity Parkinson's disease while other patients were given a placebo.

The study used the Unified Parkinson's Disease Rating Scale to track symptoms of Parkinson's disease such as slowness, stiffness and tremors. They saw patients every 12 weeks over the course of the trial and would give them an overnight period without the Parkinson's drug.

A year into the study, patients in the placebo group had deteriorated about two or three points on the scale, which is a typical progression of the disease, Foltynie said. However, patients who had received exenatide had remained entirely stable and their symptoms had not worsened at all. Even after the patients stopped using the drug for a 12-week period and it was out of their system, symptoms still hadn't worsened, he added..

Foltynie said the results were quite exciting but they need to be reproduced in a second, larger cohort of patients to make sure findings are consistent and robust.

Neurologist, physician and specialist in movement disorders Michele Tagliati aims to do just that and is currently running a similar study to Foltynie's in Los Angeles. Tagliati's study is also randomizing patients with Parkinson's disease to receive an active compound or placebo for 52 weeks.

The difference is Tagliati is using a different compound that is more effective at treating diabetes than the one Foltynie used, so they are hoping as a result it will be better at treating Parkinson's disease.

Although there are no results yet from his study as it is in progress, Tagliati said duplicating Foltynie's study is very important and brings the test a step further.

"With parallel research, parallel tools, we can confirm that what we're actually doing is to improve the brain function of these patients," Tagliati said.

Using diabetes medication to try and treat Parkinson's disease -- in this case, exenatide -- is a process called "repurposing" and can be very useful by saving scientists years of research and money.

"In its purest form, drug repurposing means you take a drug that has been tested in thousands of other patients with other diseases and it's gone through all the safety testing and you know exactly that you can give it to humans in certain doses and so on," Brundin said.

The specialists agreed repurposing drugs saves time and money and also helps stimulate new thoughts. Parkinson's disease was traditionally seen as a motor disorder, but in the past 15 years scientists have discovered a set of up to 30 nonmotor symptoms as well. These symptoms can include loss of smell, constipation, mood disorders, and many more.

"It's a much more complex disease than the textbook I read in medical school in the 1980s was telling us," Brundin said.

Focusing on the nonmotor symptoms rather than the motor symptoms can lead to a better understanding of Parkinson's disease.

"Traditionally neurologists have thought of the brain inside the skull as being 'in the box,'" Foltynie said. "Parkinson's neurologists are thinking outside the box."

The Grand Challenges in Parkinson's Disease event will be running parallel to the Rallying to the Challenge, which is focused more on advocacy of patients with Parkinson's disease. Both are featured at the Van Andel Institute this week.

http://www.mlive.com/news/grand-rapids/index.ssf/2017/09/parkinsons_specialists_gather.html