A New Drug for Parkinson's? Not Quite.

July 17, 2012 / by Jordana Lenon

In a June paper published in PLoS, "A monoclonal antibody-GDNF fusion protein is not neuroprotective and is associated with proliferative pancreatic lesions in parkinsonian monkeys", Marina Emborg, M.D., Ph.D., and colleagues with the Preclinical Parkinson's Research Program at the Wisconsin National Primate Research Center revealed that a novel therapy proposed to treat Parkinson's disease had unexpected, undesirable consequences in rhesus macaques.

The work emphasizes the importance of the nonhuman primate model as a precursor for human clinical trials, even when data in other species, such as mice or rats, looks promising.

"This paper is a classic example of why scientists need to conduct in-depth preclinical research using nonhuman primates," Emborg said. "Publishing these negative results is as critical to moving the entire scientific enterprise forward as is announcing a successful preclinical study."

Emborg, an associate professor of medical physics in the School of Medicine and Public Health, had hoped that the GDNF fusion protein would be in human trials soon as a safe and effective way to treat Parkinson's disease. She has had recent success with moving other therapies into human trials.

"But there is clearly more to understand and further work to do concerning this avenue of treatment," she said.

Nature Reviews Neurology, available by subscription only, wrote a review on the paper entitled, "Primate study highlights health risks of a monoclonal antibody-GDNF fusion protein designed to treat Parkinson disease."