UNMC conducts clinical trial on Leukine to test unique immune therapy in people with Parkinson's

The Nebraska Neuroscience Alliance at the University of Nebraska Medical Center, is embarking on a translational clinical trial using Leukine, a drug used to boost the immune system in cancer patients, to test a unique immune therapy in people with Parkinson's disease.

The NNA, formed in 2011, unites three of UNMC's top neuroscience programs - the departments of neurological sciences and pharmacology and experimental neuroscience as well as the Munroe-Meyer Institute.

Parkinson's disease is caused by the loss of neurons that produce dopamine, a nerve signaling chemical which controls movement and balance. The Parkinson's Disease Foundation estimates that about 1 million people in the United States and more than 4 million people worldwide have the disease.

Degeneration and loss of these dopamine-producing neurons typically occur after age 60, and it is estimated that one person in 20 over the age of 80 has Parkinson's.

Neurodegeneration occurs when a normal protein, called alpha synuclein, clumps, changes shape, and accumulates in the brain. The accumulation of protein clumps alert the immune system, which launches an attack, causing inflammation and eventual destruction of dopamine-producing nerve cells.

"The project is a collaboration between neurologists and neuroscientists," said Dr. Howard Gendelman, principal investigator and chair of the department of Pharmacology and Experimental Neuroscience at UNMC.

Specifically , "the study will determine whether the drug Leukine can transform the immune system in Parkinson's disease from one that causes harm to the brain to one that protects it, elicits nerve cell repair and ultimately affects disease symptoms," said R. Lee Mosley, Ph.D., a co-principal investigator and associate professor in the department of pharmacology and experimental neuroscience.

In a year-long, double-blind clinical trial, set to begin this fall, 16 patients will be monitored using magnetoencephalography (MEG) imaging to pinpoint those areas of the brain affected by the disease and determine if Leukine works.

This is the first time MEG has been used to monitor Parkinson's disease and the effects of a treatment, said Tony Wilson, Ph.D., an assistant professor in the UNMC Department of Pharmacology and Experimental Neuroscience, who will be working with the assembled team of neurologists, nurses, statisticians and researchers.

If this therapy proves to be useful, Dr. Gendelman said, it sets the stage to launch yet more comprehensive studies looking at the effectiveness of other potential vaccine and immune changing therapies.

"Since current therapies have not led to a breakthrough it is certainly worthwhile to consider new approaches," said John Bertoni, M.D., Ph.D., a professor of neurological sciences in the College of Medicine and director of the Parkinson's clinic at UNMC. "This is the first step and we are cautiously optimistic but we will need to analyze the information we gain very carefully."

Gendelman and his team have, for more than 12 years, worked to not only understand Parkinson's disease progression but also to slow it through immune therapy. The work would not have been possible without a vigorous collaboration between neurologists, statisticians, psychologists and neuroscientists.