The purpose of this study is to determine if Leukine (sargramostim) can be safely
administered to Parkinson's disease patients for an extended period of time (56 days) and
restore immune deficits seen in Parkinson's patients compared to controls. The development of
magnetoencephalography (MEG) as a monitoring tool for PD will also be explored. At enrollment
and repeating again at two 4-week intervals, whole blood from PD patients and controls will
be obtained for analyses and the results will be used to calculate immune response profiles
as a baseline for comparison after drug treatment. Physical examinations and motor
assessments will also be performed on PD patients. After the 8-week baseline data collection,
control participation will end and drug treatment of PD patients will begin. PD patients will
be randomized, and half will receive drug and half will receive placebo. Leukine at a dosage
of 6 µg/kg or saline as placebo will be administered by subcutaneous injection daily for 56
days (8 weeks). During drug treatment, PD patients will be monitored every two weeks by
physical examinations, motor assessments, and blood analyses. As follow-up, four weeks after
drug administration has stopped, subjects will again have physical examinations, motor
assessments, and blood analyses. MEG will be performed on PD patients and controls at the
start of drug treatment, and on PD patients at the end of the drug treatment period and 4
weeks after drug is stopped. In addtion, at the second cohort of 8 PD subjects, we will
evaluate the potential Leukine-induced motor control and mobility improvements. Also, levels
of the neurotransmitters glutamate, glutamine, serotonin, acetylcholine, GABA, norepinephrine
and epinephrine in serum/plasma will be analyzed to correlate with changes in motor function
and drug treatment.
Phase:
Phase 1
Details
Lead Sponsor:
Howard Gendelman, MD
Collaborators:
National Institute of Neurological Disorders and Stroke (NINDS) Nebraska Neuroscience Alliance Sanofi UNeMed