Biomarker Validation Following Sargramostim Treatment in Parkinson's Disease

Prior treatment

• Active treatment with a neuroleptic at time of study entry
• Brain surgery for parkinsonism (DBS, cell implantation, gene therapy)
• Immunostimulatory or immunosuppressive treatment (including amphetamines or systemic corticosteroids) within 90 days
• Anyone who has previously been treated with granulocyte-macrophage colony-stimulating factor (GM-CSF) as an immunomodulatory therapy
• Subjects that have received a flu shot within the past 3 weeks.

Other illness

• Atypical features indicative of a Parkinson-Plus disorder (Progressive Supranuclear Palsy (PSP), Multiple System Atrophy (MSA), Corticobasal Degeneration (CBD)) including cerebellar signs, supranuclear gaze palsy, apraxia and other cortical signs, or prominent autonomic failure
• History of repeated strokes with stepwise progression of parkinsonism
• History of repeated head injury
• History of definite encephalitis
• Hematological malignancy or coagulopathy
• Serious medical illness or co-morbidity that may interfere with participation in the study
• History of an autoimmune disorder or systemic inflammatory disorder deemed significant by physician
• Known hypersensitivity to granulocyte-macrophage colony-stimulating factor (GM-CSF), yeast-derived products
• Anyone who has any illnesses or events that would cause a neurological abnormality, apart from Parkinson's disease.
• Subjects with allergies or sensitivities to yeast products.

Lab values

• Abnormal blood analyses: hematocrit \<30; white blood cell count \>11.5; clinically significant laboratory data (e.g. alanine aminotransferase (ALT) or aspartate aminotransferase (AST) 3x the upper limit of normal (ULN), or any abnormal laboratory value that could interfere with the assessment of safety in the judgment of the investigator; significant abnormalities on the clinical examination, vital signs, and clinical chemistry or hematology results (excluding findings of Parkinson's disease), that may interfere with the study or present a safety risk for the subject as judged by the clinical investigator charged in the care of study participants

Age

• Prominent gait imbalance early in the course (\< 5 years)

Other

• Neuroleptic treatment at time of onset of parkinsonism
• More than one blood relative diagnosed with PD
• Mini-mental state examination score \<26
• Exclusively unilateral parkinsonism for longer than 3 years
• Current lithium treatment
• Individuals with current diagnoses of alcohol or substance abuse/dependence
• Anyone who is not appropriate for participation in this research protocol as deemed by the principal or co-investigator
• Anyone with poor venous access

• Onset of bradykinesia and 1 or both of the following: rest tremor and/or rigidity
• Asymmetric onset of clinical signs
• Progressive motor symptoms
• Age at onset 35-85 years
• Duration of PD symptoms of at least 3 years
• Female subjects must be either:
  • Not pregnant, not breastfeeding, and not planning on becoming pregnant during the study;
  • Not of childbearing potential, defined as one who has been postmenopausal for at least 1 year and with follicle stimulating hormone (FSH) levels in the laboratory defined postmenopausal range, or has been surgically sterilized, or has had a hysterectomy at least 3 months prior to the start of this trial; or
  • If of childbearing potential, must agree to use an effective method of avoiding pregnancy to the end of the trial and must have a negative serum beta-human chorionic gonadotropin (β-HCG) test. Effective methods of avoiding pregnancy are contraceptive methods used consistently and correctly (including implantable contraceptives, injectable contraceptives, oral contraceptives, transdermal contraceptives, intrauterine devices, diaphragm with spermicide, male or female condoms with spermicide, or cervical cap), abstinence, or a sterile sexual partner.
• Must be stage 4 or less according to the Hoehn and Yahr scale