Study of the Safety & Efficacy of Leukine® in the Treatment of Alzheimer's Disease
Study of the Safety & Efficacy of Leukine® in the Treatment of Alzheimer's Disease
A medicine that is FDA-approved for bone marrow stimulation (called Leukine) will be tested
 for its ability to be tolerated by Alzheimer's disease patients and potentially to improve
 their memory.
Preliminary preclinical results demonstrated that GM-CSF (Granulocyte macrophage
 colony-stimulating factor, e.g. Leukine®/Sargramostim) rapidly reduced cerebral amyloid
 deposition and completely reversed memory deficits in transgenic mouse models of Alzheimer's
 Disease (AD). To assess the efficacy of GM-CSF in humans, the investigators performed a
 retrospective analysis of a cognition study of human patients undergoing hematopoietic cell
 transplantation for cancer and who garner cognitive impairments from the chemotherapy or
 irradiation. In the patients that received a colony-stimulating factor (CSF) to stimulate the
 bone marrow and recover immune system function, the investigators found that those who
 received GM-CSF (Leukine®/Sargramostim) plus G-CSF (Filigrastim) significantly improved in
 cognitive function as compared to those who received G-CSF alone. These findings combined
 with over two decades of accrued safety data using recombinant human GM-CSF,
 Leukine®/Sargramostim, in elderly leukopenic patients, suggested that Leukine® should be
 tested as a treatment to reverse cerebral amyloid pathology and cognitive impairment in AD.
Inclusion Criteria:

 1. age 55 to 85 years;

 2. should have a mild-to-moderate AD diagnosis (MMSE 10-26 inclusive);

 3. should have evidence of elevated cortical amyloid by PET using florbetapir F18
 (Amyvid) [i.e. a positive scan], assessed qualitatively according to the Amyvid
 product label.

 4. if on anti-dementia treatment should be on stable treatment for at least 2 months
 (i.e. cholinesterase inhibitor and/or Memantine or Axona);

 5. stable on all other medications for at least 30 days prior to screen;

 6. should be fluent in English;

 7. should be physically able to participate by medical history, clinical exam and tests;

 8. should have a study partner to accompany them to scheduled visits.

 Exclusion Criteria:

 1. clinically relevant arrhythmias;

 2. a resting pulse less than 50;

 3. active cancer other than non-melanoma skin cancers;

 4. use of another investigatory drug within 2 months of screening;

 5. significant stroke or head trauma by history or MRI;

 6. contraindication for having a MRI;

 7. diagnostic and Statistical Manual of Mental Disorders-IV criteria for a current major
 psychiatric disorder;

 8. sensitivity to yeast or yeast products;

 9. impaired kidney function as measured by a Glomerular Filtration Rate less than 60
 milliliters/min;

 10. preexisting fluid retention, pulmonary infiltrates, or congestive heart failure;

 11. history of moderate-to-severe lung disease;

 12. history of moderate-to-severe liver disease;

 13. pregnant women, or any women who feel they are likely to become pregnant during the
 study;

 14. prisoners.