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Pilot Leukine® Trial Update

Our previous studies showed that the FDA-approved drug Leukine® quickly reduces amyloid deposits responsible for Alzheimer’s plaques and reverses cognitive impairment in animal models of Alzheimer’s disease. Our Pilot Leukine® Safety Trial is aimed at establishing whether the drug is safe in individuals with mild-to-moderate Alzheimer’s disease. In this study, subjects receive either Leukine® or placebo for 5 days/week for 3 weeks. So far, 32 subjects have completed this 3-week trial, with 13 subjects receiving Leukine® and 19 subjects receiving placebo. Our analyses to date show that Leukine® treatment is safe, since no negative impacts have been reported on Leukine® treated subjects, which is in contrast to the brain swelling and hemorrhaging caused by other candidate Alzheimer’s disease drugs tested recently in humans. Also, Leukine® treated subjects showed significant improvements in cognition at the end of treatment based on two different tests: 1) the MMSE (Mini-Mental State Exam) that measures changes in thinking and memory, and 2) the ADL (Activities of Daily Living) that measures ability to perform basic activities needed for independent living. This unexpected finding is exciting because it provides an early indication that Leukine® may be beneficial in treating Alzheimer’s disease.

Thanks to a generous gift from the Sprout Foundation, we were able to add amyloid PET imaging to the final 20 out of a total 40 subjects in the trial. Each of the remaining 20 subjects will undergo two PET imaging studies — one before treatment and one at 45 days after treatment — for a total of 40 PET images. Adding the additional information from these amyloid PET images will give us stronger data on whether or not the Leukine® decreases amyloid deposits in the human brain. To date, we have obtained seven out of the 40 total amyloid PET images that will be needed to assess whether Leukine® reduces amyloid deposits. We expect that the next group of 10 subjects will have completed their treatment by the end of July, thus allowing the next data safety meeting and assessment.

Based on results to date, the RMADC was awarded an Alzheimer’s Association grant to cover one-fourth of a longer Leukine® Safety/Efficacy Trial. We will still need to raise approximately $3M to cover the costs of the full trial. In this longer trial, 42 individuals will have amyloid-PET imaging at the start of the study, and then two-thirds of them will receive daily injections of Leukine® and one-third of them will receive placebo 5 days/week for 6 months, after which they will have a second amyloid-PET scan. Our goal is to determine whether Leukine® clears amyloid plaques from the brain and whether or not it improves cognitive function in individuals with mild-to-moderate Alzheimer’s disease. We have FDA clearance to run this longer trial, and we will soon be submitting the trial for clearance from Colorado regulators. We aim to launch this trial in 2017 with the hope of moving this potential new Alzheimer’s therapy closer to market as quickly as possible.