ADDF Announces New Research Investments Supporting Alzheimer’s Drug Development and Biomarker Research

The Alzheimer’s Drug Discovery Foundation (ADDF) has announced six new research investments totaling nearly $10.8 million supporting innovative clinical trials, as well as novel drug discovery and biomarker programs for Alzheimer’s disease and related dementias. These programs range from a phase 1 trial modulating the immune system to decrease inflammation, to a small molecule drug that promotes clearance of toxic materials in the brain that can contribute to Alzheimer’s disease.

“With these new investments, the ADDF continues its role as a leader in supporting promising programs that target all of the novel biological factors that contribute to Alzheimer’s,” said Dr. Howard Fillit, ADDF Co-Founder and Chief Science Officer. “Developing treatments to address all of the underlying factors that contribute to Alzheimer’s is essential so that physicians can one day combine them in precision medicine approaches for personalized patient care.”

CLINICAL TRIALS

Tom Megerian, MD, PhD, Cognito Therapeutics
Cognito Therapeutics - A Novel Digital Therapeutic for Neurodegenerative Diseases
$4,556,150

Synchronized audio-visual stimulation delivered through a wearable device can evoke changes in the brain that may protect neurons and improve the course of Alzheimer’s disease. This phase 3 trial by Dr. Megerian and the team at Cognito Therapeutics will test the effects of one hour per day of audio-visual brain stimulation on patients with mild-to-moderate Alzheimer’s at 40 sites across the United States. The researchers will monitor several potential biomarkers to assess its effects, including blood and spinal fluid biomarkers, amyloid PET scans, MRIs and EEG neuroimaging.

Sean Joseph, PhD, The Scripps Research Institute
Long-Acting GM-CSF Fusion Protein (PDM608) for the Treatment of Alzheimer’s Disease
$3,000,000

Dr. Joseph and the team at Scripps have developed a novel drug called PDM608, designed to spur a patient’s own immune system to decrease inflammation in the nervous system. Emerging evidence suggests that decreasing inflammation can be a powerful approach to modifying the course of Alzheimer’s disease. After showing a robust and durable effect of PDM608 in animals, the team at Scripps is developing a phase 1 study to provide critical information regarding its safety, tolerability, and pharmacodynamic effects in healthy volunteers to inform the design and maximize chances of a successful clinical trial in patients with Alzheimer’s.

Hussein Yassine, MD, University of Southern California
Effect of Early Supplementation with High Dose DHA on Cognitive Outcomes in APOE4 Carriers: "PreventE4 Trial"
$250,483 of add-on funding

Carrying the APOE4 allele is the strongest genetic risk factor for developing late-onset Alzheimer’s disease. Animal studies suggest that early, high doses of docosahexaenoic acid (DHA, an omega-3 fatty acid) can prevent cognitive decline. In this randomized, controlled two-year clinical trial, the team at USC is testing whether supplementation with high doses of DHA can slow cognitive decline in carriers of the APOE4 allele when started before the onset of cognitive impairment.

DRUG DISCOVERY

Christiane Wrann, DVM, PhD, Massachusetts General Hospital
Leveraging Exercise to Improve Cognitive Function in Alzheimer’s Disease
$600,000

Studies suggest that physical activity, especially endurance exercise, can improve cognitive function in part by reducing neuroinflammation. The team at Mass General has shown that the hormone irisin, which is secreted from muscles during exercise, may play an important role in improving cognitive function. Their current work is evaluating irisin as a treatment for Alzheimer’s in mice. If proven successful, irisin would be the first drug to reproduce the neuroprotective effects of exercise.

Michael Parker, DPhil, St. Vincent's Institute of Medical Research
Development of Small Molecule Inhibitors of a Microglia Receptor as Treatments for Alzheimer’s Disease
$567,653

The team at St. Vincent’s is developing drugs to help clear toxic materials in the brain that are associated with Alzheimer's disease. The drugs target a receptor on the surface of immune cells in the brain that acts like a “handbrake,” stopping the immune system from clearing brain toxins. By binding to this receptor, the drugs aim to release the handbrake so the immune cells can do their work.

BIOMARKERS

John Sninsky, PhD, Molecular Stethoscope, Inc.
Development of Cell Free Messenger RNA-Based Non-Invasive Diagnostic Biomarker for Alzheimer's Disease
$1,820,426

This study by Dr. Sninsky and the team at Molecular Stethoscope aims to establish and validate a blood test measuring circulating levels cell free-mRNA (cf-mRNA) as a way to evaluate molecular changes in the brain that are specific to Alzheimer’s disease. In this next phase, the team will expand on their earlier proof-of-concept work by rigorously testing their blood test in well-characterized groups of Alzheimer’s patients.