Alzheimer's Matters Blog

New Drug Targets Are Highlight of Alzheimer’s Conference

September 24, 2014

Category: Developing Drugs

Dr. Frank Longo


Every year, the Alzheimer’s Drug Discovery Foundation (ADDF) brings together leading researchers for the only conference focused on Alzheimer's drug discovery and development. Researchers at this year’s conference showcased an array of promising research, including exciting new targets for Alzheimer's drugs and biomarkers that could advance clinical trials.

Drug Targets

Targeting epigenetics to improve memory. The conference kicked off with plenary speaker Li-Huei Tsai of MIT exploring the role that epigenetics plays in the aging brain. “Epigenetics” describes modifications that are made to our DNA that affect the way genes are expressed. If we can alter the way genes are expressed in the brain as we age, we may be able to improve learning and memory and prevent the neurodegeneration that comes with Alzheimer’s disease. Rodin Therapeutics, an ADDF-funded biotechnology company, is developing drugs targeting epigenetic mechanisms that could protect memory.

Using tau as a therapeutic target for Alzheimer’s disease. Tau-related drug discovery is growing, and researchers are examining what aspect of diseased tau (which forms the tangles seen in the brains of Alzheimer’s patients) should be targeted. Michael Gold from UCB Biosciences presented an overview of therapies targeting tau. ADDF-funded Yuma Therapeutics also presented on a strategy to remove tau tangles from the brain by inhibiting HSP90. The company recently received a major grant from the NIH to further work that was initiated with funding from the ADDF.

Investigating drugs that protect neurons from damage and death. If we can prevent brain cells from dying, we can prevent Alzheimer’s disease. ADDF-funded scientist Frank Longo of Stanford University discussed his neuroprotective drug approach—started with funding from the ADDF in 2000, which is now in a phase 2a human clinical trial. Carmela Abraham of Boston University shared her drug, which aims to increase the anti-aging protein Klotho, boost longevity, and protect the brain from Alzheimer’s disease. And Grace Stutzmann of Rosalind Franklin University presented her work developing a drug that targets the ryanodine receptor, which mediates the release of calcium. Dr. Stutzmann has identified new compounds that may be able to restore normal calcium signaling in the brain and prevent the deterioration associated with Alzheimer’s disease.

Exploring the genetics of Alzheimer’s. The ADDF is funding a number of scientists whose research focuses on apolipoprotein E (APOE), the primary carrier of cholesterol in the brain and a major genetic risk factor for Alzheimer’s. But there’s another apoplipoprotein that hasn’t received as much attention: ApoJ, also known as clusterin, is also a genetic risk factor for Alzheimer’s disease. Ling Li of University of Minnesota is testing a small peptide derived from clusterin for its potential to reduce inflammation, improve vasculature, and protect the brain from the insults of Alzheimer’s disease.


Identifying new biomarkers to accelerate drug discovery. In order to successfully develop new therapeutics for Alzheimer’s disease, we must find a way to select the right patients for clinical trials and monitor their responses to treatment. Els Fieremans of NYU has developed a new brain imaging technique to look at white matter changes in the brain. Dr. Fieremans has identified changes that occur very early on in the disease process and continue as the disease progresses. She is now working to combine this novel test with other Alzheimer’s biomarkers to increase sensitivity and specificity for the disease.

The conference gave us the opportunity to check on the progress ADDF-funded scientists have made in their efforts to treat Alzheimer's.