Research

When Cure Alzheimer’s Fund was created in late 2004, our mission was to end the disease by: 

  1. Identifying all risk genes;
  2. Using those genes to reveal underlying disease mechanisms; and
  3. Aggressively pursuing potential therapies based on the knowledge gained from Alzheimer’s genes.

While we have not yet stopped the disease, we have come much closer to the goal line through substantial progress in these three key benchmarks.

What’s Next: Genes to Therapies

The ultimate goal of Genes to Therapies, our next major initiative, is the development of effective interventions at several points in the pathological cascade of Alzheimer’s disease.

Of the currently identified Alzheimer’s genes and candidate genes, 59 are being screened for mutations/functional variants in the Whole Genome Sequencing project. Of these, we will prioritize approximately 15 genes initially that fit three important criteria for immediate and thorough investigation:

  1. High genetic impact or ranking in Alzheimer’s pathology;
  2. “Druggable,” as defined by being in known biological systems and producing proteins that appear to be most readily accessed and modified by typically successful therapeutic agents, such as small molecules or biologicals, e.g., antibodies; and
  3. Affect the most obvious intervention points, which include Abeta/plaque production and clearance, tangle formation/spreading and neuroinflammation.

 

Emerging Consensus: A Model of Alzheimer’s Disease 

Using our genetic discoveries as guideposts, Cure Alzheimer’s Fund has sponsored dozens of studies investigating the central mechanisms of action behind the Alzheimer's disease.

We also have a more thorough understanding of how Alzheimer’s pathology progresses from the earliest to latest stages of the disease. This model of Alzheimer’s allows us to identify three basic strategies for intervention in the process.

More on our disease model and targets for intervention

 

Foundations: The Alzheimer’s Genome Project™

Cure Alzheimer’s Fund has dedicated substantial resources to identifying the full complement of Alzheimer’s genes. The Alzheimer’s Genome Project™ was launched in 2005—and the first phase of this study led to the identification of more than 100 new Alzheimer’s candidate genes. This was the first large-scale, family-based study of the human genome specific to Alzheimer’s disease, and the first to report novel AD genes with statistical significance.

Additionally, a critical step was taken to identify not just which genes are associated with Alzheimer’s risk, but also all of the DNA variants and mutations in those genes that increase or decrease risk for late-onset Alzheimer’s disease. This was accomplished by Whole Genome Sequencing (WGS), which was used to read the entire genome of individuals with Alzheimer’s—all 3 billion base pairs of DNA across all 46 chromosomes. This allowed us to identify nearly 1,000 new genetic mutations in more than 50 different Alzheimer’s and frontotemporal lobar dementia genes, all of which functionally cause or protect against the disease. In identifying these new gene mutations, Dr. Rudolph Tanzi and his team effectively have identified the key biological causal agents that drive Alzheimer’s pathology in the brain.

Latest Research Updates

A Trio of Breakthroughs

Recent months have witnessed three remarkable developments in projects supported by Cure Alzheimer’s Fund.

“Game Changer”: New York Times trumpets “giant step forward” by Tanzi lab

For the first time, and to the astonishment of many of their colleagues, researchers created what they call Alzheimer’s in a Dish — a petri dish with human brain cells that develop the telltale structures of Alzheimer’s disease.

An “Inside-Out View” of Alzheimer’s: Study Offers New Take on Amyloid Hypothesis

A new "alternative amyloid hypothesis” from the lab of Dr. Charles Glabe, at the University of California at Irvine, helps explain precisely how neurons (nerve cells) die in Alzheimer’s disease and how known genetic mutations initiate a chain reaction in this long process. The important new hypothesis was driven by research supported by Cure Alzheimer’s Fund, and has just been being published in the Journal Neurobiology of Disease. Dr.

Are Brain Cell Regeneration Drugs Ready for Prime Time?

A promising first-in-class drug stimulates the creation of new nerve cells in the brains of Alzheimer's mice and will soon be tested in the brains of human patients, thanks to new research by Dr. Sam Gandy, member of Cure Alzheimer's Fund's Research Consortium, at Mount Sinai School of Medicine in New York.

Alzheimer’s and Down Syndrome: Overlap and Opportunity

The world already is very familiar with both Alzheimer’s disease (AD), primarily a disease that occurs in the elderly, and Down syndrome (DS), a genetic condition present at birth. What many don’t realize is that these two conditions also overlap. By age 40, nearly all people born with Down syndrome have begun accruing the plaque and tangle hallmarks of Alzheimer’s. By age 60, most exhibit signs of dementia.