Alzheimer’s Disease Research at NYSCF

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About Alzheimer’s Disease

 

Alzheimer’s Definition

Alzheimer’s is a devastating neurological disease that causes dementia (cognitive decline severe enough to interfere with normal life) and eventually leads to death.

Alzheimer’s Symptoms

Alzheimer’s symptoms manifest differently in each patient. However, most patients become forgetful and confused in early Alzheimer’s disease stages. As the disease advances, patients can experience symptoms such as:

  • long-term memory loss
  • difficulty completing familiar tasks
  • wandering or restlessness
  • mood swings
  • difficulty in speech, thinking, concentrating, and understanding
  • personality changes
  • loss of appetite
  • poor judgment
  • depression
  • aggression & agitation

Alzheimer’s Statistics

  • 4 million people in the United States are living with this neurological disease and 8.2 million more will be diagnosed by 2050.
  • Alzheimer’s disease is the 6th leading cause of death in the United States.
  • 1 in 3 seniors will die with Alzheimer’s or another dementia.
  • Every 65 seconds, someone in the United States develops the disease.
  • In 2018, the direct costs to American society of caring for those with Alzheimer’s will total an estimated $277 billion, including $186 billion in Medicare and Medicaid payments.
  • The cost of treating Alzheimer’s is expected to quadruple, to more than $1 trillion in the U.S. by 2030, if no breakthrough treatments reach the market.

Alzheimer’s Causes

The two hallmarks of Alzheimer’s pathology are amyloid-beta plaques and neuron tangles—structural abnormalities found in the brain. However, we do not know exactly how these abnormalities arise or what role they play in the disease.

Alzheimer’s is also known to be influenced by genetics, with certain genetic variants increasing one’s risk of developing the disease. Using the power of stem cells, our scientists have the ability to study an unprecedentedly large number of genetically diverse cells from people with Alzheimer’s, enabling us to examine how genetics influence disease onset and progression as well as identify new risk-related genes.

Alzheimer’s Cure

There is currently no cure for Alzheimer’s disease. There are a few approved treatments that temporarily slow cognitive decline and address symptoms of the disease such as mood and sleep changes, but there are no therapies that reverse the disease course. Finding effective Alzheimer’s disease treatments and ultimately, an Alzheimer’s disease cure, is one of NYSCF’s central goals. We are working toward achieving this goal by using stem cells to study the actual human cells affected by the disease and discern its underlying causes.

Alzheimer’s Disease Research at NYSCF

At NYSCF, we are developing new Alzheimer’s disease treatment strategies on several fronts:

  1. At the NYSCF Research Institute, we are using our own, powerful robotic system to create stem cells for research. Our NYSCF Global Stem Cell ArrayTM can rapidly, cleanly, and reproducibly create stem cells from skin or blood, and then reprogram these cells to become the brain cells implicated in Alzheimer’s (neurons, microglia, oligodendrocytes, and astrocytes).
  2. Once made, these brain cells allow us to investigate the molecular and cellular causes of Alzheimer’s as it develops so that we may see the actual decline and failure of diseased cells, a model referred to as “disease-in-a-dish.”
  3. We can also use these cells to rapidly and efficiently test drug efficacy and potential toxicity, identifying the safest and most promising compounds to advance to clinical trials.

Watch a panel discussion and discover how stem cells are helping researchers overcome traditional limitations to Alzheimer’s research.

Alzheimer’s Disease Stem Cell News

Publications

Below are select publications outlining recent advancements in Alzheimer’s Disease research from NYSCF scientists.

CRISPR/Cas9-Correctable mutation-related molecular and physiological phenotypes in iPSC-derived Alzheimer’s PSEN2N141I neurons.
Ortiz-Virumbrales M, Moreno CL, Kruglikov I, Marazuela P, Sproul A, Jacob S, Zimmer M, Paull D, Zhang B, Schadt EE, Ehrlich ME, Tanzi RE, Arancio O, Noggle S, Gandy S.
Acta Neuropathologica Communications. 2017. doi: 10.1186/s40478-017-0475-z.

In this study, NYSCF scientists in collaboration with researchers at the Icahn School of Medicine at Mount Sinai refined a technique to turn skin cells from patients with mild cognitive impairment into the brain cells that degenerate first in Alzheimer’s disease.

Characterization and Molecular Profiling of PSEN1 Familial Alzheimer’s Disease iPSC-Derived Neural Progenitors.
Sproul AA, Jacob S, Pre D, Kim SH, Nestor MW, Navarro-Sobrino M, Santa-Maria I, Zimmer M, Aubry S, Steele JW, Kahler DJ, Dranovsky A, Arancio O, Crary JF, Gandy S, Noggle SA.
PLoS One. 2014. doi: 10.1371/journal.pone.0084547.

This study outlines how NYSCF scientists in collaboration with scientists at the Icahn School of Medicine at Mount Sinai successfully generated a stem cell model of familial Alzheimer’s disease and identified genes that contribute to its pathology and onset.

Generation of iPSC lines from archived non-cryoprotected biobanked dura mater.
Sproul AA, Vensand LB, Dusenberry CR, Jacob S, Vonsattel JP, Paull DJ, Shelanski ML, Crary JF, Noggle SA.
Acta Neuropathologica Communications. 2014. doi: 10.1186/2051-5960-2-4

This paper details how NYSCF scientists were able to generate induced pluripotent stem cell lines from bio-banked tissue taken from Alzheimer’s patients.

FAQs

How many people have Alzheimer’s?

5.4 million people in the United States are living with Alzheimer’s and 8.2 million more will be diagnosed by 2050.

Alzheimer’s vs dementia: how are they different?

Alzheimer’s is a form of dementia. Dementia is defined as a decline in mental ability severe enough to interfere with normal life. It is an umbrella term that encompasses aspects of many diseases such as Huntington’s disease, lewy body dementia, and vascular dementia, among others. As a form of dementia, Alzheimer’s is characterized by a progressive loss of memory and cognitive function, often appearing with brain abnormalities such as plaques and tangles. Alzheimer’s accounts for 60-80% of dementia cases.

What are the early onset Alzheimer’s symptoms?

Early onset Alzheimer’s is defined as Alzheimer’s that begins prior to age 65. Early-onset Alzheimer’s symptoms are the same as those for the typical form of the disease and can include:

Poor memory (asking for the same information again and again)

Impaired problem solving

Poor judgement

Changes in mood or personality

Trouble speaking and communicating

Why study Alzheimer’s with stem cells?

We need to study human cells from people with Alzheimer’s in order to find effective therapies—and stem cells allow us to do this. Most laboratory research on Alzheimer’s is conducted in animals, tissues from these animals, or artificial cell lines. Mice do not get Alzheimer’s disease. Not surprisingly, none of the treatments that have looked promising in these animal models have slowed disease progression or cured people with Alzheimer’s, likely because these systems do not sufficiently mimic what occurs in people with the disease. At NYSCF, we turn stem cells into the brain cells affected by Alzheimer’s to study their dysfunction and test drugs.

We have built a comprehensive library of cells from people with and without Alzheimer’s. This resource allows us to look at the mechanisms of cell damage and responses to promising therapy in each of many different forms of Alzheimer’s. We can also compare patients who have Alzheimer’s-related genetic changes but no symptoms to healthy individuals. At NYSCF, this collection of cells, in combination with the NYSCF Global Stem Cell ArrayTM, represent a unique and incredibly powerful tool for increasing our understanding of Alzheimer’s, uncovering safe and effective treatments, and finding cures.