Alzheimer’s disease (AD) is a devastating neurological disease that causes dementia and eventually leads to death. NYSCF undertakes advanced stem cell research to identify the causes and understand the progression of AD to ultimately find a cure.
Undifferentiated Alzheimer's iPS cell
Andrew Sproul, PhD,
NYSCF Postdoctoral Associate
Alzheimer’s symptoms manifest differently in each patient. Commonly, early-stage AD patients become forgetful and confused. As it advances, patients experience long-term memory loss, mood swings, difficulty in speech, and personality changes. Neurological changes affect vital functions such as swallowing, balance, and coordination.
Difficult to definitively diagnose, Alzheimer’s disease symptoms are often mistaken for age-related memory loss or other forms of dementia. If suspected in a patient, AD is generally diagnosed by neurological exam and brain imaging; however, only post-mortem autopsy can accurately confirm AD.
Alzheimer’s disease is the sixth leading cause of death in the United States, and 5.4 million Americans are living with this disease. While symptoms can be lessened or slowed, there is no cure.
With NYSCF support, scientists have made progress in understanding the causes and discovering therapeutic candidates for Alzheimer’s disease.
Learn more about stem cells:
NYSCF Lab Director Dr. Scott Noggle
How can stem cell research help us find better treatments and cures for Alzheimer’s disease?
Stem cells provide a living window onto Alzheimer’s disease. Researchers at the NYSCF Research Institute derive the actual forebrain neurons implicated in this disease from Alzheimer’s patients’ skin samples. These cells mature, get sick, and die off in a dish as in a patient. We can, for the first time, scrutinize what goes wrong on a cellular level that leads to what goes wrong in a patient.
Dr. Scott Noggle, the Director of the NYSCF Laboratory and the NYSCF – Charles Evans Senior Research Fellow for Alzheimer’s Disease, leads the Alzheimer’s disease team. He, with fellow NYSCF scientists, model AD in a dish with patient-specific stem cells. Through advanced techniques, they revert patients’ skin samples back into an embryonic-like state, producing induced pluripotent stem (iPS) cells, which can become any of the cell types implicated in AD. Coupled with the medical histories of patients, these cells serve as an incredibly powerful tool to increase our understanding and to find treatments.
The NYSCF Alzheimer's disease team is developing a massive collection of iPS cell lines on The NYSCF Global Stem Cell Array (Array) from hundreds of Alzheimer's patients’ skin samples. The Array fully automates the generation of standardized iPS cells that can be used to study Alzheimer’s and, importantly, for chemical compound screens to find new drugs.
In Phase II of this project, NYSCF researchers are using these iPS cells to create Alzheimer's neurons for drug testing to identify a candidate that not only arrest the progression of Alzheimer’s but may also treat the symptoms. The goal of the drug discovery phase is to generate lead compounds that pharmaceutical partners can develop into clinically viable drugs.
Granted that Alzheimer’s disease may only be definitively diagnosed post-mortem, Dr. Noggle with his team has developed a novel protocol to produce AD cell models from cadaveric brain tissue. The group, by “reanimating” these cells, may compare the stem cell-derived neurons as they experience AD to the neurons of the AD patient. He announced this new research tool in a TEDU talk at the 2013 TED Conference.