Alzheimer’s Stem Cells are Uncovering Disease Mechanisms, and NYSCF is Making Them Widely Available to the Research Community

One of the great advantages of stem cells is that they can be used to make personalized models of disease — they let us study exactly how the human cells affected by devastating conditions behave, pointing us toward new therapeutic options. NYSCF regularly provides high-quality stem cells to other laboratories, allowing for in-depth study of diseases for which the causes remain mysterious, such as Alzheimer’s. In partnership with longtime NYSCF collaborator Marc Tessier-Lavigne, PhD, President of Stanford University, the NYSCF Research Institute will soon make stem cells carrying Alzheimer’s-associated mutations available to the wider research community.

Dr. Tessier-Lavigne’s laboratory recently published a study in Neuron examining neurons derived from stem cells that have been engineered to carry genetic mutations that increase risk for Alzheimer’s disease. The findings reveal new ways in which these mutations impact a cell’s ability to transport proteins, which often goes awry in Alzheimer’s, via altered processing of APP (the precursor to the plaques that build up in the brains of patients). Understanding more about how these dysfunctions contribute to Alzheimer’s will help scientists develop treatments that target them.

The new findings build on an ongoing collaboration between NYSCF and Dr. Tessier-Lavigne aimed at using stem cells to uncover Alzheimer’s mechanisms (initial results from this partnership were published in Nature in 2016). The NYSCF Research Institute will now generate and distribute the stem cells used in the current study to scientists around the world.

“This is an incredible resource,” noted Celeste Karch of Washington University in St. Louis in an article from Alzforum. “These tools are already revealing critical insights into disease mechanisms that have implications for the forms of APP that are being targeted therapeutically.”

The stem-cell-derived neurons are genetically altered to contain mutations in the genes PSEN1 and APP associated with familial Alzheimer’s disease, a form that can show onset as early as a patient’s 30s or 40s. Distributing stem cells that carry these mutations to labs around the world will accelerate studies of Alzheimer’s, helping us understand more about how genetics influences the disease and uncovering new targets for treatment development, like those identified by Dr. Tessier-Lavigne’s study.

“Democratizing access to stem cell resources is a critical part of NYSCF’s mission to advance disease research,” remarked NYSCF CEO Susan Solomon, JD. “We are thrilled to be partnering with Dr. Tessier-Lavigne’s lab to make these stem cell lines widely available and look forward to the insights they will provide into the causes of Alzheimer’s.”