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Improving the prevention and treatment of Alzheimers disease
Improving the prevention and treatment of Alzheimer's disease
This is due, in part, to the inherent complexity of the human brain and to the drug discovery process, much of which relies on experiments using animal cell lines or non-neuronal, human cells. This represents a significant compromise since neither reflects accurately the actual disease process seen in patients.
A $500,000 gift from an anonymous donor is helping to change this paradigm by launching the Alzheimer's Induced Pluripotent Stem Cell (iPSC) Initiative Fund at Harvard Medical School. The initiative will enable HMS scientists to generate for the first time millions of patient-specific neurons and to use them to develop and test new drug candidates for the disease.
iPSCs are derived from patient skin samples. The skin fibroblasts are manipulated in the lab to create the stem cells, which are then differentiated into neurons. Although the resulting neuronal cell lines are artificially created, they are faithful, genetic copies of the patient's own neurons and can be used to study the same disruptive processes likely to occur in patients.
"We no longer assume that one Alzheimer's drug will be effective for all patients," says Adrian Ivinson, director of the Harvard NeuroDiscovery Center. "It is more likely that some patients will respond best to one particular drug, while others may respond to a different drug."
"By developing iPSC-derived neurons from many different patients, we will be able to design a drug discovery process that takes into account the known diversity of patients."
Teamwork will be key to success. Acknowledging this, the Harvard NeuroDiscovery Center plans to work with others to generate iPSC-derived neurons from many patient volunteers, and then share those cell lines with other investigators and collaborators.
"Finding effective ways to treat and prevent dementia is one of the crucial challenges of the decade, even while federal support for medical research is being curbed," said the donor. "The Harvard NeuroDiscovery Center is developing new ways to study the brain that effectively leverage the value of our donation to bring a cure closer to reality."
To create and nurture a diverse community
of the best people committed to leadership in
alleviating human suffering caused by disease