Stem Cells Help Evaluate Experimental Alzheimer’s Drugs

Researchers evaluate experimental Alzheimer’s drugs by observing effects on neurons derived from stem cells. PHOTO: GETTY IMAGES

Researchers evaluate new treatments by observing their effects on neurons derived from stem cells

Some Alzheimer’s disease researchers are using stem cells in a novel way—as a model of the disease outside the human body, enabling more efficient testing of experimental drugs in the lab rather than in clinical trials with people.

Finding a drug to slow the onset of Alzheimer’s, the major cause of dementia in older people, has been frustratingly elusive. That is partly because animal testing, where most research begins, doesn’t often produce results that predict very well what will happen to people with Alzheimer’s. And clinical trials of drugs in humans are costly and time consuming.

Though still early days in the field, scientists at the University of California, San Diego, Harvard University and a number of other institutions along with a growing bevy of biotech and pharmaceutical companies, are developing better ways to study the disease outside of the human body and without using animals.

In an early development, scientists at University of California, San Diego used stem cells, which can differentiate into a number of specialized cell types, to create neurons with characteristics of Alzheimer’s. Then they tested two experimental drugs, including one that had previously been withdrawn from a clinical trial because it seemed to worsen disease symptoms rather than help. In testing the drug on the stem cell-derived neurons, the researchers found that for certain patients with a particular genetic mutation, the dosage received in the clinical trial was likely ineffective.

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