“We’ve got 50 million people around the world suffering from these diseases, $650 billion in economic costs, and lots of families like mine that have been affected,” says Cole.
In all of these disorders, proteins that play important roles in the brain become mis-folded. When these mis-folded proteins are put into yeast cells, they die. The first step is to find drug compounds that keep the yeast cells from dying. These are then put into human neurons derived from induced pluripotent stem cells created from people who have the disease.
If the drug seems effective in human cells, it is then put back into yeast to try and figure out what molecular target it affects. By this back-and-forth approach (yeast, then human, then yeast, then human again) it may be possible to find and understand new drugs for the disease.