In what is being hailed a “game changer” for Alzheimer’s research, researchers Rudolph E. Tanzi, PhD, and Se Hoon Choi, PhD, both BrightFocus grantees, have done the impossible. Together with their colleagues, they have managed to grow human brain cells in a laboratory for use in Alzheimer’s testing. The discovery is predicted to supplement the traditional practice of testing on mice (known as the mouse model), dramatically improving the speed and lowering the costs of research.
The Benefits of the Petri Dish Model
The new model’s improvements to speed and efficiency will make it possible to test new drug compounds in a fraction of the time and at an estimated one-tenth of the cost of using mice.
Mice’s anatomical similarity to humans has made the mouse model the preferred method of medical testing for decades. However, the actual process for using mice in Alzheimer’s testing is slow, often taking up to a year or more for the signature plaques and tangles of Alzheimer’s to develop. Conversely, the petri dish model starts to develop these plaques and tangles within just a few weeks of the introduction of the Alzheimer’s gene.
The benefits of this new testing method go beyond cost savings. The petri dish model has granted insight into the nature of the course of Alzheimer’s. As mentioned, there are two major components of the Alzheimer’s disease process: the clumping of the proteins known as beta amyloid into plaques and of the tau proteins into tangles. Using the mouse model, scientists had been able to reproduce only the beta amyloid plaques—not the tau tangles. This has limited their ability to test compounds targeting tau, making it a challenge to ascertain the exact course of the disease’s progression. While it has been known that these proteins clump together and form the lethal plaques and tangles that stifle neuronal function, —the petric dish model shows specifically that the disease begins with beta amyloid overproduction, followed by the formation of tau tangles.
There are, of course, limitations to using lab-grown brain cells. For one, they lack various components of living brains, such as immune cells, blood vessels and other cell types that may affect the disease process. In order to develop Alzheimer’s disease very quickly, the petri dish model relies on genes from early onset Alzheimer’s, so if there are differences in late onset and early onset disease, this method may not develop them. As a result, the petri dish model is not expected to totally supplant the mouse model.
Tanzi, a four-time BrightFocus grant recipient, is already using the new method to test 1,200 drugs currently on the market and 5,000 experimental drugs that have made it through the first phase of clinical testing. The next steps are to evaluate dosing and effectiveness, and there is hope that the petri dish approach will narrow the field to only the most promising candidates.
To attempt a screening of this type and at this scale in mouse studies would be nearly impossible, making this an exciting time for Alzheimer’s research.
BrightFocus will continue to fund research into potential disease-modifying targets and therapeutic approaches against Alzheimer’s, as well as screening techniques that might shorten the time it takes to investigate new compounds.
We are committed to advancing the fields of Alzheimer’s, macular degeneration, and glaucoma research. If you’re interested in learning more about promising breakthroughs like the petri dish model, see the research we fund.
This content was last updated on: June 29, 2015