GET MOST OF THE ATTENTION, BUT SCIENTISTS KNOW THAT PROTEINS ENCODED BY
THOSE GENES ACTUALLY DO MOST OF THE NITTY-GRITTY WORK OF KEEPING US ALIVE
AND WELL. They also know that these same proteins, gone out of kilter, cause
most of the problems associated with disease, both acquired and inherited.
That knowledge holds out promise for drug therapy: the right molecular compound
can alter what a specific protein does, thus restoring the biochemical balance
that characterizes good health.
Hitting a bulls-eye is not easy. Understanding how any of the proteins in the human body affect a specific disease and identifying a target for intervention are challenging enough. But with hundreds of thousands of known drug-like compounds to test for effectiveness, screening can be even more daunting.
Now it is less so. Emory recently received a $9 million grant from the NIH as one of 10 universities chosen for a new nationwide network of academic drug-screening centers. Using high-throughput robotic technology once available only to the biggest and richest pharmaceutical companies, these universities are searching for drug-like compounds targeted to proteins not being studied by big pharma.
A robotic pipette system picks up minute amounts of liquid containing different compounds and combines them in tiny wells with potential protein targets. A second robot uses self-focusing electron microscopes to observe and record what happens to the protein when combined with the different compounds, and the resulting millions of pieces of data are sorted almost instantaneously through bioinformatics computer technology to find the most active compounds. After re-screening identifies the likeliest candidates, their ability to modify the drug targets as desired can be ascertained. One of Emory’s first goals: to find a protein involved in the cell-to-cell conversation that allows cancer cells to survive and spread, then to identify a compound capable of targeting that particular protein and disrupting the conversation. When the identified compound is modified and transformed into a new drug, it will foil the survival of tumor cells, effectively destroying the cancer. Bulls-eye!
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