We've only just begun to hit new targets

GENES 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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	Sharing the secrets to fight cancer
	Sharing the secrets to fight cancer

	Any scientist who knows the secrets involved in discovering new cancer drugs and speeding them to patients is likely to be on the guest faculty of Emory’s Drug Development and Pharmacogenomics Academy. Created by the Winship Cancer Institute and the Department of Pharmacology, the academy is all about sharing knowledge. Twice a month, dozens of researchers and clinicians gather at Emory for a lecture by a nationally recognized scientist, followed by an intense round-table discussion. The first speaker was the father of kinase therapy, whose research recently led to development of breakthrough drugs for chronic myelogenous leukemia. Topics range from bench research to improving clinical trials to regulatory policies, but the goal is always the same: new, better, more effective drugs to tackle cancer. And as fast as possible, please. In a spirit of collegiality and the belief that the battle to develop new drugs needs all the good soldiers it can get, Emory has opened the star-studded academy, without charge, to the entire scientific and medical community in Atlanta and the region.



	Hit new targets:


	> FILL EMPYU LAB COATS. Emory’s new NIH-supported drug-screening center has a library of compounds and robotics equipment to test these compounds against potential cancer-causing targets. What they need now to make the center more operational is people. Two scientists to use the robotics equipment for drug discovery for two years cost $300,000. A center director could be endowed in perpetuity for $2 million.


	> GET CANCER'S NUMBER. Number-crunching may not have the TV glamour of test tubes and imaging equipment, but bioinformatics is the Rosetta Stone, the tool that can make sense out of literally billions of bits of biologic data. Costs for biostatistical analysis mount quickly. A typical study, to determine causes of a specific type of lung cancer, for example, costs roughly $115,000. Equipping and staffing a full-scale biostatistics center dedicated to cancer could be done for $2 million.


	> PICK A POISON. Researchers at the Winship Cancer Institute are developing the first large-scale effort to link genetic, clinical, and other factors to predict why some patients experience toxicity to different cancer treatments and others don’t. Help needed: dedicated research time for biostatistical analysis and computer technology, beginning at $25,000. Payoff: eventual big-time federal funding for a study to help patients get maximum benefit from therapies with minimal or no deleterious side effects.


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