Powerful X-ray technology developed at the U.S. Department of Energy's (DOE's) national laboratories is revealing new insights into diseases ranging from Alzheimer's to the swine flu, and, most recently, enabled the discovery of a groundbreaking new drug treatment for malignant melanoma, the deadliest form of skin cancer. The drug, Zelboraf (vemurafenib), received Food and Drug Administration (FDA) approval on Wednesday. In showing the structures of diseased and disease-causing molecules at their basic level, these extremely bright light sources enable scientists to suggest potential new treatments.
"This technology is a wonderful example of how innovations at our national laboratories lead to discoveries in a wide variety of fields," said Energy Secretary Steven Chu. "In this case, we are pleased to have been involved in research that has shown great promise in the battle against life-threatening melanoma."
An increasing number of drug discovery companies and medical researchers are turning to the powerful X-ray facilities at the DOE national laboratories to probe the causes of disease and develop new treatments. Researchers from Plexxikon Inc., the drug discovery company that developed the melanoma treatment, used X-ray light sources at three national laboratories --SLAC National Accelerator Laboratory, Argonne National Laboratory and Lawrence Berkeley National Laboratory--to determine the specific, 3-D protein structure of a mutated enzyme that tells melanoma cancer cells to multiply uncontrollably.
The researchers used a technique called "macromolecular X-ray crystallography" to develop a drug that would prevent the enzyme from doing this. The newly FDA-approved drug, Zelboraf (vemurafenib), was extremely successful during clinical trials in disrupting the disease and extending the lives of those diagnosed with it.
"Plexxikon's drug discovery approach is critically dependent on harnessing the power of X-ray crystallography, and the role of DOE facilities in enabling the development of compounds like vemurafenib has been fundamental," said Gideon Bollag, Senior Vice President for Research at Plexxikon. "With the insight we gain from the three-dimensional structures, we have an atomic road map to rationally optimize our drug candidates."
In addition to this treatment for melanoma, the extremely bright light sources at these Office of Science labs have revealed new insights into diseases such as Alzheimer's disease, swine flu, autoimmune disorders, bird flu, hepatitis, and the common cold. In showing the structures of diseased and disease-causing molecular machines at their basic level, the tools for discovery enable scientists to suggest potential new treatments and offer brighter hopes for tomorrow.
The Department of Energy supports five of these advanced X-ray light sources, which are typically football field-sized installations optimized to produce precise, high intensity X-ray beams. Scientists from across the nation and around the globe are actively taking advantage of these scientific user facilities at the national laboratories to make discoveries in a diverse array of disciplines ranging from advanced energy research and materials science to biology and medicine.
The Department's Office of Science is the single largest supporter of basic research in the physical sciences in the Unites States, and is working to address some of the most pressing challenges of our time. For more information, please visit the Office of Science website.