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Has BYU prof found AIDS cure?
Compound could be long-sought breakthrough
By Bob Mims
The Salt Lake Tribune

Researchers, including a BYU scientist, believe they have found a new compound that could finally kill the HIV/AIDS virus, not just slow it down as current treatments do.
And, unlike the expensive, drug cocktails 25 years of research have produced for those with the deadly virus, the compound invented by Paul D. Savage of Brigham Young University appears to hunt down and kill HIV.
Although so far limited to early test tube studies, CSA-54, one of a family of compounds called Ceragenins (or CSAs), mimics the disease-fighting characteristics of anti-microbial and anti-viral agents produced naturally by a healthy human immune system.
Under a study sponsored by Ceragenix Pharmaceuticals, Savage and his colleagues developed and synthesized the compound for Vanderbilt University's School of Medicine. In his Nashville, Tenn., laboratories, Derya Unutmaz, an associate professor of Microbiology and Immunology, tested several CSAs for their ability to kill HIV.
While issuing a cautious caveat about his early results, Unutmaz acknowledged Monday that CSAs could be the breakthrough HIV/AIDS researchers have sought for so long.
"We received these agents [from BYU] in early October and our initial results began to culminate by November 2005. We have since reproduced all our results many times," he said. "We have some preliminary but very exciting results [but] we would like to formally show this before making any claims that would cause unwanted hype."
What studies to date show is a compound that attacks HIV at its molecular membrane level, disrupting the virus from interacting with their primary targets, the "T-helper" class white blood cells that comprise and direct the human immune system. Further, CSAs appear to be deadly to all known strains of HIV.
That would be a welcome development for the estimated 40.3 million people now living with HIV/AIDS globally, including nearly 5 million newly infected in the past year alone.
"We have devoted considerable resources to understand the mechanism of these compounds. We think this knowledge will enable us in collaboration with Dr. Savage to design even better compounds," Unutmaz said.
In addition to being a potential checkmate to HIV, the compounds show indications of being just as effective against other diseases plaguing humankind - among them influenza, possibly even the dread bird flu, along with smallpox and herpes.
Savage said he and his BYU research team had been studying CSAs for eight years, noting the compounds' value against microbial and bacteria infections. It was only a year ago they saw that CSAs killed viruses, too.
"They kill viruses very effectively and in a way paralleling our own, natural defenses," Savage said, noting that beyond the obvious use as a weapon against the AIDS pandemic, CSAs could help many others with non-HIV immune deficiencies.
Further, the compounds appear to have few limits on how they are delivered to patients. Although early indications are for application

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of CSAs with an ointment or cream, pills or injections may also be developed - if the compound gets to market.
BYU and Vanderbilt have jointly filed a patent on CSA technology, which has been licensed exclusively to Ceragenix.
Ceragenix CEO and Chairman Steven Porter said only further research will tell, but he was optimistic about the application of CSAs in the war on HIV/AIDS. There are indications that it could help battle antibiotic- and antiviral-resistance strains of disease as they manifest themselves.
"We are encouraged . . . that CSAs may provide a completely unique family of anti-infectives, potentially active against a wide range of viral, fungal and bacterial targets, including those resistant to current therapies," he said.
Assuming continued positive test results in animal and eventual human trials, Porter estimates it could be three to seven years before the compound is available by prescription. That transition could be accelerated, however, if the Food and Drug Administration should decide to fast-track the drug.
That day is still a long way off, though. First, researchers plan to publish their results in scientific journals, seeking peer review and independent confirmation of their findings. Assuming no flaws are found, several rounds of testing would follow.
Most of the nation's leading AIDS experts were attending the Conference on Retroviruses and Opportunistic Infections in Denver on Monday. The event's policies prohibits on-site news conferences or releases during the conference, and efforts to reach scientists there were not successful.
Of the few AIDS research luminaries reached, all said they preferred not to comment on the Vanderbilt tests until full results are published.
bmims@sltrib.com

l Paul Savage and his Brigham Young University research team have invented CSA-54, a chemical compound that holds the promise of killing the HIV virus.

l CSA-54 is one of a family of compounds called Ceragenins that mimic the disease-fighting characteristics of a healthy human immune system.

l Tests at Vanderbilt University indicate the BYU compound also could be effective against influenza, small pox and herpes.

l Assuming continued positive results, CSA-54 could be available in three to seven years.

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