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URMC to share $3.4M grant for AIDS drug research

Rochester Business Journal
August 28, 2014

Drugs to fight HIV infections independently developed at the University of Rochester Medical Center and the University of Nebraska Medical Center could prove to be a potent new cocktail to curb the AIDs-causing virus.

Teams of URMC and UNMC scientists have jointly won a $3.4 million National Institutes of Health grant to explore the effectiveness and safety of drugs each team has been independently developing for several years as a combined treatment, URMC officials said Thursday.

In combination, the experimental drugs appear to rid human immune cells of HIV and keep the deadly virus in check for long periods. This could mean an improvement over current HIV treatments, which require patients to take pills daily.

A compound called URMC-099 being developed under a National Institutes of Health grant by a team led by Harris Gelbard M.D., director of the URMC Center for Neural Development and Disease, has been shown in tests on mice genetically engineered to mirror human immune systems to slow HIV-related inflammation and nerve damage.

The UNMC drug is an antiviral agent being developed by UNMC researcher Howard Gendelman, a longtime collaborator of Gelbard’s.

The URMC drug works by inactivating an enzyme called MLK3 that plays a role in HIV-related inflammation. URMC-099 was always intended to be used with a protease inhibitor, the class of anti-viral agents Gendelman’s drug falls into.

Not known was whether the drugs Gendelman and Gelbard were independently working on would fight each other or be able to be safely taken together. When they conducted tests of the drugs joint action, the scientists found that URMC-099 has an unsuspected effect on human immune cells that actually appears to make for a more powerful treatment.

“Our original work showed that inactivating MLK3 had beneficial effects on the brain, but it now appears that the effects are broader,” Gelbard said. “MLK3 inactivation appears to produce beneficial effects in white blood cells that allowed this protease inhibitor to work much more effectively.”
(c) 2014 Rochester Business Journal. To obtain permission to reprint this article, call 585-546-8303 or e-mail

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