A team of scientists at the University of Rochester, led by Dr. Harris Gelbard and Stephen Dewhurst, UR vice dean of research, may have developed the first drug to improve memory and cognitive ability in patients with certain types of illnesses.
And a new company, Camber NeuroTherapeutics, has been founded to usher the drug called URMC-099 through human trials in 2016.
Conditions such as multiple sclerosis, Parkinson's, and HIV have a common characteristic. Neuroinflammation of the brain in patients with these illnesses often damages nerve cells and inhibits their normal function, causing memory and cognitive problems. This leaves some patients unable to perform simple tasks.
For instance, Gelbard says that even though HIV is typically thought of as an immune system malfunction, it's also a condition of the brain. When HIV finds its way into the brain, it causes progressive neuroinflammation and numerous cognitive disorders. Even the first description of the neurology of AIDS was of a Parkinson's-like syndrome, Gelbard says.
"One out of two people living with HIV today has some type of brain disease," he says. "There are close to 34 million people living with HIV, which makes it no small problem."
While the inflammation begins early in people with HIV, he says, it's a more gradual process over decades for people with Alzheimer's and Parkinson's.
Post-operative cognitive function is another area where the researchers say that the new drug can have a positive impact. Cognitive function can be impaired after surgery. The problems seem to go away in a few months in some patients, but in others they can last longer or even become permanent.
Gelbard and Dewhurst targeted most of their research on a "worker bee" enzyme called MLK3, which plays a pivotal role in kick-starting the neuroinflammatory process. Their drug, URMC-099, "turns off" the enzyme.
Some of the researchers' work, which is funded by $25 million in grants from the National Institutes of Health, was published in the Journal of Neuroscience and the Journal of Medicinal Chemistry. The articles showed that the inflammation was stopped in studies involving mice.
Camber NeuroTherapeutics is preparing to submit an application to the Food and Drug Administration so that URMC-099 can begin human trials next year. The UR has granted the company, which is based in West Henrietta, the worldwide patent rights to the drug.