Purdue University researchers have begun the first phase of human clinical trials to test the efficacy of an Alzheimer's drug designed in-house.
The drug is based on an inhibitor developed by a team of researchers at the university. The researchers say that it may help intercept and disable the disease at an early stage.
"Millions of people suffer from this devastating disease and treatment options are very limited," said Professor Arun Ghosh, who led the creation of the treatment molecule.
"Current drugs manage the symptoms, but this could be the first disease-modifying therapy. It may be able to prevent and reverse the disease," Ghosh, who did his MSc from MSc, Indian Institute of Technology, Kanpur in 1981.
CoMentis Inc., a San Francisco-based biopharmaceutical firm co-founded by Professor Ghosh and Oklahoma Medical Research Foundation (OMRF) scientist Jordan Tang, is initiating the clinical trials of the experimental drug CTS-21166.
The trial, which comprises of 48 healthy volunteers, will measure safety, tolerability, and pharmacokinetics of CTS-21166 at various doses.
Its results are expected to be out by the end of 2007, and the researchers expect to begin phase II studies in Alzheimer's patients in 2008.
In 2000, Professor Ghosh had built an inhibitor that had the ability to bind to and inactivate the beta-secretase enzyme, which is significant to the progression of Alzheimer's. Since then he has been leading the structure-based design of these inhibitors for therapeutic intervention of the disease.
His most recent work, which led to the creation of beta-secretase inhibitor CTS-21166, was published in the Journal of Medicinal Chemistry.
"The molecule is both highly potent and highly selective, meaning it does not appear to affect other enzymes important to brain function or cause harmful side effects," Professor Ghosh said.
"It took years of work and evaluation of hundreds of molecules to achieve one with the strength and safety necessary for clinical potential," he added.