Purdue-Designed Novel Alzheimer's Drug Goes To Human Clinical Trials

Filed in archive Drugs, Vaccines and Therapeutics on June 30, 2007

Professor Arun Ghosh of Purdue University and Jordan Tang of the Oklahoma Medical Research Foundation developed the drug CTS-21166 - an Alzheimer's disease drug that could prevent and reverse this neurogenerative disease, as opposed to currently available drugs that only manages the symptoms.

The said drug was the result of Tang's identification in 2000 of a beta-secretase, a key enzyme in the progression of Alzheimer's that triggers the formation of amyloid plaques in the brain and Ghosh later building a molecule that binds to the enzyme and inhibits its activity.

According to Professor Ghosh:

"These molecules fit together like puzzle pieces. We created a molecule that fits with a key piece of the Alzheimer's disease puzzle. When the treatment molecule binds to the enzyme, it changes the shape of that puzzle piece so that it no longer fits in its original spot. This halts the chain reaction that leads to the devastating symptoms of Alzheimer's disease.

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. It took years of work and evaluation of hundreds of molecules to achieve one with the strength and safety necessary for clinical potential."

The recent development on this Alzheimer's drug was reported in the May 17 issue of the Journal of Medicinal Chemistry.

The hopeful good news now is that the drug is currently undergoing human clinical trials.

The trial, comprised of 48 healthy volunteers, will measure safety, tolerability and pharmacokinetics of CTS-21166 at various doses.

The company expects to begin generating human clinical data by the end of 2007 and to begin phase II studies in Alzheimer's patients in 2008.

These clinical trial phases are the first steps in a lengthy process necessary before the Food and Drug Administration approves a drug to be available on the market.

According to Ghosh, the drug could be administered at any stage of the disease.

Read the full report.

[In Photo: Shown is an image of the X-ray crystal structure of the molecule bound to the enzyme responsible for amyloid plaque deposits in the human brain. The bond disables the enzyme and prevents the plaques that cause Alzheimer's disease. (Ghosh laboratories image)]