“In the last ten years we’ve gone from knowing relatively little [about the disease] to knowing something about its beginnings,” he says.

This summer, researchers at the Centre for Research in Neurodegenerative Diseases identified a molecule (scyllo-cyclohexanehexol, or AZD-103) that prevents a protein – beta-amyloid peptide – from fragmenting.

It is thought that Alzheimer disease begins when beta-amyloid peptides fragment and then clump, creating the toxic “plaque” that damages neurons and impairs cognitive functioning.

The discovery of this process and the molecule, in addition to ongoing clinical drug trials (a vaccine that initially proved to be allergic is being reworked), are indicators of the great strides made in understanding the disease, says Dr. St. George-Hyslop.

But, there is a need to be cautious about AZD-103, he says, as the history of medicine generally and drug trials in particular are often marked by slow progress.

“What we will find is that some are effective, some aren’t,” says St. George-Hyslop, explaining that it will be “at least” three to five years before researchers know if drugs like AZD-103 are effective and safe.

“All of these ideas – their potential is quite tremendous, but there are many slips between cup and lip,” he says. “Things that look good initially might have toxicity problems.”

St. George-Hyslop points to gene identification as another critical research direction in the struggle to find ways to mitigate or prevent Alzheimer disease.

His team at the Centre has discovered five genes that would show familial predisposition to the disease.

If research can show who might be predisposed to Alzheimer disease, drug therapy can begin before any early signs are detected.

“To identify these genes is a very important health measure,” says St. George-Hyslop.

Hyslop’s work, which is funded primarily by the Alzheimer Society of Ontario, is encouraging, says the Society’s Executive Director, Linda Stebbins.

It is difficult, because of the tragic nature of the disease and its burden on families, to show caution when a treatment breakthrough appears imminent, says Stebbins.

“Our grand vision is a world without Alzheimer disease,” says Stebbins. “We’re cautious but encouraged.”