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For an unknown number of people, their heart disease is caused by a condition called ATTR amyloidosis.
For this widely under-diagnosed, progressive and often fatal disease, there are few options for treatment.
When the protein transthyretin (TTR) falls apart, it can clump together and morph into amyloids – a cluster of misfolded proteins that stick together. These are acutely dangerous when deposits form in the heart, as they can result in amyloid-TTR amyloidosis (ATTR amyloidosis): a type of heart failure long hidden in the shadows, poorly studied.
Princess Margaret Cancer Centre Senior Scientist Avi Chakrabartty says the problem of ATTR amyloidosis being under-recognized is compounded by the fact that standard heart medications – including blood thinners, statins and digoxin – may not work, and may in fact be harmful.
"There seems to be an unmet need for diagnosis and treatment," Dr. Chakrabartty says. "That was the impetus for starting the research."
His lab discovered an antibody target that is hidden when a protein acts normally, but exposed when it falls apart. Less than a decade later, this set the stage for a novel clinical trial that began in late March.
The trial is not accruing patients at this time.