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Drug Shows Promise for Rare Genetic Disorder

New therapy may combat amyloidosis, the potentially deadly disease caused by protein build-up in organs


"This is a very exciting report, but it's also a very early report," said Dr. Raymond Comenzo, director of the Blood Bank and Stem Cell Processing Laboratory at Tufts Medical Center in Boston. "There was clear-cut evidence of safety and of effectiveness in reducing circulating levels of transthyretin."

However, more research will need to be done to show that the drug not only reduces TTR amyloid levels but also helps improve amyloidosis symptoms in patients, Comenzo added.

"One has to wonder what the road ahead is, how will the clinical development process work its way out," Comenzo said. "The [U.S. Food and Drug Administration] is going to want to see a benefit that's measured in terms more than just levels of circulating TTR."

For example, he added, regulators will want to see a reduction in organ damage or an overall improvement in survival rates.

Vaishnaw said he expects to be able to show those kinds of results, given that previous studies have shown that amyloid deposits will begin flushing from a person's organs if the levels of amyloid in the bloodstream decrease dramatically.

"It's allowing the organs to clear the deposits that are already there," Vaishnaw said. "We're hoping that over time we'll allow clearing of the existing deposits, which has been seen in other amyloidosis disorders."

However, it will likely be years before the medication passes through drug trials and receives FDA approval, he added.

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