wheelchair-bound as the buildup of abnormal proteins along the nerves causes painful neuropathy in their arms and legs, Vaishnaw said. Transthyretin amyloids also lodge in the heart, causing heart disease that can lead to irregular heartbeat and heart failure.
Nearly all transthyretin amyloids are produced by the liver and, up to now, liver transplant has been the only effective treatment for TTR amyloidosis, Vaishnaw said.
The new medication tested in the trials in two versions, called ALN-TTR01 and ALN-TTR02 is delivered via an intravenous infusion and works by inhibiting the genetic process that prompts the creation of transthyretin. Blocking transthyretin production in the liver causes blood levels of the amyloid to drop.
The drug produced no major side effects in the patients tested, Vaishnaw said, noting that one patient did suffer an infusion reaction unrelated to the drug.
“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