Engineered therapy for blood clotting disorder shows early promise
Congenital TTP is characterized by blood clots in small blood vessels throughout the body. If untreated, people with TTP can develop strokes, heart attacks, or kidney damage. Today, the most common therapy for TTP consists of plasma infusions, in which individuals with severe cases of the disease must go to a hospital to receive the blood product from a donor to replenish the missing enzyme in the blood. However, many patients become intolerant to plasma. They develop severe allergic reactions, which make it nearly impossible to treat them. If they can receive treatment, it's under very close supervision and with precautions. "Today, TTP patients are under-treated because of the complications associated with blood plasma infusions, which has remained the standard treatment for at least half a century," said senior study author Bruce Ewenstein, MD, PhD, of Shire in Cambridge, MA. "Plasma as a source of enzyme replacement is a sledgehammer approach to treatment, bu...