A small study of people with a rare disorder that prevents them from processing protein is an early attempt at creating “living” medicines.
trenches of World War I, thousands of soldiers on both sides fell ill with dysentery, a diarrheal disease often spread by contaminated water. Curiously, one German soldier deployed in the Balkans didn’t become sick when the rest of his comrades did. When scientist Alfred Nissle isolated a strain ofShigellaE. coli Nissle
—has been used as a probiotic to promote gut health and treat gastrointestinal conditions like inflammatory bowel disease. Now, scientists are genetically engineeringe to perform specific functions in hopes of creating a new class of “living” medicines.from a small study showing that its engineered version of the bacteria provided some benefit to patients with a rare genetic disease called phenylketonuria, or PKU.
In the Phase 2 trial, the company showed that its engineered bacteria, which 20 volunteers drank mixed into liquid, lowered levels of that amino acid.
PKU is typically treated with a carefully restricted low-protein diet. Newborns who test positive are placed on a special formula as soon as possible. Children and adults with the disease must avoid meat, fish, eggs, and dairy products, and many continue to drink a medical formula or take supplements to make sure they’re getting enough nutrients. The disease varies in severity, with some patients being able to consume just a few grams of protein a day.
Aoife Brennan, president and CEO of Synlogic, says the company wants to help patients have more freedom in what they can eat. “They understand the importance of keeping their Phe in control for their brain health. But what they really want is some relief from this incredibly strict diet,” she says.to produce an enzyme found in plants, yeast, and other bacteria whose job is to gobble up phenylalanine. They also removed a gene from the bacteria so that it wouldn’t replicate in the gut.
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