According to a story from cnbc.com, a group of volunteers in a clinical study drank a concentrated brew that was filled with bacteria. This may sound unpleasant or even dangerous, but these bacteria were not going to infect anyone. They had been specially modified for a specific purpose: to break down phenylalanine. Most people can do this on their own, but in people with phenylketonuria, or PKU, this substance can build up in the body and cause harmful effects.
PKU is a genetic disorder that prevents or reduces the metabolic processing of phenylalanine. This genetic disorder is typically inherited from a person’s parents, and it is caused by mutations that affect the PAH gene. When phenylalanine accumulates within the body, it can have devastating effects. It can cause symptoms such as seizures, intellectual disability, a musty body odor, pale skin, mental disorders, and behavioral issues. In many countries, newborns are screened for this disorder, as prompt intervention is essential to avoid the impact of these symptoms. PKU can be managed effectively with a strict, low protein diet, along with nutritional supplements, that minimizes the introduction of phenylalanine into the body. To learn more about PKU, click here.
The modified bacteria are a product of the field of synthetic biology, which has primarily focused in modifiying bacteria in order to fulfill certain tasks. So far, the medical application of this approach has been fairly limited; however, it has taken off in the industrial field. Modified bacteria are able to churn out molecules important for drug or clothing manufacture.
A New Approach
The bacterial concoction being developed for the treatment of PKU is a project of the company Synlogic, and early tests with mice and monkeys suggest that it could be a viable treatment for the disorder. The latest trial with healthy volunteers also shows promise so far, as all test subjects could tolerate the bacteria and also had phenylalanine in their urine, which suggests that the bacteria appears to be doing its job of processing the substance.
The next clinical trial will truly put the concept to the test to see if the bacteria can effectively lower phenylalanine levels in PKU patients.
Check out the original research here.