Researchers from Synologic have found a way to treat a rare inherited disease called phenylketonuria by creating a peppermint concoction filled with billions of bacteria. They did so by manipulating microbes to break down the naturally occurring toxins in the blood.

Phenylketonuria (PKU) is a rare inherited disease where the body rejects a key constituent of an amino acid called phenylalanine. A build-up of phenylalanine in the blood could potentially damage neurons in the brain which could lead to psychiatric disorders, delayed development and intellectual disability.

Patients with PKU have limited treatment options available. Traditionally, treatment for PKU is a low protein diet avoiding meat and cheese along with shakes filled with nutritional supplements.

Synologic’s president and chief executive officer, JC Guitierrez-Ramos said, “It is a serious disease that can be managed, but takes a toll on individuals and certainly, the more we interact with PKU patients and their families, the more we understand what they need. We think that this program could lead to a very important drug, and that’s what we’re working toward.”

Researchers were able to insert genes into the bacteria’s DNA to break down phenylalanine the way our own cells do once they arrive in the gut. The phenylalanine is then sucked up by a pump that is encoded by new gene and broken into fragments. Once the bacteria encode new genes, it then gets released as urine.

Today, drugs to drive down levels of phenylalanine are available. However, they come with their own side effects and only work with a fraction of patients.

The executive director of the National PKU Alliance, Christine S. Brown says, “the current tools that we have available are not good enough”.

Research teams have wanted to use synthetic biology to form microbes that treat us from inside the body.

This bacterial drink could become the first synthetic biology-based medical treatment to receive approval from the Food and Drug Administration. Before this, bacterial treatments have been industrial. This is promising in the scientific field known as scientific biology.

References

• The Scientist. (https://www.the-scientist.com/news-opinion/synthetic-bacteria-help-treat-phenylketonuria-in-mice-64656). August 17, 2018. Diana Kwon. Synthetic Bacteria help Treat Phenylketonuria in Mice.
• RareDR. (https://www.raredr.com/news/synb1618-pku-enters-the-clinic). April 19, 2019. Mathew Shanley. Can Probiotic Bacteria Treat PKU? Potential Therapy Enters the Clinic.
• World AIDS Day. (http://www.worldaidsday.org/about/). About World AIDS Day The New York Times. (https://www.nytimes.com/2018/09/04/health/synthetic-biology-pku.html). September 4, 2018. Carl Zimmer. Scientists Are Retooling Bacteria to Cure Diseases.