Probiotic against phenylketonuria
A genetically modified bacterium is offered as a means of combating hereditary metabolic disease
Marina Astvatsaturyan, Echo of Moscow
A genetically engineered probiotic, which is already undergoing clinical trials, will help patients with phenylketonuria to adhere to a less strict diet than the one thanks to which they maintain their existence. A strict diet is prescribed for people with phenylketonuria in order to prevent the accumulation of the amino acid phenylalanine, which is part of proteins, fraught with severe neurological and cognitive consequences.
Scientists of the Massachusetts biotechnology company Synlogic reported in the journal Nature Biotechnology about the first results of preclinical tests of a genetically modified bacterium that removes phenyalanine from the blood of experimental animals.
The probiotic they created significantly reduced the level of phenylalanine in both model mice with phenylketonuria and healthy monkeys. The experimental drug SYNB1618 is a reprogrammed version of a widely used probiotic, non-pathogenic E. coli, Escherichia coli Nissle, also known as Mutaflor.
The modified probiotic, which the rodents received orally, reduced the level of phenyalanine injected into them by 38 percent compared to control animals that did not receive SYNB1618.
In April of this year, the US Food and Drug Administration (FDA) granted Synlogic's drug the status of accelerated review, and now scientists have already begun the initial phase of its clinical trials on adults with phenylketonuria.
According to Aoife Brennan, a representative of the developer company, cited by The Scientist, probiotic treatment is supposed to be two-stage. The task of the first stage is to keep the level of phenylalanine in the blood under control, at the second stage it should ensure a weakening of the diet and then patients will be able to introduce normal protein food into the diet.
Approaches to the treatment of phenylketonuria are also being developed by other biotech companies. So, earlier this year, the FDA approved replacement therapy with phenylalanine hydrolase, an enzyme that normally metabolizes phenyalanine, but is not produced in sick people. Research is also underway in the field of gene therapy of phenylketonuria.
And Synlogic is currently working on bacterial preparations for the treatment of a number of other metabolic diseases, in particular, urea cycle disorders. Recent preclinical data have confirmed the reliability of Synlogic's chosen "probiotic" platform.
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