A naturally occurring chemical found in soy could prove to be an effective new treatment for a fatal genetic disease that affects children, according to University of Manchester scientists.
Brian Bigger, from the University”s MPS Stem Cell Research Laboratory, found that genistein – derived from soya beans and licensed in the US as an osteoporosis drug – had a dramatic effect on mice suffering from the human childhood disease Sanfilippo.
“Sanfilippo is an untreatable mucopolysaccharide (MPS) disease affecting one in 89,000 children in the United Kingdom,” said Bigger.
“Children with Sanfilippo disease experience progressive deterioration of mental function, similar to dementia, in early childhood, with other symptoms including severe behavioural problems, hyperactivity and ultimately death in early teens,” Bigger said.
In the study mice with Sanfilippo disease were fed with high doses of genistein over a nine-month period.
Treated mice showed a significant delay in their mental decline, including a third reduction in the amount of excess sugars found in the brain as a result of the disease, and a sixth reduction in inflammation in the brain.
Importantly, the research, carried out with colleagues at St Mary”s Hospital in Manchester, also showed that the hyperactivity and other abnormal behaviour normally seen in Sanfilippo mice were fully corrected by genistein treatment.
The study was published in the journal Public Library of Science One.
Sanfilippo syndrome, or Mucopolysaccharidosis III (MPS-III) is a rare autosomal recessive lysosomal storage disease caused by a deficiency in one of the enzymes needed to break down the glycosaminoglycan heparan sulfate (which is found in the extra-cellular matrix and on cell surface glycoproteins).
Although undegraded heparan sulfate is the primary stored substrate, glycolipids such as gangliosides are also stored despite no genetic defect in the enzymes associated with their breakdown.
The condition is named for Sylvester Sanfilippo, the pediatrician who first described the disease.