Japanese scientists have discovered a chemical compound that could improve the learning ability of people with Down syndrome by helping their nerve cells to develop during intra-uterine growth. Although prenatal screening for Down syndrome exists, no treatment is currently available to normalise the brain functions of these patients. The team led by Masatoshi Hagiwara, a Professor at Kyoto University, selected the compound known as Algernon from over 700 candidate molecules. Tested on iPS cells obtained from the cells of Down syndrome patients, the compound has proved capable of inhibiting the gene that limits neural stem cell proliferation. Furthermore, following oral administration for five days to mice carrying offspring with Down syndrome, the compound triggered normal cerebral cortex formation and prevented the onset of abnormal behaviour in the young.
Initially looking for a compound to regenerate nerve cells, the Masatoshi Hagiwara team will, in future, focus on developing treatment for brain infarction and other disorders presented by individuals with Down syndrome. Their discovery could also lead to treatment for other nervous diseases such as Alzheimer’s or Parkinson’s disease.
The announcement of this discovery should be followed by a publication in the U.S. Scientific Journal Proceedings of the National Academy of Sciences between now and Friday.
 Induced pluripotent stem cells.
The Japan Times (5/09/2017)