A Phase II clinical trial conducted at the Massachusetts General Hospital in the US to evaluate scyllo-inositol (ELND005) for the treatment of young adults with Down syndrome has demonstrated a positive outcome.

Scyllo-inositol was originally being developed as a potential drug candidate to treat Alzheimer’s disease.

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The results indicated that out of the total 23 patients aged 18-45, 22 have completed the trial without significant deviations from the protocol.

The four-week trial compared 250mg of either daily or twice-daily dosages of scyllo-inositol with placebo.

The presence of an additional copy of chromosome 21 reportedly causes Down syndrome, which is characterised by different degrees of intellectual disability and a high risk of Alzheimer’s dementia.

“The results indicated that out of the total 23 patients aged 18-45, 22 have completed the trial without significant deviations from the protocol.”

Excess activity of chromosome 21’s genes such as the amyloid precursor protein is said to contribute to the cognitive challenges associated with Down syndrome, while another chromosome 21 gene affects the metabolism of myo-inositol, which may lead to the severity of symptoms.

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Scyllo-inositol is believed to possess the potential to improve cognition related with Down syndrome by reducing amyloid levels, as well as by regulating myo-inositol dependent signalling in the brain.

Massachusetts General Hospital Down Syndrome Programme co-director and the trial’s site principal investigator Brian Skotko said: “Through this study, members of the Down syndrome community have demonstrated loudly and clearly that they are eager to participate in clinical trials, particularly studies that provide promise for the treatment of Alzheimer’s disease.”

The trial did not show any serious adverse events or changes in physical findings such as vital signs and laboratory tests, and the trial duration is considered to be too short to assess the cognitive or behavioural changes.

Scyllo-inositol is being developed by Transition Therapeutics, while the trial was supported by Elan Pharmeceuticals.