Alzheimer’s treatment explored as potential Down’s syndrome therapy

Phase II study finds Alzheimer’s disease-modifying drug is safe for patients with Down’s syndrome, but concludes more research is needed to test effects on cognitive function.

Young man with Down's syndrome

Cognitive impairment in Down’s syndrome shares some features with Alzheimer’s disease (AD), including overproduction of amyloid-beta and high levels of myo-inositol in the brain. People with Down’s syndrome are also at increased risk of developing AD.

In a phase II placebo-controlled study, researchers explored the safety and pharmacokinetics of a candidate disease-modifying therapy for AD, ELND005, in 23 young adults with Down’s syndrome. Participants received 250mg of ELND005 either once or twice daily, or placebo.

After four weeks, there were no adverse events attributed to the study drug and no serious adverse events. There were also no remarkable changes to laboratory findings, vital signs, electrocardiogram results or other physical findings.

The authors, reporting in the Journal of Alzheimer’s Disease
(online, 11 May 2017), say the results show the treatment is well tolerated, but the study wasn’t long enough or powered to detect efficacy. Future studies should examine its effects on cognitive and behavioural measures, they conclude.


[1] Rafii M, Skotko B, McDonough M et al. A randomized, double-blind, placebo-controlled, phase II study of oral ELND005 (scyllo-Inositol) in young adults with Down syndrome without dementia. J Alzheimer’s Dis 2017; 58:401-411. doi: 10.3233/JAD-160965

Last updated
Clinical Pharmacist, CP, August 2017, Vol 9, No 8;9(8):DOI:10.1211/PJ.2017.20203055

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