Brain supplement choline alfoscerate draws attention for cognitive impairment treatment

Growing interest surrounds the standards used to evaluate treatments for mild cognitive impairment, after a clinical re-evaluation of choline alfoscerate found statistically significant improvements in the per-protocol set — participants who adhered to dosing requirements — as well as in secondary indicators.

Mild cognitive impairment is a pre-dementia condition in which some cognitive functions decline while most daily activities remain intact. Because causes and progression patterns vary widely among patients and the disease advances slowly, differences between treatment and placebo groups may not be clearly apparent in a 48-week clinical trial, experts have noted.

"It is not easy to assess whether cognitive function is maintained or improved over 48 weeks in mild cognitive impairment patients whose cognition is still relatively intact," said Lee Jae-hong, a neurology professor at Asan Medical Center. "Even in the early stages of Alzheimer's dementia, a minimum follow-up of at least 18 months — or 78 weeks — is standard practice for primary efficacy evaluation."

In the current trial, the pre-specified primary endpoint did not meet the threshold for statistical significance. However, analysis of the per-protocol set and secondary indicators showed statistically significant improvements. The degree of improvement also tended to widen as treatment duration and medication adherence increased.

Against that backdrop, the re-evaluation has been assessed as clinically meaningful: even within the relatively short span of 48 weeks, statistically significant improvements were observed in the per-protocol set and secondary indicators.

Kwon Soon-uck, also a neurology professor at Asan Medical Center, said the results "suggest that if a longer-term clinical trial were conducted, the effect of choline alfoscerate on maintaining and improving cognitive function could emerge more clearly."

Choline alfoscerate is an oral medication that is convenient to take and, even under selective benefit coverage, carries a lower cost burden than expensive new drugs. With few adequate alternatives — such as ginkgo biloba preparations or nicergoline — a long-term oral treatment option holds particular importance in managing pre-dementia patients. Long-term data further support the drug's clinical relevance. A 2025 cohort study tracking 510,000 people found that those who took choline had a 10.1 percent lower risk of progressing to Alzheimer's dementia and a 16.8 percent lower risk of progressing to vascular dementia compared with non-users.

"In a super-aged society, a balanced discussion is needed on how to maintain and evaluate realistic treatment options for pre-dementia patients," an industry official said.