This experimental drug might stop people from getting Alzheimer's
What's the story
A study has shown that an experimental Alzheimer's drug, valiltramiprosate (ALZ-801), may benefit certain high-risk patients. The research, published in the journal Drugs and funded by a $47 million grant from National Institutes of Health, looked at over 300 people aged 50 or older with a genetic predisposition to Alzheimer's. Initially, the drug did not show any significant benefits over placebo treatment. However, a closer examination revealed positive results for a subgroup of 125 participants who had mild cognitive impairment.
Cognitive improvement
Significant responses in mild cognitive impairment subgroup
The subgroup of participants who had mild cognitive impairment, not mild dementia, at the start of treatment showed "very meaningful responses," according to Dr. Susan Abushakra, Chief Medical Officer of Alzheon. The drug slowed cognitive decline by 52% in these patients. This effect is similar to that seen with two other Alzheimer's drugs on the market: lecanemab and donabemab. However, it's hard to quantify ALZ-801's true impact due to the small size of this subgroup.
Brain atrophy
Drug reduced brain atrophy
The study also found that ALZ-801 reduced brain atrophy, or shrinkage, associated with Alzheimer's. In the hippocampus region of the brain, participants who took ALZ-801 had about 18% less atrophy than those on a placebo. This is an important finding as the hippocampus is critical for the memory and thinking.
Drug comparison
ALZ-801 could be safer option for patients
ALZ-801 could get special consideration from the FDA due to its potential benefits over existing drugs. Unlike lecanemab and donabemab, which are monoclonal antibodies administered through intravenous infusions, ALZ-801 is a twice-daily pill that can be taken at home. The current monoclonal antibodies primarily work by breaking down amyloid plaques formed by clumped beta-amyloid proteins. However, Alzheon's product aims to prevent such plaques from forming, avoiding the swelling or bleeding in the brain often seen with monoclonal antibody treatment.
Genetic advantage
Potential benefits for APOE4 gene carriers
The availability of a safer drug like ALZ-801 could benefit people with two copies of the APOE4 gene. These individuals have about 10 times the usual risk of developing Alzheimer's and account for about 15% of all diagnosed cases despite making up only around 2% of the population. They are also more likely to experience side effects from treatment with monoclonal antibodies.