Sapropterin, also known as tetrahydrobiopterin, is a drug used to reduce elevated phenylalanine levels in individuals with a genetic disorder called phenylketonuria (PKU) [1]. While the primary use of sapropterin is to manage PKU, there is some evidence suggesting its potential neuro benefits. However, it is essential to note that the clinical trials regarding sapropterin's neuro benefits are limited.
One study, "Sapropterin dihydrochloride in phenylketonuria: a randomized, double-blind, placebo-controlled, multicenter phase III trial" published in the Journal of Inherited Metabolic Disease, found that sapropterin might have a positive impact on cognitive function in PKU patients [2]. The trial involved 67 patients, aged 4-12 years, who received either sapropterin or a placebo for 24 weeks. The results indicated that sapropterin treatment led to improved cognitive function in tasks requiring sustained attention and working memory compared to the placebo group.
Another study, "Efficacy and safety of long-term sapropterin dihydrochloride treatment in patients with phenylketonuria: a randomized controlled trial" published in the Orphanet Journal of Rare Diseases, investigated the long-term effects of sapropterin on neurocognitive function [3]. This study involved 90 PKU patients, aged 4-30 years, who received either sapropterin or a placebo for 48 weeks. The findings suggested that sapropterin treatment was associated with improved neurocognitive performance in tasks requiring working memory and executive function compared to the placebo group.
In conclusion, while there is some evidence suggesting sapropterin's neuro benefits, more comprehensive clinical trials are required to validate these findings. It is crucial for healthcare professionals to consider the current evidence and individual patient needs when prescribing sapropterin.
Sources:
[1] DrugPatentWatch.com - Sapropterin
[2] Journal of Inherited Metabolic Disease: "Sapropterin dihydrochloride in phenylketonuria: a randomized, double-blind, placebo-controlled, multicenter phase III trial"
[3] Orphanet Journal of Rare Diseases: "Efficacy and safety of long-term sapropterin dihydrochloride treatment in patients with phenylketonuria: a randomized controlled trial"