This site uses cookies to measure how you use the website so it can be updated and improved based on your needs and also uses cookies to help remember the notifications you’ve seen, like this one, so that we don’t show them to you again. If you could also tell us a little bit about yourself, this information will help us understand how we can support you better and make this site even easier for you to use and navigate.

Increased CSF APPs-α levels in patients with Alzheimer disease treated with acitretin


Endres, Kristina, Fahrenholz, Falk, Lotz, Johannes, Hiemke, Christoph, Teipel, Stefan, Lieb, Klaus, Tüscher, Oliver, Fellgiebel, Andreas


Neurology, Volume: 83, No.: 21, Pages.: 1930-1935

Year of Publication



Objective: We investigated induction of α-secretase A disintegrin and metalloprotease 10 (ADAM10) by the synthetic retinoid acitretin (Neotigason; Actavis, München-Riem, Germany) in patients with mild to moderate Alzheimer disease (AD) via measurement of CSF content of α-secretase-derived amyloid precursor protein (APPs-α).; Methods: Twenty-one patients clinically diagnosed with mild to moderate AD received acitretin (30 mg per day) or placebo in a 4-week double-blind study. Primary endpoint was the difference of CSF APPs-α ratios calculated from the APPs-α levels after treatment and at baseline. We monitored safety and tolerability of the treatment. In addition, we assessed biomarkers such as β-amyloid 42 (Aβ42) under treatment conditions.; Results: The acitretin group showed a significant increase in CSF APPs-α levels compared with the placebo group (difference 0.38, 95% confidence interval 0.03-0.72, p = 0.035) within this rather short treatment period. The synthetic retinoid acitretin was overall safe and well tolerated.; Conclusions: Our pilot study highlights that acitretin is able to enhance the nonamyloidogenic APP processing in human patients. Clinical consequences of this regulation should be investigated in larger and longer trials in patients with AD to evaluate acitretin’s potential to serve as a novel therapeutic drug.; Classification Of Evidence: This study provides Class III evidence that in patients with AD, oral acitretin increases CSF APPs-α levels.; © 2014 American Academy of Neurology.

Bibtex Citation

@article{Endres_2014, doi = {10.1212/wnl.0000000000001017}, url = {}, year = 2014, month = {oct}, publisher = {Ovid Technologies (Wolters Kluwer Health)}, volume = {83}, number = {21}, pages = {1930--1935}, author = {K. Endres and F. Fahrenholz and J. Lotz and C. Hiemke and S. Teipel and K. Lieb and O. Tuscher and A. Fellgiebel}, title = {Increased {CSF} {APPs}-~ levels in patients with Alzheimer disease treated with acitretin}, journal = {Neurology} }


acitretin, aged, alzheimer disease, amyloid, amyloid precursor protein secretases, apps, biological markers, cerebrospinal, cerebrospinal fluid, diagnosis, double-blind method, drug therapy, female, fluid, humans, levels, male, middle aged, pilot projects, precursor, prospective studies, protein, therapeutic use, treatment outcome

Countries of Study


Types of Dementia

Alzheimer’s Disease

Types of Study

Randomised Controlled Trial

Type of Outcomes


Type of Interventions

Pharmaceutical Interventions

Pharmaceutical Interventions