Acetaminophen: The Case for a Link to Neurodegenerative Diseases

Testing the Hypothesis

Animal models and in vitro models of neurodegenerative and retinal degenerative diseases exist [4]. Acetaminophen can be tested in these model systems.

However, in vivo and in vitro models do not directly mimic human disease. As such the most exact method to evaluate the hypothesis would be a long term prospective cohort trial in humans. Since retrospective questionnaire studies are less expensive and more time efficient such an approach might be more practical. Data mining of existing large databases as was recently done for statin use and neurodegenerative disease is another potential approach [11, 12].

Implications of the Hypothesis

This paper raises the possibility that the widely used analgesic and anti-pyretic, acetaminophen, may be a trigger or co-factor in neurodegenerative and retinal degenerative diseases. Given the huge public health implications that such a link would have, proving (or disproving) this link should have a high priority. Indeed, the US Food and Drug Administration (FDA) is under pressure, primarily due to several recent high profile failures, to increase post-marketing surveillance [13,14].

With the aging of the population in the US and most developed countries, the prevalence and incidence of neurodegenerative and retinal degenerative diseases is at epidemic levels. Elimination of even a single negative co-factor in these disorders would do much to reduce the cost of healthcare as well as relieve much suffering.