Alzheimer’s Research on the Cheap

Is it possible? You can’t eliminate costly clinical trials but you might be able to avoid drug development costs, now that the amyloid theory is circling the drain. The trick is to re-purpose existing drugs, but it’s not easy.

Jack H. Florin MD | Your Neurologist in Orange County.

The Alzheimer’s research landscape is littered with massive industry-sponsored phase 3 clinical trials undertaken without prior proof of concept, efficacy evidence or despite negative phase 2 studies. Examples include secretase inhibitors, amyloid-beta antibodies and “small molecules” such as methylene blue.

Add to this heap minocycline, an old, cheap, antibiotic (think acne). A recently completed phase 3 trial failed to show benefit. It can be faulted because it skipped phase 2 but there was preclinical evidence. Nevertheless, it previously failed in trials of MS, ALS, Huntington’s, and schizophrenia. It was, however, pragmatic — not industry-sponsored, with eligibility based on clinical features, not expensive biomarkers.

Ongoing is an expensive phase 3 trial of another repurposed drug, Rybelsus (semaglutide), widely used to treat diabetes (full disclosure — we are a site in the study). Many other drugs with evidence to support use in Alzheimer’s include anti-virals, anti-hypertensives, anti-epileptics, and psychotropic drugs.

Cost-efficient research is possible.

Adapted from the Editorial by Schneider in JAMA Neurology, February 2020.

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