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Clinical trial failure ends controversial trajectory of promising treatment for Alzheimer’s disease

American biopharmaceutical company Cassava Sciences announced that its great prospect for treating Alzheimer’s disease, the experimental drug simufilam, failed the final phase of clinical trials. Contrary to expectations, the drug was unable to neutralize changes in proteins in the brain associated with the onset of the disease. Volunteers who took the compound did not perform better on cognitive tasks than those who were given a placebo.

The failure puts an end to the journey of a drug that has been marked by controversy. In 2022, the journal PLOS ONE retracted five papers by neuroscientist Hoau-Yan Wang of the City University of New York, one of the discoverers of the compound. Two of the papers, coauthored by Lindsay H. Burns, chief scientist at Cassava Sciences, were related to the protein targeted by simufilam. Wang’s work was reviewed by a university panel that identified “egregious misconduct” in relation to his work for Cassava.

The journal Science revealed that the US Food and Drug Administration found flaws in the laboratory procedures adopted by Wang in a simufilam study. He was indicted in June for defrauding the National Institutes of Health, the USA’s leading funding agency for biomedical research, of approximately US$16 million. Two months ago, Cassava Sciences agreed to pay US$40 million to the US Securities and Exchange Commission after it was accused of misleading investors about the results of previous clinical trials of simufilam.

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