A group of scientists released a study in 2006 (a paper that has since been called a “landmark” and cited in thousands of other researchers’ work) hypothesizing that the elusive cause of Alzheimer’s could be traced to a specific protein. Now, the US Department of Justice (DOJ) has reportedly opened an investigation into an Alzheimer’s drug candidate, under suspicion that data could have been falsified.
According to a report following a six-month investigation by journalists with Science magazine, Cassava Sciences had come up with Simulfilam, a drug candidate they claimed benefited Alzheimer’s patients by repairing a protein that blocked sticky brain deposits of the protein in question, amyloid beta. However, two neuroscientists raised suspicions that some data supporting the drug’s effectiveness may have been falsified.
Now, Reuters has reported that DOJ has kicked off a criminal investigation into Cassava Sciences, stating evidence indicates the biotech company may have manipulated research results for its experimental Alzheimer's drug. The case seeks to determine whether the company or individuals have set out to mislead or defraud government agencies, consumers, or investors.
Raymond Tesi, CEO of biotech firm INmune Bio, told Outsourcing-Pharma that he believes the industry’s pursuit of the amyloid-tau theory of Alzheimer’s has been a “folly,” pursued at the detriment of other, more suitable methodologies.
“Science is hypothesis-driven; once a hypothesis is tested and fails, good scientists develop an alternative hypothesis to test,” Tesi commented. “Amyloid became a religious quest. Data that was not consistent with the hypothesis was rejected, instead of the hypothesis being rejected.”
After the DOJ launched its investigation into Cassava Sciences, Tesi added, the rest of the research community and drug development industry is likely to show more caution.
“Journals will become more critical of the publications they review before publications,” he surmised. “This will prevent outright fraud, but it won’t prevent a “wrong” publication—that is, sometimes the conclusions can’t be replicated. There are many reasons for that; most are not due to fraud.”
As for Cassava Sciences, the company has officially denied the allegations of data fraud and manipulation. In a statement on the corporation’s website, president and CEO Remi Barbier said the charges are baseless.
“I have said from the onset that allegations of research misconduct are false,” Barbier wrote. “No government agency has informed us that it has found supporting evidence of research misconduct or any other wrong-doing, and for good reason; there is no supporting evidence for allegations of research misconduct.”
INmune, Tesi said, is developing a protein biologic called XPro1595 that targets neuroinflammation to slow the progression of Alzheimer’s disease. The treatment reportedly has shown promise in reducing inflammation in a Phase I trial, though a next-stage study has been put on pause due to manufacturing concerns.
And, despite the impact the allegations are apt to have on Alzheimer’s research and drug development, the work toward viable treatment will continue, Tesi said.
“Science will march on,” he remarked. “The science of drug development is a hard business with much more failure than success, but when patients benefit, all those frustrations and failures seem trivial.”