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IOM to review Potti research, clinical trials

The Institute of Medicine will conduct a study of the “scientific underpinnings” of three clinical trials that were based on the work of Duke cancer researcher Dr. Anil Potti, the organization announced Wednesday.

However, the scope of the 18-month study, which is anticipated to produce a report by 2012, will extend beyond the evaluation of Potti’s research, said Christine Stencel, media relations officer for the IOM. The study, led by Sharyl Nass, director of the National Cancer Policy Forum at the IOM, will establish criteria for evaluating research on “omics”, such as genomics and proteomics, the study of proteins. Those guidelines will then be used to evaluate the trials based on Potti’s work.

“To be helpful to the research community, it made sense for the study to look into the broader issue [of using genomic predictors in clinical trials],” Stencel said. “[The investigation] is not going to be focused solely on the clinical studies undertaken at Duke.”

Committee formation is currently underway so the number of members is still unknown, Stencel said, adding that those selected will all be volunteers who are experts in their fields.

After reviewing and discussing the published literature, the IOM committee will recommend an evaluation process for determining when predictive tests based on “omics” technologies are suitable for use in clinical trials, according to the study’s web page. In addition, criteria will be established in order to assess predictive tests. These criteria will then be used to assess the predictive models used in the three trials conducted by Duke that were based on Potti’s research. Enrollment in the trials was suspended when concerns about Potti’s work emerged in July.

“The committee may assess the analytical methods used to generate and validate the predictive models, examine how the source data that were used to develop and test the predictive models were generated or acquired, assess the quality of the source data, and evaluate the appropriateness of the use of the predictive models in clinical trials,” the web page states.

Dr. Victor Dzau, chancellor for health affairs and CEO of the Duke University Health System, said he could not say how the IOM study might affect Potti’s status at the University until the study concludes.

The IOM study is one of two ongoing investigations related to Potti’s research. The other is a Duke research misconduct investigation that began after questions were raised regarding Potti’s research and credentials this summer. A separate University investigation which concluded in late August found “issues of substantial concern” in Potti’s resume.

In 2006, Potti and a team of researchers introduced a new gene-based method of predicting how individual patients would respond to certain chemotherapy drugs. The approach stirred debate and excitement in the scientific community because it offered a potential solution to doctors’ inability to predict which drugs patients would respond to best.

However, concerns regarding Potti’s science have contributed to the discussion of the need for standards in genomics.

“Nationally or scientifically, there are no current guidelines or specific steps to my knowledge that exist everywhere that [explain] how one should evaluate these predictors,” Dzau said. “This is not a Duke issue—this is a much broader issue.”

Stencel also noted that there are bigger questions about the issue of using “omic” predictors to personalize medicine. It has been difficult for scientists to find a consistent way of determining whether laboratory discoveries will be useful in a clinical setting, she said.

She added that the IOM study will be useful in determining the best way to handle this kind of data in the future.

Dzau noted that although Duke will give the IOM “everything [it] has” for the investigation, the University will not otherwise be involved. Stencel said Duke will be required to provide all records related to the three clinical trials.

Following the evaluation of the three Duke trials, the committee will issue a report of its findings. The committee will also provide recommendations of criteria for using genomic models in clinical trials, as well as guidelines for ensuring that the scientific community adheres to the proposed process.

Stencel declined to say how the study might affect “omics” fields or Potti.

“We don’t want to speculate on what exactly will come out of this,” Stencel said. “Ultimately, our committees will go where the science and evidence commit them to go.”