NIH Suspends Heart Failure Stem Cell Trial After "False Data" Uncovered.
Recent calls for the retraction of journal articles in related fields of cell therapy research have raised concerns about the scientific foundations of the trial, agency officials said.
"While none of the articles in question derive from the CONCERT-HF trial itself, the NHLBI convened CONCERT-HF's Data and Safety Monitoring Board (DSMB) out of an abundance of caution to ensure the study continues to meet the highest standards for participant safety and scientific integrity," NHLBI said in a statement. "Informed by the DSMB recommendations of October 25, 2018, the NHLBI is pausing the trial. While the DSMB did not have any participant safety concerns, this pause enables the DSMB to complete its review."
The agency noted that patient safety in clinical trials is the primary concern.
NHLBI will continue the follow-up protocol during this pause for all participants who have already been treated in the study.
Participants are being notified of the status of the trial and how to request additional information.
The CONCERT-HF trial seeks to determine whether c-kit+ cells, either alone or in combination with mesenchymal stem cells derived from bone marrow, are safe and benefit patients with chronic heart failure, who have very limited treatment options.
Despite significant medical and surgical advances, patients with heart failure continue to experience a low quality of life and about half of them will die within five years of receiving a diagnosis.
The scientific basis of CONCERT-HF is supported by a body of evidence in several preclinical models in a number of studies in a variety of laboratories and was reviewed by a Protocol Review Committee (PRC) independent of the trial.
The cell therapies that CONCERT-HF is testing are under an investigational new drug (IND) designation which is overseen by the FDA. The cells are produced by an accredited laboratory independent of the clinical sites.
In addition, as part of standard oversight of clinical trials, the DSMB routinely reviews and monitors CONCERT-HF to ensure participant safety and that the study continues to ask compelling scientific questions with implications for patient care, NHLBI said.
"The DSMB's review will be conducted as expeditiously as possible and will inform NHLBI's future actions that will ensure the highest standards of participant safety and scientific integrity," the agency stated. "The National Institutes of Health will pause human testing of an experimental stem cell therapy for heart failure while a board charged with overseeing patient safety reviews the taxpayer-funded trial, in light of emerging questions about the scientific foundation for the treatment." [Emphasis added.]
"A years-long Harvard investigation uncovered 'false and/or fabricated data' in 31 scientific papers from the laboratory of Piero Anversa, a researcher whose surprising findings raised hope there were stem cells in the heart that could repair damaged muscle," The Washington Post reported.
The heart cells Anversa identified are being injected into the hearts of some of the trial participants, though Anversa himself is not participating. Further, the scientific basis of the trial came from preclinical animal studies not conducted by Anversa.
David Goff, director of the Division of Cardiovascular Sciences at NHLBI, told The Post that the suspension of the trial came from "an abundance of caution."
"I think this is a reasonable and prudent step," Charles Murry, director of the Institute for Stem Cell and Regenerative Medicine at the University of Washington, told The Post.
Murry has been studying whether heart muscle cells grown from embryonic stem cells can repair heart attack damage.
According to The Post, Anversa did not immediately respond to questions about the trial, but has continually said problems discovered in the lab work involved a long-time colleague. He said he stands by his research.
He continually asserted that problems discovered in his lab came solely from a longtime colleague, with whom he collaborated closely for at least a quarter of a century. He has said the results from his laboratory still stand.
"We were aware of some of the controversies," Victor Dzau, the former chair of the committee that reviewed the initial trial protocol, told The Post. "Patient safety is No. 1, as long as you can make sure there is [patient] consent and patient safety, the issue is we need to know if this whole area is worth pursuing or not."
Richard T. Lee, a stem cell biologist at Harvard, told The Post: "I think it's obviously the responsible thing to do, to pause and reevaluate the scientific basis for a clinical trial like this, and to really carefully ask the question: Is there still a good scientific basis for it. I won't make that judgment, but I think it's quite obvious, you can't do trials on patients when there is a question about the basis."
NOTE: This report was first published in Stem Cell Research News, November 5, 2018.
Contact: Read the full September 29, 2018, Washington Post article by Carolyn Y. Johnson at https://wapo.st/2zdexA8
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|Title Annotation:||Clinical Trials|
|Publication:||Stem Cell Lab World|
|Date:||Nov 12, 2018|
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