Massimo Valicchia/NurPhoto/Corbis

Potential patients have offered vocal support for Staminas stem-cell treatment in Italy.

A series of damning documents seen by Nature expose deep concerns over the safety and efficacy of the controversial stem-cell therapy promoted by Italys Stamina Foundation. The leaked papers reveal the true nature of the processes involved, long withheld by Staminas president, Davide Vannoni. Other disclosures show that the successes claimed by Stamina for its treatments have been over-stated. And, in an unexpected twist, top Italian scientists are dissociating themselves from an influential Miami-based clinician over his apparent support for the foundation.

Stamina, based in Brescia, claims that it successfully treated more than 80 patients, mostly children, for a wide range of conditions, from Parkinsons disease to muscular dystrophy, before the health authorities halted its operations in August 2012. A clinical trial to assess the treatment formally was approved by the Italian government last May, and an expert committee was convened by the health ministry to study Staminas method and to recommend which illnesses the trial should target.

Stamina says that its technique involves extracting mesenchymal stem cells from a patients bone marrow, culturing them so that they turn into nerve cells, and then injecting them back into the same patient. But full details of the method have never been revealed, and Vannoni provided the full protocol to the expert committee only in August.

In October, the committees report prompted health minister Beatrice Lorenzin to halt plans for the clinical trial. That led to public protests in support of Stamina, and, after an appeal by Vannoni, a court ruled in early December that the expert committee was unlawfully biased. Some members had previously expressed negative opinions of the method, the ruling said. As a result, Lorenzin appointed a new committee on 28December, reopening the possibility of a clinical trial.

Staminas protocol, together with the original committees report, was leaked to the press on 20 December (Nature has also been shown transcripts of the committees deliberations). The leaked papers reveal that the original expert committee identified serious flaws and omissions in Staminas clinical protocol. It did not apply legally required Good Manufacturing Practice standards, the committee says. The protocol exposed an apparent ignorance of stem-cell biology and relevant clinical expertise, the report argues, as well as flawed methods and therapeutic rationale (see Protocol opinion).

What the expert committee said on Staminas methods.

The report of the original expert committee tasked with looking at Staminas clinical protocol includes the following opinions:

The protocol contains no method for screening for pathogens such as prions or viruses, even though the culture medium used could contain them.

Link:
Leaked files slam stem-cell therapy