Italian Parliament Orders Clinical Trial of Controversial Stem Cell Treatment

ROME—A controversial Italian stem cell therapy that scientists say is unproven will undergo its first solid scientific test. The Italian Senate today voted in favor of a new bill, already approved by the Chamber of Deputies on 16 May, that sets aside €3 million for a clinical trial of the treatment, devised by the Stamina Foundation in Turin. Meanwhile, the foundation can continue treating 12 patients at a hospital in Brescia who are already undergoing the disputed therapy.

"This will probably be the first time that a parliament orders a clinical trial," says Elena Cattaneo, director of UniStem stem cell center at the University of Milan.

The merits of Stamina's treatments have long been under dispute in Italy. The foundation says that it has found a way to transform a patient's own mesenchymal stem cells, derived from bone marrow, into newly minted nerve cells that can be used to treat neurodegenerative diseases such as amyotrophic lateral sclerosis, Parkinson's, and Alzheimer's. But many stem cell scientists have dismissed those claims; the International Society for Stem Cell Research recently said that there is no "compelling evidence from clinical trials that such cells provide benefit to patients with neurological conditions."

Under existing Italian law, unproven stem cell therapies can be administered on a case-by-case basis to patients with untreatable, severe illnesses who have no other options—but only if there are enough published data on safety in internationally recognized journals and if therapies are prepared by authorized hospital labs under the Italian rules for the production of stem cells. Stamina has treated 12 patients at the Spedali Civili, a public hospital in Brescia, since 2011. But in 2012, the Italian Medicines Agency (AIFA) halted the treatments there after it had identified several irregularities.

In March of this year, then-health minister Renato Balduzzi—under severe pressure from patients—expressed his support for the treatment and proposed a law to settle the controversy. The first version of his bill horrified scientists because it provided that the treatment could be administered to thousands of patients, without any prior clinical trials, and apparently outside the European Union's regulation for so-called advanced therapies.

Today, the Italian Senate gave its final green light to an amended bill that will allow Stamina to continue giving the injections to patients whose treatment had already begun in Brescia; the foundation can't accept new patients, however. In addition, AIFA, the Italian National Health Institute, and the National Italian Transplant Centre will lead a €3 million clinical trial of the treatment. The law offers no specifics on the study's setup, or which disease it should target; it does provide for the creation of a scientific board to design the trial.

Davide Vannoni, a psychologist at the University of Udine and the director of the Stamina Foundation, could not be reached today for comment on the Senate vote. In an interview a few weeks ago, Vannoni told ScienceInsider that his treatment is effective against a variety of neurodegenerative disorders and that it is based on in vitro and preclinical studies published in Chinese scientific journals; he did not provide copies of the papers, however. That's not convincing, says Francesca Pasinelli, general manager of Telethon, an Italian nonprofit foundation for the advancement of research into genetic diseases. "The language of the international scientific community is English," she says, adding that even Chinese researchers use English if they seek international credibility.

On his Facebook page, Vannoni today said that his treatment cannot be prepared under international quality standards known as Good Manufacturing Practice, as required by the new law, because this could hamper its efficacy.

Cattaneo says that the trial is the result of Balduzzi's "original mistake" of supporting the therapy. "This is the only thing that could be done at this point," she says. Three million euros is a very large amount, Cattaneo says, considering that stem cell research last received support at the national level in 2009, for only €8 million.

"It's a waste of money," says Massimo Dominici, a cell biologist at the University of Modena and Reggio Emilia. But he adds that the lack of regular support for stem cell research is part of the problem. "If the government would provide enough research funding, we could translate research into [therapies] under scientific rules, rather than this way," Dominici says.