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SMA is a hereditary, muscle-wasting disease with no known cure and the most common genetic cause of infant death.
The girl’s case was launched to international attention last month when Pope Francis met and prayed with the family. Italy’s health ministry said last week it was appointing a new panel of experts to assess its protocols governing the use of stem cell treatments for the desperately ill, including small children.
The announcement came shortly after the Lazio Regional Administrative Court suspended a previous federal panel, upholding an appeal from David Vannoni, president of Italy’s Stamina Foundation which supports the stem-cell treatment that critics say has been untested and could be dangerous.
In their order the judges, who have set June 11 for a review of the matter, urged the health ministry to conduct a serious investigation of the so-called Stamina treatment.
The health ministry and the scientific community should give priority to a full investigation “so thorough as to leave no more room for doubt,” the judges said in that case.
Health Minister Beatrice Lorenzin echoed that, saying the new committee must act quickly “because I believe that in this matter no one can leave patients and families in doubt”.
She said the new committee would be appointed very quickly and include foreign experts.
The first expert panel that the Italian government appointed had been asked to come up with a trial design for the therapy.
However, it concluded in September that the Stamina treatment has no scientific foundation and that there was no point in doing the wider study, for which the Italian government had allocated three million euros.
The Stamina Foundation challenged that finding before the Lazio regional court, arguing that the composition of the healthy ministry’s commission had been flawed because the experts were not impartial.
It said that some members of the commission had expressed opposition to Stamina treatment before the commission’s work even began.
Stamina therapy involves extracting bone marrow stem cells from the patient, turning them into neurons by exposing them to retinoic acid for two hours, and injecting them back into the patient.
Supporters of the therapy say it could be a cure for fatal degenerative nerve diseases while detractors say it is devoid of scientific merit.