There’s an emotional and even dramatic stem cell regulatory scenario unfolding in Italy. It’s quite a dust-up, with patient-rights activists and a small stem cell company championing legalization of an unproven, unpublished cell-replacement therapy. So far, the patient side has the upper hand, using lawsuits and sit-ins to get support in the courts, and even in the Italian Parliament. The 'compassionate use' lobby has managed to mute the outrage of the international mainstream of science; indeed, the scientists have been demonized as uncaring, corrupt tools of big pharma.
The Italian Senate is supposed to take up the situation later this week and could restore sanity. Or not.
The therapy in question is a mixture of bone marrow (mesenchymal) cells developed by a privately owned clinic called the Stamina Foundation. Davide Vannoni, who runs Stamina, freely admits that no published data supports use of the cells, but claims to have treated more than 80 patients with diseases ranging from Parkinson’s disease to muscular dystrophy. Lesioni spinali
, spinal cord injury, is listed on the Stamina website as a treatment category.
Stamina’s cells were being given to a terminally ill child last year but were banned by the Italian version of the FDA (AIFA) after it inspected Stamina’s laboratories. This led to a series of legal challenges by families of patients, and in March, the Italian Health Minister allowed the child to continue using the Stamina treatment. The Ministry then issued a decree on March 21 allowing 32 other patients, mainly children who were already using the treatment, to continue it.
That decree may not stand, but as the Parliament envisions it, the Italian government may actually fund Stamina $3.9 million to get some data to support use of its cells.
A science blogger, a physician who goes by the name Orac
, points out just how mad this is:
… the Italian government is on the brink of giving free rein to stem cell quacks, but it’s worse than that. This order, as the scientists point out [see below], in essence allows government-funded facilities to collaborate with stem cell quacks and facilitate their treating patients.
Here’s a good place to start, with an open letter in the journal EMBO signed by prominent stem cell scientists from eight countries, including the United States: “Regulation of Stem Cell Therapies Under Attack in Europe: For Whom the Bell Tolls
…the Italian case is the first in which unproven ‘stem cell therapies’ may be de facto made legal, rather than being stopped by regulatory bodies and the government. Thus, this is the first case in which unproven stem cell treatments are officially recognized as a bona fide treatment, without having been tested in rigorous clinical trials, and based on flimsy and highly debated preclinical evidence, to be made part of a publicly funded, public health care system.
Say the scientists, the Italian case is of global concern.
The protection of patients from potential fraud was the main reason why drug regulation first arose (first enacted with the Pure Food and Drug Act in 1906, applied to the historical case of ‘snake oil’ (USDA, 1917), and later evolved into the FDA). The Italian case is the first instance in the western world in which this vital regulatory barrier might be breached.
The argument was offered in the Italian case that safety is not a concern in the face of severely ill children or adults, for whom there are no therapeutic alternatives. However, the terminally ill need extra safety and protection, not less. Exposing the weakest people to unknown risks is ethically unacceptable. Recourse to unproven and unsafe therapy is said to be ‘compassion’, or to fall into an arbitrary category of ‘compassionate treatment’. This is not the case at all. Compassion only applies when one offers a safe and potentially effective remedy. That a remedy is effective must be supported by published clinical data. If such data are not available, there is no legitimate assumption of effectiveness in the individual patient, and therefore no ‘compassion’.
More from Orac:
One point that the scientists didn’t really nail, and that’s the issue of informed consent. One notes that Vannoni’s stem cell quackery has no evidence for it published in the peer-reviewed biomedical literature nor any compelling clinical trial results. Consequently, if claims are being made for this treatment it is impossible to give informed consent because there is no evidence upon which to base even a rough estimate of the chances for success weighed against the risks of the treatment. Even worse, we don’t even know that these are really stem cells. Seriously. As the scientists point out, there is no transparency, and if there’s an area of clinical research where transparency is essential, it’s stem cell research.
The journal Nature, which is following the story
, reported today that the Stamina therapy may be reeled in, and only made available in the context of clinical trials. A final decision is expected May 25. Meanwhile, Stamina head Vannoni says he will not comply with the requirement to provide the therapy for trials according to good manufacturing practice.