The battle for stem cell therapy: a war against pseudoscience

The battle for stem cell therapy: a war against pseudoscience

We paid a high personal price for this war. The past 18 months have been like riding a roller coaster, screaming in hope, disappointment, victory and anger. We spent countless hours communicating with each Other and convincing politicians.

Scientists can get the most out of their long-term work with like-minded partners, but sometimes they also take on other responsibilities, even if it means missing the funding deadline and receiving a threatening letter. Elena Cattaneo, director of the Stem Cell Research Center at the University of Milan in Italy, and Gilberto Corbellini, a bioethicist at the University of Milan in Rome, pointed out in a review article that when loose clinical standards jeopardize Italy's health system and patient health, many people leave comfortable labs and offices. They are one of them when they get evidence.

Since its inception in 2009, the Italian folk organization Stamina Foundation has claimed that stem cells extracted from human bone marrow can become neuronal cells exposed to retinoic acid, an important molecule in embryonic development. The founder of the foundation, Davide Vannoni, believes that injection of these cells can treat Parkinson's disease, muscular dystrophy and spinal muscular atrophy.

Cattaneo and Corbellini stated that Vannoni has not received training as a scientist or physician, nor has he published a peer-reviewed paper. (Using the literature service search system to search for Vannoni's "stem cells" or "neural cells" has not yielded any results.) Cattaneo pointed out in the article: "His laboratory moved around and even left Italy, indicating that Vannoni hopes to A place that manages less strict places."

Many scientists and government officials have found that the Stamina Foundation's stem cell preparation protocol is flawed and that treatment is lacking. Despite this, the Italian National Health Service still paid bills for these treatments, and the Parliament even approved a grant of 3 million euros for its clinical trials.

For most of the past two years, Cattaneo, Corbellini and other researchers (especially stem cell experts Paolo Bianco and Michele De Luca) have strongly opposed these treatments. This made them have to miss the funding deadline and professional meetings. Corbellini said: "We learn to use investigative skills outside of professional disciplines, and we also attach great importance to helping people who are not scientists to seize valuable information."

A recent victory for Cattaneo et al. came from May 28, when the European Court of Human Rights ruled that patients had no power to receive treatment without scientific evidence. But they won't let it go. In early June, the Vice-President of the Stamina Foundation, Marino Andolina, was appointed as an acting member of the Brescia Public Hospital, and a court even allowed him to perform “Stamina treatment” for a child.

Desperate patients are always vulnerable to sales. “We want to share our experience to help other investigators join the camp against pseudoscience,” Cattaneo and Corbellini said.

Committed to the battlefield

In August 2012, Cattaneo and Corbellini began to pay attention to the Stamina Foundation's statement. At that time, the Italian Medicines Agency investigators closed the foundation's operations at Brescia Hospital, which they thought was not safe. However, patients have resorted to the law to require Stamina therapy in the final stage of the disease, and their costs should be included in public health care.

In August 2012, a court ruled that a child with spinal muscular atrophy could receive the therapy. Since then, most of the 500 courts that patients have sought help have decided to support the therapy. In the winter of 2012, Cattaneo et al. began to remind patients, politicians and the media that the treatment lacked management precedents and scientific principles and did not meet the criteria for sympathy.

Together with De Luca and Bianco, Cattaneo and Corbellini began checking network information late at night. They mentioned: "We found that although the Stamina Foundation branded itself as a private charity, its address was a commercial company that was fined for misleading advertising of dietary supplements." So the researchers began collecting evidence. To show that Vannoni tried to lobby government officials and members of Congress to approve management exemptions from its operations and to make the National Health Program cover untested agreements.

Cattaneo et al. found that the Stamina Foundation's patent application was rejected because the US Patent Office considered it lacking specificity and pointed out that under the conditions described, it is unlikely that the collected cells will be induced into specific types of cells. But no one has bothered to dig into the detailed information.

By the beginning of 2013, some people who opposed the Stamina Foundation were vilified by Vannoni and some media, who claimed that the opposition prevented children from receiving life-saving treatment. The evidence that researchers spend months collecting is largely ignored. "We know that there is no sympathy without safety and effectiveness, we need to keep sound, sober and rational," Cattaneo and Corbellini said.

proceed checking

Cattaneo et al. prepared 40 pages of information for each politician they could reach, and the legislature held hearings for supporters and opponents of the Stamina Foundation. Vannoni can't even remember the name of the clinician he worked with.

In May 2013, the government promised to allocate $3.9 million for clinical trials, even though Vannoni did not provide evidence of animal and cellular research. This is a problem: the trial will waste a lot of poor public funds, but some in the Cattaneo camp think it is better than injecting unknown cells into children. At least in rigorous testing, cells will be prepared under strict quality control by an authorized laboratory and the protocol will be reviewed in detail.

In August 2013, Italian President Giorgio Napolitano appointed Cattaneo and Nobel Prize winner in physics, Carlo Rubbia, as Senator of the House of Lords – a position usually reserved for politicians. These appointments are part of Italy’s efforts to strengthen scientific power. As part of the clinical trial requirements, Vannoni revealed its hypothetical method of preparing cells.

But a scientific committee appointed by the country's health minister found that the technique used to evaluate cell characterization was flawed and lacked a basic scan of the pathogen. Early analysis of frozen cells collected by the Stamina Foundation only found blood cells, no nerve cells. In October 2013, the planned trial was cancelled.

In December 2013, another court ruled that members of the committee had publicly opposed the Stamina Foundation, so the results were biased and called for another committee to re-examine the agreement. In the same month, the Minister of Health said that the situation of 36 patients who participated in the treatment of the Foundation did not improve.

Last month, International Stem Cell published an article by Andolina describing a boy with a neurodegenerative disease who injected the relevant cells. The three-page paper did not contain any data, detailed methods and supplements, but claimed that the boy's "action and relationship with parents" improved. At the same time, Cattaneo et al. said that the authors of the above paper claimed that they "had no economic interest", but some scientists expressed concern about writing to the magazine.

At the same time, the Stamina Foundation continues to ferment. In April, after four years of investigation, the prosecutor accused Vannoni of attempting to defraud public funds. But Vannoni insists that he is innocent.

Rights struggle

"We paid a high personal price for this 'war'," Cattaneo and Corbellini said. "The past 18 months have been like riding a roller coaster, screaming in hope, disappointment, victory and anger. We have spent countless hours on each other. Communicate and persuade politicians.” They prepared at least six volumes and dozens of slides, and interviewed and commented on the media almost every week.

“We look at the 'war situation' every morning. When the Stamina Foundation wins media, politicians or management conflicts, we need to change the plan at the last minute.” Since June 2013, Cattaneo, Corbellini, De Luca and Bianco have continued Represented by the university community, professors, scientific organizations, patient associations and other organizations on the Stamina Foundation event. “We never refused. We have estimated that everyone has sacrificed 60 to 80 weeks of lab work time and delayed the papers being submitted. We often need to communicate with students and lab members at night or by mail.”

In a few months, Cattaneo and others received threatening letters and were insulted by those who thought they lacked compassion for those who died. The university they worked for also became the target of mail and computer attacks.

However, support from the international community is invaluable. The International Society for Stem Cell Research awarded the Cattaneo, Bianco and De Luca Advocate Awards, which increased their reputation in Italy; Nobel Prize winner and Stem Cell Research Pioneer Yamanaka also expressed their support.

"It's all worth it," Cattaneo and Corbellini said, now thanks to the European court's ruling and the intervention that the House of Lords launched three months ago, "we hope that these suspicious treatments will soon be banned in Italy." They also called on all scientists to adhere to scientific methods. “Science depends on the public sector and should be committed to the public interest, and we have an obligation to fight for it.”


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